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Gene BRAF
Variant V600E
Impact List missense
Protein Effect gain of function
Gene Variant Descriptions BRAF V600E (previously reported as V599E) lies within the activation segment of the kinase domain of the Braf protein (PMID: 15035987). V600E confers a gain of function to the Braf protein as demonstrated by increased Braf kinase activity, downstream signaling, and the ability to transform cells in culture (PMID: 15035987, PMID: 29533785, PMID: 18697864).
Associated Drug Resistance
Category Variants Paths

BRAF mutant BRAF act mut BRAF V600E/K BRAF V600E

BRAF mutant BRAF V600X BRAF V600E/K BRAF V600E

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Transcript NM_004333.6
gDNA chr7:g.140753336A>T
cDNA c.1799T>A
Protein p.V600E
Source Database RefSeq
Genome Build GRCh38/hg38
Transcript gDNA cDNA Protein Source Database Genome Build
XM_005250045 chr7:g.140753336A>T c.1799T>A p.V600E RefSeq GRCh38/hg38
NM_004333.5 chr7:g.140753336A>T c.1799T>A p.V600E RefSeq GRCh38/hg38
NM_001354609.2 chr7:g.140753336A>T c.1799T>A p.V600E RefSeq GRCh38/hg38
NM_004333.6 chr7:g.140753336A>T c.1799T>A p.V600E RefSeq GRCh38/hg38
NM_004333 chr7:g.140753336A>T c.1799T>A p.V600E RefSeq GRCh38/hg38
NM_001378474.1 chr7:g.140753336A>T c.1799T>A p.V600E RefSeq GRCh38/hg38
NM_001378468.1 chr7:g.140753336A>T c.1799T>A p.V600E RefSeq GRCh38/hg38
NM_001354609.1 chr7:g.140753336A>T c.1799T>A p.V600E RefSeq GRCh38/hg38

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
BRAF V600E melanoma sensitive Dabrafenib FDA approved - On Companion Diagnostic Actionable In a Phase III clinical trial (BREAK-3) that supported FDA approval, Tafinlar (dabrafenib) improved median progression-free survival compared to Deticene (dacarbazine) (5.1 vs 2.7 months, HR=0.3, p<0.0001) in patients with BRAF V600E positive melanoma (PMID: 22735384; NCT01227889). detail... detail... 22735384
BRAF V600E melanoma sensitive Vemurafenib Phase I Actionable In a Phase I trial, Zelboraf (vemurafenib) treatment resulted in an overall response rate of 81% (26/32; 2 complete responses, 24 partial responses), and inhibition of tumor Erk and Ccnd1, and reduced cell proliferation in metastatic melanoma patients harboring BRAF V600E (PMID: 20818844; NCT00215605). 20818844
BRAF V600E melanoma sensitive Vemurafenib FDA approved - On Companion Diagnostic Actionable In a Phase III trial (BRIM-3) that supported FDA approval, Zelboraf (vemurafenib), as compared to Deticene (dacarbazine), resulted in an improved overall survival (OS) (13.6 vs 9.7 months, HR=0.81, p=0.03) in patients with BRAF V600E-positive metastatic melanoma, with estimated OS rates of 56%, 30%, 21%, and 17% at 1, 2, 3, and 4 years, respectively (PMID: 28961848, PMID: 21639808; NCT01006980), and BRAF V600E is included on the companion diagnostic (FDA.gov). 28961848 detail... detail... 21639808
BRAF V600E skin melanoma sensitive Ganetespib Preclinical - Cell line xenograft Actionable In a preclinical study, the Hsp90 inhibitor Ganetespib destabilized BRAF, especially BRAF V600E, resulted in loss of cell viability in culture and antitumor effects in cell line xenograft models of melanoma (PMID: 24398428). 24398428
BRAF V600E melanoma sensitive RXDX-105 Preclinical - Cell line xenograft Actionable In preclinical studies, CEP-32496 (RXDX-105) reduced tumor volume and promoted tumor regression in xenograft models of a BRAF V600E mutant human melanoma cell line (PMID: 22319199). 22319199
BRAF V600E colon carcinoma sensitive RXDX-105 Preclinical - Cell line xenograft Actionable In preclinical studies, CEP-32496 (RXDX-105) reduced tumor volume and promoted tumor regression in xenograft models of a BRAF V600E mutant human colon carcinoma cell line (PMID: 22319199). 22319199
BRAF V600E Advanced Solid Tumor sensitive PLX8394 Preclinical Actionable In a preclinical study, PLX8394 had been shown to block survival and growth of vemurafenib/PLX4720-resistant cells harboring distinct BRAF V600E splice variants (PMID: 24422853). 24422853
BRAF V600E melanoma sensitive PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, PLX4720 inhibited growth of melanoma cells harboring BRAF V600E in culture and in cell line xenograft models (PMID: 18287029). 18287029
BRAF V600E melanoma no benefit Sorafenib Phase II Actionable In a Phase II study, Nexavar (sorafenib) displayed negligible efficacy in melanoma patients with BRAF V600E mutations (PMID: 16880785, PMID: 22394203). 22394203 16880785
BRAF V600E melanoma sensitive Encorafenib Preclinical - Cell culture Actionable In a preclinical study, Braftovi (encorafenib) treatment inhibited Erk phosphorylation and reduced proliferation of melanoma cells harboring monomeric BRAF V600E in culture (PMID: 30559419). 30559419
BRAF V600E melanoma sensitive Encorafenib Preclinical - Cell line xenograft Actionable In preclinical studies, Encorafenib (LGX818) treatment of human melanoma xenograft models with BRAF V600E significantly decreased Mek activation and resulted in tumor regression (Cancer Res: 72(8) Suppl 1, Abstract #3790). detail...
BRAF V600E lung carcinoma resistant Dasatinib Preclinical Actionable In a preclinical study, Sprycel (dasatinib) failed to induce apoptosis in lung carcinoma cells expressing BRAF V600E (PMID: 22649091). 22649091
BRAF V600E lung non-small cell carcinoma sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, a non-small cell lung cancer patient harboring a BRAF V600E mutation had a complete response after treatment with Zelboraf (vemurafenib) (PMID: 23733758). 23733758
BRAF V600E lung non-small cell carcinoma sensitive Vemurafenib Phase II Actionable In a Phase II trial (MyPathway), Zelboraf (vemurafenib) treatment resulted in an objective response of 43% (6/14, 1 complete response, 5 partial response) in patients with non-small cell lung cancer harboring BRAF V600E, and stable disease lasting more than 120 days in 2 patients (PMID: 29320312; NCT02091141). 29320312
BRAF V600E lung non-small cell carcinoma sensitive Vemurafenib Phase II Actionable In a Phase II trial (VE-BASKET), responses were seen in patients with non-small cell lung cancer harboring BRAF V600E (n=63) when treated with Zelboraf (vemurafenib), including 23 patients with a partial response (PMID: 32029534; NCT01524978). 32029534
BRAF V600E lung non-small cell carcinoma sensitive Vemurafenib Clinical Study Actionable In a retrospective analysis, non-small cell lung cancer patients harboring BRAF V600E achieved an overall response rate of 54% (13/24, 2 complete responses, 11 partial responses, and 10 with stable disease) and a disease control rate of 96% following treatment with Zelboraf (vemurafenib) (PMID: 26200454). 26200454
BRAF V600E lung non-small cell carcinoma sensitive Vemurafenib Guideline Actionable Zelboraf (vemurafenib) is included in guidelines as a first-line therapy for advanced or metastatic non-small cell lung cancer patients harboring BRAF V600E mutations who can not tolerate the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) (NCCN.org). detail...
BRAF V600E lung non-small cell carcinoma sensitive Dabrafenib Phase II Actionable In a Phase II trial, 33% (26/78) of previously treated non-small cell lung carcinoma patients harboring BRAF V600E demonstrated an overall response, which included all partial responses, when treated with Tafinlar (dabrafenib) while 67% (4/6) receiving Tafinlar (dabrafenib) as a first-line treatment achieved partial responses (PMID: 27080216; NCT01336634). 27080216
BRAF V600E lung non-small cell carcinoma sensitive Dabrafenib Guideline Actionable Tafinlar (dabrafenib) is in guidelines as a first-line therapy for patients with advanced or metastatic non-small cell lung cancer with BRAF V600E mutations who can not tolerate the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) (NCCN.org). detail...
BRAF V600E melanoma sensitive Refametinib Preclinical - Cell line xenograft Actionable In a preclinical study, Refametinib (BAY86-9766) inhibited growth of melanoma cell lines harboring BRAF V600E in culture and suppressed tumor growth in cell line xenograft models (PMID: 19706763). 19706763
BRAF V600E melanoma sensitive Selumetinib Phase II Actionable In a Phase II trial, a favorable response rate to selumetinib (AZD6244) was observed in mutant BRAF but not BRAF wild-type melanoma patients (PMID: 22048237). 22048237
BRAF V600E melanoma sensitive Ganetespib + TAK-733 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the Hsp90 inhibitor Ganetespib in combination with the MEK1/2 inhibitor TAK-733 resulted in tumor regression in Zelboraf (vemurafenib)-resistant cell line xenograft models of melanoma harboring BRAF V600E (PMID: 24398428). 24398428
BRAF V600E colorectal cancer sensitive Regorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Stivarga (regorafenib) inhibited proliferation of colorectal cancer cells harboring BRAF V600E in culture and suppressed angiogenesis and tumor growth in cell line xenograft models (PMID: 21170960). 21170960
BRAF V600E colorectal cancer sensitive Trametinib Preclinical Actionable In a preclinical study, colorectal cancer cells harboring a BRAF V600E mutation had increased sensitivity to Mekinist (trametinib) compared to other colorectal cancer lines in culture (PMID: 25309914). 25309914
BRAF V600E colorectal cancer sensitive Sorafenib Preclinical Actionable In a preclinical study, colorectal cancer cell lines harboring a BRAF V600E mutation were sensitive to Nexavar (sorafenib) in culture (PMID: 24885690). 24885690
BRAF V600E colorectal cancer sensitive Dabrafenib Preclinical Actionable In a preclinical study, colorectal cancer cell lines harboring a BRAF V600E mutation had increased sensitivity to Tafinlar (dabrafenib) in culture compared to cell lines with wild-type BRAF (PMID: 24885690). 24885690
BRAF V600E thyroid cancer sensitive Lapatinib + Vemurafenib Preclinical Actionable In a preclinical study, the combination of Tykerb (lapatinib) and Zelboraf (vemurafenib) inhibited growth of thyroid cancer cells harboring a BRAF V600E mutation in culture and in BRAF-mutant mouse models of thyroid cancer (PMID: 23365119). 23365119
BRAF V600E colorectal cancer sensitive Lapatinib + Panobinostat Preclinical Actionable In a preclinical study, Tykerb (lapatinib) and Faridak (panobinostat) worked synergistically to inhibit growth of BRAF V600E mutant colorectal cancer cells in culture (PMID: 21464044). 21464044
BRAF V600E colorectal cancer sensitive Gefitinib + Vemurafenib Preclinical Actionable In a preclinical study, the combination of Zelboraf (vemurafenib) and Iressa (gefitinib) decreased the number of viable colorectal cancer cells harboring a BRAF V600E mutation in cell culture (PMID: 22448344). 22448344
BRAF V600E colorectal cancer sensitive Erlotinib + Vemurafenib Phase Ib/II Actionable In a Phase Ib/II trial (EVICT), the combination of Zelboraf (vemurafenib) and Tarceva (erlotinib) resulted in an overall response rate of 16% (5/31; 5 partial responses), a clinical benefit rate of 65% (20/31), a median progression-free survival of 3.9 months, and a median overall survival of 6.3 months in patients with colorectal cancer harboring BRAF V600E (PMID: 36638198). 36638198
BRAF V600E colorectal cancer sensitive Erlotinib + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Zelboraf (vemurafenib) and Tarceva (erlotinib) resulted in improved inhibition of tumor growth in BRAF V600E mutant human colon cancer cell line xenograft models compared to either drug as monotherapy (PMID: 22448344). 22448344
BRAF V600E colorectal cancer sensitive Erlotinib + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Zelboraf (vemurafenib) treatment in combination with Tarceva (erlotinib) enhanced tumor growth inhibition and increased survival in a cell line xenograft model of colorectal cancer harboring BRAF V600E compared to either agent alone (PMID: 22180495). 22180495
BRAF V600E colorectal cancer sensitive Cetuximab + Vemurafenib Case Reports/Case Series Actionable In a clinical case study, the combination of Zelboraf (vemurafenib) and Erbitux (cetuximab) was tolerated and showed clinical benefit in a patient with BRAF V600E mutant colorectal cancer (PMID: 24523613). 24523613
BRAF V600E colorectal cancer sensitive Cetuximab + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, Zelboraf (vemurafenib) and Erbitux (cetuximab) combination treatment inhibited survival of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 27312529). 27312529
BRAF V600E colorectal cancer sensitive Cetuximab + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Zelboraf (vemurafenib) treatment in combination with Erbitux (cetuximab) enhanced tumor growth inhibition and increased survival in a cell line xenograft model of colorectal cancer harboring BRAF V600E (PMID: 22180495). 22180495
BRAF V600E colorectal cancer sensitive Encorafenib Phase I Actionable In a Phase I trial, Encorafenib (LGX818) showed activity in patients with advanced metastatic colorectal cancer harboring a BRAF V600E mutation, resulting in a median progression-free survival of 4 months and a best response of stable disease in 66.7% (12/18) (Ann Oncol (2014) 25 (suppl 4): iv182-iv183). detail...
BRAF V600E colorectal cancer sensitive Refametinib Preclinical Actionable In a preclinical study, Refametinib (BAY86-9766) inhibited growth of colorectal cancer cell lines harboring BRAF V600E in culture and suppressed tumor growth in cell line xenograft models (PMID: 19706763). 19706763
BRAF V600E colorectal cancer sensitive Dabrafenib + Panitumumab Phase I Actionable In a Phase I trial, combination therapy consisting of Tafinlar (dabrafenib) and Vectibix (panitumumab) resulted in an overall response rate of 10% (2/20, 1 complete response, 1 partial response), stable disease in 80% (16/20), and a median progression-free survival of 3.5 months in patients with BRAF V600E colorectal cancer (PMID: 29431699; NCT01750918). 29431699
BRAF V600E colorectal cancer sensitive Dabrafenib + Panitumumab Phase Ib/II Actionable In a Phase Ib/II trial, treatment with the combination of Vectibix (panitumumab) and Tafinlar (dabrafenib) resulted in stable disease in 7/8 colorectal cancer patients harboring a BRAF V600E mutation (J Clin Oncol 32:5s, 2014 (suppl; abstr 3515)). detail...
BRAF V600E colon cancer sensitive PD-0325901 Preclinical - Cell line xenograft Actionable In a preclinical study, PD-0325901 demonstrated antitumor activity against BRAF V600E colon cancer cell line xenografts (PMID: 16273091). 16273091
BRAF V600E colorectal cancer sensitive Panitumumab + Vemurafenib Phase I Actionable In a Phase I trial, 83% (10/12) of patients with colorectal cancer carrying a BRAF V600E mutation demonstrated tumor regression when treated with a combination of Zelboraf (vemurafenib) and Vectibix (panitumumab) (PMID: 25589621). 25589621
BRAF V600E colorectal cancer sensitive Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Selumetinib (AZD6244) decreased tumor growth in a cell line xenograft model of colorectal cancer harboring BRAF V600E (PMID: 23942066). 23942066
BRAF V600E melanoma sensitive Navitoclax + PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, PLX4720 and navitoclax (ABT-263) worked synergistically to inhibit growth and increase apoptosis of BRAF V600E mutant melanoma cells in culture and in xenografts (PMID: 24983357). 24983357
BRAF V600E ovarian cancer sensitive CI-1040 Preclinical - Cell line xenograft Actionable In a preclinical study, CI-1040 inhibited growth of a human ovarian cancer cell line harboring BRAF V600E in culture, and inhibited tumor growth in xenograft models (PMID: 19018267). 19018267
BRAF V600E colon cancer sensitive Gedatolisib Preclinical Actionable In a preclinical study, Gedatolisib (PKI-587) inhibited Braf V600E in vitro and inhibited growth of human colon cancer cells harboring BRAF V600E in culture (PMID: 21325073, PMID: 24042735). 24042735 21325073
BRAF V600E melanoma sensitive CCT196969 Preclinical - Pdx Actionable In a preclinical study, CCT196969 inhibited growth of a human melanoma cell line harboring BRAF V600E in culture, and induced tumor regression in several BRAF V600E-mutant melanoma patient-derived xenograft models (PMID: 25500121). 25500121
BRAF V600E melanoma sensitive CCT196969 Preclinical - Cell culture Actionable In a preclinical study, CCT196969 treatment inhibited viability of a melanoma cell line harboring BRAF V600E in culture (PMID: 31618628). 31618628
BRAF V600E melanoma sensitive CCT241161 Preclinical - Pdx Actionable In a preclinical study, CCT241161 inhibited growth of a human melanoma cell line harboring BRAF V600E in culture, and induced tumor regression in several BRAF V600E-mutant melanoma patient-derived xenograft models (PMID: 25500121). 25500121
BRAF V600E colorectal cancer sensitive CCT196969 Preclinical - Cell culture Actionable In a preclinical study, CCT196969 inhibited growth of BRAF-mutant colorectal cancer cell lines in culture (PMID: 25500121), which have been reported to harbor BRAF V600E (PMID: 15294323). 25500121 15294323
BRAF V600E colorectal cancer sensitive CCT241161 Preclinical Actionable In a preclinical study, CCT241161 inhibited growth of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 25500121, PMID: 15294323). 25500121 15294323
BRAF V600E melanoma sensitive Gefitinib + PLX4720 Preclinical Actionable In a preclinical study, Iressa (gefitinib) in combination with PLX4720 inhibited proliferation and tumorigenicity in human melanoma cell line harboring BRAF V600E and resistant to Braf inhibition in culture and in animal models (PMID: 23242808). 23242808
BRAF V600E melanoma sensitive Dasatinib Preclinical Actionable In a preclinical study, Sprycel (dasatinib) inhibited cell invasion, cell signaling, and proliferation in human melanoma cell lines harboring BRAF V600E that are resistant to Braf inhibition in culture and in animal models (PMID: 23242808). 23242808
BRAF V600E melanoma sensitive Saracatinib Preclinical Actionable In a preclinical study, saracatinib inhibited proliferation of human melanoma cell lines harboring BRAF V600E that are resistant to Braf inhibition in culture (PMID: 23242808). 23242808
BRAF V600E melanoma sensitive S3I-201 Preclinical Actionable In a preclinical study, S3I-201 inhibited cell invasion and Stat3 signaling in human melanoma cell lines harboring BRAF V600E that are resistant to Braf inhibition in culture (PMID: 23242808). 23242808
BRAF V600E hairy cell leukemia sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in complete resolution of the brain lesions after 3 months of treatment, decreased spleen size, and resolution of leukemic retinopathy in a patient with hairy cell leukemia harboring BRAF V600E (PMID: 36713531). 36713531
BRAF V600E hairy cell leukemia sensitive Vemurafenib Phase II Actionable In two Phase II clinical studies, patients with refractory hairy cell leukemia harboring BRAF V600E responded to Zelboraf (vemurafenib) with overall response rates of 96% (25/26) and 100% (24/24) as well as complete response rates of 35% (9/26) and 42% (10/24) with median follow up times of 8 and 12 weeks, respectively (PMID: 26352686). 26352686
BRAF V600E melanoma sensitive BI-69A11 Preclinical Actionable In a preclinical study, BI-69A11 inhibited proliferation and Akt/mTOR signaling in human melanoma cell lines harboring BRAF V600E in culture (PMID: 26603897, PMID: 20531415). 20531415 26603897
BRAF V600E melanoma sensitive SBI-0640756 Preclinical Actionable In a preclinical study, SBI-0640756 inhibited proliferation and Akt/mTOR signaling in human melanoma cell lines harboring BRAF V600E in culture (PMID: 26603897, PMID: 20531415). 20531415 26603897
BRAF V600E melanoma sensitive SBI-0640726 Preclinical Actionable In a preclinical study, SBI-0640726 inhibited proliferation and Akt/mTOR signaling in human melanoma cell lines harboring BRAF V600E in culture (PMID: 26603897, PMID: 20531415). 20531415 26603897
BRAF V600E melanoma sensitive SBI-755199 Preclinical Actionable In a preclinical study, SBI-755199 induced cell death in human melanoma cell lines harboring BRAF V600E in culture (PMID: 26603897). 26603897
BRAF V600E melanoma sensitive SBI-0640756 + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Zelboraf (vemurafenib) in combination with SBI-0640756 inhibited the association of eIF4G1 and eIF4E in Zelboraf (vemurafenib) resistant human melanoma cell lines harboring BRAF V600E in culture and reduced tumor growth in xenograft models (PMID: 26603897). 26603897
BRAF V600E melanoma sensitive E6201 Case Reports/Case Series Actionable In a Phase I trial, a patient with metastatic melanoma and brain metastasis harboring BRAF V600E demonstrated an ongoing near complete response with an overall survival of more than 8 years, and preclinical analysis of melanoma cell lines harboring homozygous or heterozygous BRAF V600E in culture were sensitive to treatment with E6201 (PMID: 30264293). 30264293
BRAF V600E melanoma sensitive E6201 Preclinical Actionable In a preclinical study, E6201 inhibited Mapk pathway activation and proliferation of melanoma cell lines harboring BRAF V600E mutation in culture (PMID: 24448821). 24448821
BRAF V600E thyroid cancer sensitive CLM3 Preclinical Actionable In a preclinical study, CLM3 inhibited growth, Egfr signaling, and CCND1 expression in thyroid cancer cells harboring BRAF V600E in culture (PMID: 24423321). 24423321
BRAF V600E lung non-small cell carcinoma sensitive Navitoclax + Vemurafenib Preclinical Actionable In a preclinical study, Navitoclax (ABT-263) enhanced the inhibitory effect of Zelboraf (vemurafenib) on human non-small cell lung cancer cells harboring BRAF V600E in culture (PMID: 25665005). 25665005
BRAF V600E colon cancer sensitive Navitoclax + Vemurafenib Preclinical Actionable In a preclinical study, Navitoclax (ABT-263) enhanced the inhibitory effect of Zelboraf (vemurafenib) on human colon cancer cells harboring BRAF V600E in culture (PMID: 25665005). 25665005
BRAF V600E melanoma sensitive Navitoclax + Vemurafenib Preclinical Actionable In a preclinical study, Navitoclax (ABT-263) enhanced the inhibitory effect of Zelboraf (vemurafenib) on human melanoma cells harboring BRAF V600E in culture (PMID: 25665005). 25665005
BRAF V600E lung non-small cell carcinoma sensitive Navitoclax + Trametinib Preclinical Actionable In a preclinical study, Navitoclax (ABT-263) enhanced the inhibitory effect of Mekinist (trametinib) on human non-small cell lung cancer cells harboring BRAF V600E in culture (PMID: 25665005). 25665005
BRAF V600E colon cancer sensitive Navitoclax + Trametinib Preclinical Actionable In a preclinical study, Navitoclax (ABT-263) enhanced the inhibitory effect of Mekinist (trametinib) on human colon cancer cells harboring BRAF V600E in culture (PMID: 25665005). 25665005
BRAF V600E melanoma sensitive Navitoclax + Trametinib Preclinical Actionable In a preclinical study, Navitoclax (ABT-263) enhanced the inhibitory effect of Mekinist (trametinib) on human melanoma cells harboring BRAF V600E in culture (PMID: 25665005). 25665005
BRAF V600E melanoma sensitive Trametinib + TW-37 Preclinical Actionable In a preclinical study, TW-37 enhanced the inhibitory effect of Mekinist (trametinib) on human melanoma cells harboring BRAF V600E in culture (PMID: 25665005). 25665005
BRAF V600E lung non-small cell carcinoma sensitive Trametinib + TW-37 Preclinical Actionable In a preclinical study, TW-37 enhanced the inhibitory effect of Mekinist (trametinib) on human non-small cell lung cancer cells harboring BRAF V600E in culture (PMID: 25665005). 25665005
BRAF V600E melanoma sensitive TW-37 + Vemurafenib Preclinical Actionable In a preclinical study, TW-37 enhanced the inhibitory effect of Zelboraf (vemurafenib) on human melanoma cells harboring BRAF V600E in culture (PMID: 25665005). 25665005
BRAF V600E lung non-small cell carcinoma sensitive TW-37 + Vemurafenib Preclinical Actionable In a preclinical study, TW-37 enhanced the inhibitory effect of Zelboraf (vemurafenib) on human non-small cell lung cancer cells harboring BRAF V600E in culture (PMID: 25665005). 25665005
BRAF V600E melanoma sensitive Cediranib + PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, PLX4720 and Cediranib (AZD-2171) worked synergistically to inhibit cell growth and induce apoptosis in PLX4720-resistant human melanoma cell lines harboring BRAF V600E in culture and to suppress tumor growth in xenograft models (PMID: 26461489). 26461489
BRAF V600E melanoma sensitive PLX4720 + Selumetinib Preclinical Actionable In a preclinical study, PLX4720 and Selumetinib (AZD6244) worked synergistically to inhibit cell growth in PLX4720-resistant melanoma cell lines harboring BRAF V600E in culture (PMID: 26461489). 26461489
BRAF V600E melanoma sensitive Cediranib + PLX4720 + Selumetinib Preclinical - Cell culture Actionable In a preclinical study, PLX4720, Cediranib (AZD-2171) and Koselugo (selumetinib) worked synergistically to inhibit cell growth in PLX4720-resistant melanoma cell lines harboring BRAF V600E in culture (PMID: 26461489). 26461489
BRAF V600E melanoma sensitive PLX4720 + Tivozanib Preclinical Actionable In a preclinical study, PLX4720 and Tivozanib (AV-951) worked synergistically to inhibit cell growth in PLX4720-resistant melanoma cell lines harboring BRAF V600E in culture (PMID: 26461489). 26461489
BRAF V600E melanoma sensitive BI 882370 + Trametinib Preclinical Actionable In a preclinical study, xenograft models of melanoma harboring BRAF V600E treated with the combination of BI 882370 and Mekinist (trametinib) demonstrated tumor regression with no regrowth during the 5 weeks of treatment (PMID: 26916115). 26916115
BRAF V600E colorectal cancer sensitive BI 882370 + Cetuximab Preclinical Actionable In a preclinical study, colorectal cancer cells harboring BRAF V600E were sensitive to the combination of BI 882370 and Erbitux (cetuximab) in xenograft models, resulting in tumor growth inhibition and partial tumor regression (PMID: 26916115). 26916115
BRAF V600E colorectal cancer sensitive Afatinib + BI 882370 Preclinical Actionable In a preclinical study, colorectal cancer cells harboring BRAF V600E were sensitive to the combination of BI 882370 and Gilotrif (afatinib) in xenograft models, resulting in tumor growth inhibition and partial tumor regression (PMID: 26916115). 26916115
BRAF V600E colorectal cancer sensitive BI 882370 + Trametinib Preclinical Actionable In a preclinical study, colorectal cancer cells harboring BRAF V600E were sensitive to the combination of BI 882370 and Mekinist (trametinib) in xenograft models, resulting in tumor growth inhibition and partial tumor regression (PMID: 26916115). 26916115
BRAF V600E melanoma sensitive DEL-22379 Preclinical - Cell line xenograft Actionable In a preclinical study, DEL-22379 inhibited growth of melanoma cells harboring BRAF V600E with high levels of ERK dimerization in culture and inhibited tumor progression in melanoma xenograft models harboring BRAF V600E (PMID: 26267534). 26267534
BRAF V600E renal cell carcinoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, a patient with metastatic renal cell carcinoma harboring BRAF V600E demonstrated a partial response following treatment with Zelboraf (vemurafenib) (PMID: 26918217). 26918217
BRAF V600E melanoma sensitive SCH772984 Preclinical Actionable In a preclinical study, SCH772984 inhibited growth of melanoma cell lines harboring BRAF V600E in culture (PMID: 26267534). 26267534
BRAF V600E colorectal cancer sensitive PD-0325901 Preclinical Actionable In a preclinical study, PD-0325901 inhibited growth of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 26267534). 26267534
BRAF V600E melanoma conflicting PD-0325901 Preclinical - Cell line xenograft Actionable In a preclinical study, a melanoma cell line xenograft model harboring BRAF V600E treated with PD-0325901 demonstrated stable tumor growth, but by day 44, growth ensued and thus, demonstrated no benefit (PMID: 27488531). 27488531
BRAF V600E melanoma conflicting PD-0325901 Preclinical - Cell culture Actionable In a preclinical study, PD-0325901 inhibited growth of melanoma cell lines harboring BRAF V600E in culture (PMID: 26267534). 26267534
BRAF V600E melanoma conflicting PD-0325901 Preclinical - Cell line xenograft Actionable In a preclinical study, PD-0325901 treatment induced cell cycle arrest and inhibited growth of melanoma cells harboring BRAF V600E in culture (PMID: 25422890). 25422890
BRAF V600E colorectal cancer sensitive SCH772984 Preclinical Actionable In a preclinical study, SCH772984 inhibited growth of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 26267534). 26267534
BRAF V600E colorectal adenocarcinoma sensitive DEL-22379 Preclinical - Pdx Actionable In a preclinical study, DEL-22379 inhibited tumor growth in a colorectal adenocarcinoma patient-derived xenograft model harboring BRAF V600E (PMID: 26267534). 26267534
BRAF V600E melanoma sensitive GSK2126458 Preclinical Actionable In a preclinical study, Omipalisib (GSK2126458) inhibited the growth of melanoma cell lines harboring BRAF V600E in culture (PMID: 22389471). 22389471
BRAF V600E melanoma sensitive GSK2126458 + Trametinib Preclinical Actionable In a preclinical study, Mekinist (trametinib) in combination with Omipalisib (GSK2126458) inhibited growth of melanoma cell lines harboring BRAF V600E in culture (PMID: 22389471). 22389471
BRAF V600E melanoma sensitive Dabrafenib + GSK2126458 Preclinical Actionable In a preclinical study, Tafinlar (dabrafenib) in combination with Omipalisib (GSK2126458) resulted in improved growth inhibition of human melanoma cell lines harboring BRAF V600E in culture (PMID: 22389471). 22389471
BRAF V600E melanoma sensitive Dabrafenib + Trametinib Phase II Actionable In a Phase II trial, BRAF V600E positive melanoma patients who progressed on treatment with BRAF inhibitors or the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) were treated again with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) after 12 weeks off treatment, which resulted in a partial response in 35% (8/25) and stable disease in 40% (10/25) (PMID: 28268064). 28268064
BRAF V600E melanoma sensitive Dabrafenib + Trametinib FDA approved - On Companion Diagnostic Actionable In a Phase III trial (COMBI-v) that supported FDA approval, the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in an improved overall survival rate at 12 months (72% vs 65%, HR=0.69, p=0.005), median progression-free survival (11.4 vs 7.3 months, HR=0.56, p<0.001), and objective response rate (64% vs 51%, p<0.001) compared to Zelboraf (vemurafenib) in melanoma patients harboring BRAF V600E or V600K (PMID: 25399551; NCT01597908). detail... detail... 25399551
BRAF V600E melanoma sensitive Dabrafenib + Trametinib Preclinical Actionable In a preclinical study, Tafinlar (dabrafenib) in combination with Mekinist (trametinib) inhibited growth of melanoma cells harboring BRAF V600E in culture (PMID: 22389471). 22389471
BRAF V600E colorectal cancer sensitive DT01 + Fluorouracil + Oxaliplatin Preclinical - Cell line xenograft Actionable In a preclinical study, DT01 increased sensitivity of human colorectal cancer (CRC) cells harboring BRAF V600E to Eloxatin (oxaliplatin) and Adrucil (5-fluorouracil), and the combination resulted in decreased liver tumor growth in CRC cell line xenograft metastasis models (PMID: 26637369). 26637369
BRAF V600E colon adenocarcinoma not applicable N/A Phase III Emerging In a post-hoc analysis of a Phase III trial, BRAF V600E mutations in colon adenocarcinoma patients with microsatellite stable tumors were associated with a shorter disease-free survival and overall survival compared to those patients with microsatellite instability tumors, suggesting that BRAF V600E may serve as a future prognostic biomarker in this patient population (PMID: 26768652). 26768652
BRAF V600E melanoma sensitive LY3009120 Preclinical Actionable In a preclinical study, LY3009120 inhibited growth, downstream MAPK signaling and soft agar growth in human melanoma cell lines harboring BRAF V600E in culture (PMID: 26343583). 26343583
BRAF V600E melanoma sensitive LY3009120 Preclinical - Cell culture Actionable In a preclinical study, LY3009120 treatment inhibited Erk phosphorylation and reduced proliferation of a melanoma cells harboring either monomeric BRAF V600E or dimeric isoform (p61) of V600E in culture (PMID: 30559419). 30559419
BRAF V600E melanoma sensitive DETD-35 Preclinical - Cell line xenograft Actionable In a preclinical study, DETD-35 inhibited proliferation and colony formation of melanoma cells harboring BRAF V600E in culture and reduced tumor size in xenograft models (PMID: 27048951). 27048951
BRAF V600E melanoma sensitive DETD-35 + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, DETD-35 and Zelboraf (vemurafenib) synergistically inhibited proliferation and colony formation of melanoma cells harboring BRAF V600E in culture and reduced tumor size in xenograft models (PMID: 27048951). 27048951
BRAF V600E neuroendocrine tumor sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Mekinist (trametinib) and Tafinlar (dabrafenib) combination treatment resulted in a rapid and sustained clinical response in a patient with a rectal neuroendocrine tumor harboring a BRAF V600E mutation (PMID: 27048246). 27048246
BRAF V600E neuroendocrine tumor sensitive Dabrafenib + Trametinib Guideline Actionable Mekinist (trametinib) and Tafinlar (dabrafenib) combination therapy is included in guidelines for patients with unresectable or metastatic extrapulmonary poorly differentiated neuroendocrine carcinoma, large or small cell carcinoma, or mixed neuroendocrine/non-neuroendocrine neoplasms harboring BRAF V600E who have progressed on or following prior treatment (NCCN.org). detail...
BRAF V600E neuroendocrine tumor predicted - sensitive Trametinib + Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Mekinist (trametinib) and Zelboraf (vemurafenib) combination treatment resulted in a rapid and sustained clinical response in a patient with a rectal neuroendocrine tumor harboring a BRAF V600E mutation (PMID: 27048246). 27048246
BRAF V600E Advanced Solid Tumor sensitive SB590885 Preclinical Actionable In a preclinical study, SB590885 inhibited inhibited Erk phosphorylation and cell proliferation of transformed cells expression BRAF V600E in culture (PMID: 20538618). 20538618
BRAF V600E Advanced Solid Tumor sensitive RAF265 Preclinical Actionable In a preclinical study, RAF265 inhibited Erk phosphorylation and cell proliferation in BRAF V600E expressing cells in culture (PMID: 20538618). 20538618
BRAF V600E Advanced Solid Tumor sensitive PLX4720 Preclinical Actionable In a preclinical study, PLX4720 inhibited Erk phosphorylation and cell proliferation of transformed cells expression BRAF V600E in culture (PMID: 20538618). 20538618
BRAF V600E Advanced Solid Tumor sensitive CI-1040 Preclinical Actionable In a preclinical study, CI-1040 (PD184352) inhibited Erk phosphorylation and growth of transformed cells expressing BRAF V600E in culture (PMID: 20538618). 20538618
BRAF V600E melanoma sensitive Lifirafenib Preclinical - Cell culture Actionable In a preclinical study, Lifirafenib (BGB-283) inhibited Braf phosphorylation and cell proliferation in melanoma cell lines harboring BRAF V600E in culture (PMID: 26208524). 26208524
BRAF V600E melanoma sensitive Lifirafenib Preclinical - Cell culture Actionable In a preclinical study, Lifirafenib (BGB-283) treatment inhibited viability of a melanoma cell line harboring BRAF V600E in culture (PMID: 31618628). 31618628
BRAF V600E thyroid cancer predicted - sensitive Lifirafenib Case Reports/Case Series Actionable In a Phase I trial, Lifirafenib (BGB-283) treatment resulted in partial response in 15.1% (8/53) of solid tumor patients with BRAF mutations, including 2 thyroid cancer patients harboring BRAF V600E (1 in dose-escalation phase, 1 in the dose-expansion), and in the dose-expansion phase 1 of 3 thyroid cancer patients harboring a BRAF V600 mutation demonstrated a partial response and 2 demonstrated stable disease, resulting in a disease control rate of 100% (3/3) (PMID: 32182156; NCT02610361). 32182156
BRAF V600E melanoma no benefit SHP099 Preclinical Actionable In a preclinical study, SHP099 did not inhibit proliferation or ERK activation in a melanoma cell line harboring BRAF V600E in culture (PMID: 27362227). 27362227
BRAF V600E colorectal cancer resistant SHP099 Preclinical - Cell culture Actionable In a preclincial study, colorectal cancer cell lines harboring BRAF V600E demonstrated resistance to SHP099 in culture (PMID: 27362227). 27362227
BRAF V600E colon cancer resistant Cetuximab Guideline Actionable Erbitux (cetuximab), as a monotherapy, is not indicated for use in colon cancer patients with BRAF V600E (NCCN.org). detail...
BRAF V600E colorectal cancer no benefit Vemurafenib Phase II Actionable In a Phase II trial, Zelboraf (vemurafenib) did not demonstrate meaningful clinical activity as a single agent, resulted in partial response in 5% (1/21) and stable disease in 33% (7/21) of patients with metastatic colorectal cancer harboring BRAF V600E (PMID: 26460303; NCT00405587). 26460303
BRAF V600E colorectal cancer no benefit Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, colorectal cancer cell lines harboring BRAF V600E demonstrated decreased response to Zelboraf (vemurafenib) in culture (PMID: 27312529). 27312529
BRAF V600E colorectal cancer no benefit Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, colorectal cancer cell lines harboring BRAF V600E were not sensitive to growth inhibition by Zelboraf (vemurafenib) in culture or xenograft models, due to feedback activation of EGFR signaling (PMID: 22281684). 22281684
BRAF V600E colorectal cancer sensitive Cetuximab + Selumetinib Preclinical - Cell culture Actionable In a preclinical study, Koselugo (selumetinib) and Erbitux (cetuximab) combination treatment inhibited survival of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 27312529). 27312529
BRAF V600E colorectal cancer sensitive Cetuximab + Encorafenib Guideline Actionable Braftovi (encorafenib) in combination with Erbitux (cetuximab) is included in guidelines as second-line or subsequent therapy for patients with metastatic colorectal cancer harboring BRAF V600E (PMID: 36307056; ESMO.org). 36307056 detail...
BRAF V600E colorectal cancer sensitive Cetuximab + Encorafenib Guideline Actionable Braftovi (encorafenib) in combination with Erbitux (cetuximab) is included in the Pan-Asian Guidelines Adaptation (PAGA) as a second- or subsequent-line therapy for patients with advanced or metastatic colorectal cancer harboring BRAF V600E (PMID: 37236086; ESMO.org). detail... 37236086
BRAF V600E colorectal cancer sensitive Cetuximab + Encorafenib FDA approved - On Companion Diagnostic Actionable In a Phase III (BEACON CRC) trial that supported FDA approval, Braftovi (encorafenib) and Erbitux (cetuximab) combination treatment (n=113) resulted in improved median overall survival (8.4 vs 5.4 months, HR=0.60, p<0.001), confirmed response rate (20% vs 2%, p<0.001), and median progression-free survival (4.2 vs 1.5 months, HR=0.40, p<0.001) compared to control (n=107) in patients with metastatic colorectal cancer harboring BRAF V600E (PMID: 31566309; NCT02928224). detail... 31566309
BRAF V600E colorectal cancer sensitive Cetuximab + Encorafenib Preclinical - Cell culture Actionable In a preclinical study, combination therapy consisting of Erbitux (cetuximab) and Encorafenib (LGX818) inhibited survival of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 27312529). 27312529
BRAF V600E colorectal cancer sensitive Cetuximab + Encorafenib Clinical Study Actionable In a retrospective analysis, real-world treatment with Erbitux (cetuximab) and Braftovi (encorafenib) with or without Mektovi (binimetinib) led to an objective response rate (ORR) of 32.2% (57/201, 2 complete responses (CR)), disease control rate of 71.2% (126/201), median progression-free survival of 4.5 months, and median overall survival of 9.2 months in metastatic colorectal cancer patients with BRAF V600E, with an ORR of 32% (50/180, 1 CR) in patients treated with doublet therapy (PMID: 39255538). 39255538
BRAF V600E colorectal cancer sensitive Cetuximab + Encorafenib Clinical Study Actionable In a retrospective study, combination treatment with Erbitux (cetuximab) and Braftovi (encorafenib) resulted in an objective response rate of 17%, a disease control rate of 65%, a median progression-free survival of 4.6 mo, and a median overall survival of 7.2 mo in patients with metastatic colorectal cancer harboring BRAF V600E (PMID: 35696748). 35696748
BRAF V600E colorectal cancer sensitive Cetuximab + Dabrafenib Preclinical - Cell culture Actionable In a preclinical study, combination therapy consisting of Erbitux (cetuximab) and Tafinlar (dabrafenib) inhibited survival of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 27312529). 27312529
BRAF V600E colorectal cancer sensitive Alpelisib + Cetuximab + Encorafenib Phase Ib/II Actionable In a Phase Ib/II trial, treatment with the combination of Braftovi (encorafenib), Erbitux (cetuximab), and Piqray (alpelisib) resulted in an overall response rate of 18% (5/28), including 5 patients with a partial response, and led to a median progression free survival of 4.2 months and response duration of 12 weeks in colorectal cancer patients harboring BRAF V600E (PMID: 28363909; NCT01719380). 28363909
BRAF V600E colorectal cancer sensitive Alpelisib + Cetuximab + Encorafenib Preclinical - Cell culture Actionable In a preclinical study, combination therapy consisting of Erbitux (cetuximab), Encorafenib (LGX818) and Alpelisib (BYL719) inhibited survival of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 27312529). 27312529
BRAF V600E colorectal cancer sensitive Dabrafenib + Trametinib Phase Ib/II Actionable In a Phase Ib/II trial, treatment with the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) resulted in partial response or better in 12% (5/43), including 1 complete response, and stable disease in 51% (22/43) of patients with colorectal cancer harboring BRAF V600E (PMID: 26392102). 26392102
BRAF V600E colorectal cancer sensitive Dabrafenib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, combination therapy consisting of Tafinlar (dabrafenib) and Mekinist (trametinib) inhibited survival of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 27312529). 27312529
BRAF V600E colorectal cancer sensitive Cetuximab + Dabrafenib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, combination therapy consisting of Erbitux (cetuximab), Tafinlar (dabrafenib), and Mekinist (trametinib) inhibited survival of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 27312529). 27312529
BRAF V600E colorectal cancer sensitive Cetuximab + SCH772984 Preclinical - Cell culture Actionable In a preclinical study, combination therapy consisting of Erbitux (cetuximab) and SCH772984 inhibited survival of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 27312529). 27312529
BRAF V600E colorectal cancer sensitive Dabrafenib + SCH772984 Preclinical - Cell culture Actionable In a preclinical study, combination therapy consisting of Tafinlar (dabrafenib) and SCH772984 inhibited survival of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 27312529). 27312529
BRAF V600E colorectal cancer sensitive Cetuximab + Dabrafenib + SCH772984 Preclinical - Cell culture Actionable In a preclinical study, combination therapy consisting of Erbitux (cetuximab), Tafinlar (dabrafenib), and SCH772984 inhibited survival of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 27312529). 27312529
BRAF V600E melanoma sensitive BI-847325 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with BI-847325 resulted in decreased expression of Mcl-1 and Mek, and inhibited growth of melanoma cell lines harboring BRAF V600E in culture, and inhibited tumor growth in xenograft models of BRAF V600E-positive melanoma, including models with BRAF-inhibitor resistance (PMID: 25873592). 25873592
BRAF V600E thyroid cancer predicted - sensitive Trametinib Preclinical - Cell culture Actionable In a preclinical study, Mekinist (trametinib) inhibited growth of both parental thyroid cancer cell lines harboring BRAF V600E and those acquired Sprycel (dasatinib)-resistance in culture (PMID: 27222538). 27222538
BRAF V600E thyroid cancer sensitive Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Selumetinib (AZD6244) inhibited growth of both parental thyroid cancer cell lines harboring BRAF V600E and those acquired Sprycel (dasatinib)-resistance in culture (PMID: 27222538). 27222538
BRAF V600E thyroid cancer sensitive Dasatinib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, Sprycel (dasatinib) and Mekinist (trametinib) synergistically inhibited proliferation and induced apoptosis in both parental thyroid cancer cell lines harboring BRAF V600E and those acquired Sprycel (dasatinib)-resistance in culture (PMID: 27222538). 27222538
BRAF V600E thyroid cancer sensitive Dasatinib + Selumetinib Preclinical - Cell culture Actionable In a preclinical study, Sprycel (dasatinib) and Koselugo (selumetinib) synergistically inhibited proliferation and induced apoptosis in both parental thyroid cancer cell lines harboring BRAF V600E and those acquired Sprycel (dasatinib)-resistance in culture (PMID: 27222538). 27222538
BRAF V600E thyroid cancer sensitive Dasatinib + SCH772984 Preclinical - Cell culture Actionable In a preclinical study, Sprycel (dasatinib) and SCH772984 synergistically inhibited proliferation and induced apoptosis in both parental thyroid cancer cell lines harboring BRAF V600E and those acquired Sprycel (dasatinib)-resistance in culture (PMID: 27222538). 27222538
BRAF V600E melanoma sensitive PAC-1 + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PAC-1 and Zelboraf (vemurafenib) resulted in a synergistic effect when treating melanoma cells harboring BRAF V600E in culture and xenograft models, demonstrating increased apoptosis and decreased tumor volume (PMID: 27297867). 27297867
BRAF V600E melanoma sensitive PAC-1 + Trametinib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, the addition of PAC-1 to Mekinist (trametinib) and Zelboraf (vemurafenib) led to greater levels of caspase-3 activity and apoptosis in melanoma cells harboring BRAF V600E when compared to Zelboraf (vemurafenib) and Mekinist (trametinib) treatment without PAC-1 (PMID: 27297867). 27297867
BRAF V600E melanoma sensitive GDC0879 Preclinical - Pdx & cell culture Actionable In a preclinical study, GDC0879 inhibited survival of melanoma cell lines harboring BRAF V600E in cell culture, cell line xenograft and patient-derived xenograft (PDX) models (PMID: 19276360). 19276360
BRAF V600E colon cancer sensitive GDC0879 Preclinical - Cell line xenograft Actionable In a preclinical study, GDC0879 inhibited survival of colon cancer cell lines harboring BRAF V600E in cell culture and cell line xenograft models (PMID: 19276360). 19276360
BRAF V600E melanoma sensitive PLX7904 Preclinical - Cell culture Actionable In a preclinical study, PLX7904 inhibited survival of melanoma cell lines harboring monomeric BRAF V600E as well as cells harboring the Zelboraf (vemurafenib)-resistant dimeric BRAF V600E in culture (PMID: 26466569). 26466569
BRAF V600E colorectal cancer sensitive PLX7904 Preclinical - Cell line xenograft Actionable In a preclinical study, PLX7904 inhibited survival of colorectal cancer cells harboring BRAF V600E in culture and demonstrated anti-tumor activity in cell line xenograft models (PMID: 26466569). 26466569
BRAF V600E melanoma sensitive AZ628 Preclinical - Cell culture Actionable In a preclinical study, AZ628 inhibited proliferation of melanoma cell lines harboring either monomeric BRAF V600E or dimeric isoform of V600E which conferred Zelboraf (vemurafenib)-resistance in culture (PMID: 27523909). 27523909
BRAF V600E melanoma sensitive TAK-632 Preclinical - Cell culture Actionable In a preclinical study, TAK-632 inhibited proliferation of melanoma cell lines harboring either monomeric BRAF V600E or dimeric isoform of V600E which conferred Zelboraf (vemurafenib)-resistance in culture (PMID: 27523909). 27523909
BRAF V600E melanoma sensitive TAK-632 Preclinical - Cell culture Actionable In a preclinical study, TAK-632 treatment inhibited Erk phosphorylation and reduced proliferation of a melanoma cells harboring either monomeric BRAF V600E or dimeric isoform (p61) of V600E in culture (PMID: 30559419). 30559419
BRAF V600E colorectal cancer sensitive TAK-632 Preclinical - Cell culture Actionable In a preclinical study, TAK-632 inhibited proliferation of colorectal cancer cells harboring BRAF V600E in culture (PMID: 27523909). 27523909
BRAF V600E thyroid cancer sensitive TAK-632 Preclinical - Cell culture Actionable In a preclinical study, TAK-632 inhibited proliferation of thyroid cancer cells harboring BRAF V600E in culture (PMID: 27523909). 27523909
BRAF V600E melanoma sensitive SB590885 Preclinical - Cell culture Actionable In a preclinical study, SB590885 inhibited proliferation of melanoma cell lines harboring either monomeric BRAF V600E or dimeric isoform of V600E which conferred Zelboraf (vemurafenib)-resistance in culture (PMID: 27523909). 27523909
BRAF V600E thyroid cancer conflicting Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (MyPathway), Zelboraf (vemurafenib) treatment resulted in complete response in a patient with anaplastic thyroid cancer harboring BRAF V600E (PMID: 29320312; NCT02091141). 29320312
BRAF V600E thyroid cancer conflicting Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, thyroid cancer cells harboring BRAF V600E demonstrated decreased sensitivity to Zelboraf (vemurafenib) in culture (PMID: 27523909). 27523909
BRAF V600E colorectal cancer sensitive PLX4720 + TAK-632 Preclinical - Cell line xenograft Actionable In a preclinical study, PLX4720 and TAK-632 combination treatment resulted in durable inhibition of Erk signaling and tumor growth in xenograft models of colorectal cancer cells harboring BRAF V600E (PMID: 27523909). 27523909
BRAF V600E colon cancer sensitive PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, PLX4720 inhibited growth of colon cancer cells harboring BRAF V600E in culture and in cell line xenograft models (PMID: 18287029). 18287029
BRAF V600E colorectal cancer sensitive Lifirafenib Preclinical - Pdx & cell culture Actionable In a preclinical study, Lifirafenib (BGB-283) inhibited Braf phosphorylation and cell proliferation in colorectal cancer cell lines harboring BRAF V600E in culture, and resulted in partial tumor regression in both cell line and patient-derived xenograft models (PMID: 26208524). 26208524
BRAF V600E Advanced Solid Tumor sensitive Lifirafenib Preclinical - Cell culture Actionable In a preclinical study, Lifirafenib (BGB-283) inhibited viability of a variety of cancer cell lines harboring BRAF V600E in culture (PMID: 26208524). 26208524
BRAF V600E colorectal cancer sensitive Cetuximab + Lifirafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Lifirafenib (BGB-283) in combination with Erbitux (cetuximab) demonstrated enhanced tumor suppression in colorectal cancer cell line xenograft models harboring BRAF V600E (PMID: 26208524). 26208524
BRAF V600E colorectal cancer sensitive Pimasertib Preclinical - Cell culture Actionable In a preclinical study, Pimasertib (MSC1936369B) inhibited proliferation of colorectal cancer cells harboring BRAF V600E in culture (PMID: 23629727). 23629727
BRAF V600E colorectal cancer sensitive Everolimus + Pimasertib Preclinical - Cell culture Actionable In a preclinical study, Pimasertib (MSC1936369B) and Afinitor (everolimus) synergistically inhibited proliferation of colorectal cancer cells harboring BRAF V600E in culture (PMID: 23629727). 23629727
BRAF V600E colorectal cancer sensitive Pimasertib + Sorafenib Preclinical - Cell culture Actionable In a preclinical study, Pimasertib (MSC1936369B) and Nexavar (sorafenib) synergistically inhibited proliferation of colorectal cancer cells harboring BRAF V600E in culture (PMID: 23629727). 23629727
BRAF V600E colorectal cancer sensitive Pimasertib + Regorafenib Preclinical - Cell culture Actionable In a preclinical study, Pimasertib (MSC1936369B) and Stivarga (regorafenib) synergistically inhibited proliferation of colorectal cancer cells harboring BRAF V600E in culture (PMID: 23629727). 23629727
BRAF V600E melanoma sensitive RO4987655 Preclinical - Cell culture Actionable In a preclinical study, RO4987655 inhibited proliferation of melanoma cells harboring BRAF V600E in culture (PMID: 26438159). 26438159
BRAF V600E melanoma sensitive RO5126766 Preclinical - Cell culture Actionable In a preclinical study, RO5126766 (VS-6766) treatment induced cell cycle arrest, decreased Mek and Erk phosphorylation, and inhibited growth of melanoma cells harboring BRAF V600E in culture (PMID: 25422890). 25422890
BRAF V600E melanoma sensitive RO5126766 Preclinical - Cell culture Actionable In a preclinical study, RO5126766 inhibited proliferation of melanoma cells harboring BRAF V600E in culture (PMID: 26438159). 26438159
BRAF V600E melanoma no benefit Palbociclib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with Ibrance (palbociclib) in a melanoma cell line xenograft model harboring BRAF V600E resulted in no benefit, demonstrating low but continuous growth (PMID: 27488531). 27488531
BRAF V600E melanoma sensitive Palbociclib + PD-0325901 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination treatment of PD-0325901 and Ibrance (palbociclib) resulted in significant tumor regression in melanoma cell line xenograft models harboring BRAF V600E and demonstrated a 56% (5/9) complete response rate (PMID: 27488531). 27488531
BRAF V600E anaplastic thyroid carcinoma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) and Mekinist (trametinib) combination treatment resulted in a clinical and radiologic response that lasted for 9 months in an anaplastic thyroid cancer patient harboring BRAF V600E (PMID: 27697975). 27697975
BRAF V600E anaplastic thyroid carcinoma sensitive Dabrafenib + Trametinib FDA approved - On Companion Diagnostic Actionable In a Phase II trial (ROAR) that supported FDA approval, the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in an overall response rate of 61% (20/33; 3 complete responses, 17 partial responses) in patients with anaplastic thyroid cancer harboring BRAF V600E, with 12-month duration of response rate of 50%, a median progression-free survival and overall survival of 6.7 and 14.5 months, respectively (PMID: 35026411; NCT02034110). 35026411 detail... detail... detail...
BRAF V600E anaplastic thyroid carcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines for metastatic thyroid gland anaplastic carcinoma patients harboring BRAF V600E (NCCN.org). detail...
BRAF V600E anaplastic thyroid carcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines for patients with advanced or metastatic thyroid gland anaplastic carcinoma harboring BRAF V600E (PMID: 31549998, PMID: 35491008; ESMO.org). 31549998 detail... 35491008
BRAF V600E high grade glioma predicted - sensitive Everolimus + Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Selumetinib (AZD6244) and Afinitor (everolimus) synergistically inhibited growth and induced apoptosis in glioma cell lines in culture, resulted in prolonged survival in cell line xenograft models (PMID: 27217440). 27217440
BRAF V600E high grade glioma predicted - sensitive PLX4720 + Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Selumetinib (AZD6244) and PLX4720 synergistically inhibited growth and induced apoptosis in glioma cell lines in culture, resulted in prolonged survival in cell line xenograft models (PMID: 27217440). 27217440
BRAF V600E high grade glioma predicted - sensitive Everolimus + PLX4720 Preclinical - Cell culture Actionable In a preclinical study, Afinitor (everolimus) and PLX4720 synergistically inhibited growth and induced apoptosis in glioma cell lines in culture (PMID: 27217440). 27217440
BRAF V600E papillary thyroid carcinoma sensitive RO5126766 Preclinical Actionable In a preclinical study, RO5126766 inhibited Mek signaling and increased radioiodide uptake and response in transgenic animal models of papillary thyroid carcinoma driven by BRAF V600E (PMID: 27669459). 27669459
BRAF V600E melanoma sensitive Imatinib + PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Gleevec (imatinib) and PLX4720 enhanced antitumor efficacy of melanoma cells harboring BRAF V600E, demonstrating decreased cell survival in xenograft models, and decreased phosphorylation of Mapk1 in culture (PMID: 27924459). 27924459
BRAF V600E melanoma sensitive Alpelisib + PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Alpelisib (BYL719) and PLX4720 enhanced antitumor activity in a melanoma cell line harboring BRAF V600E, demonstrating decreased cell survival and increased apoptotic activity in xenograft models, and decreased Akt signaling in culture (PMID: 27924459). 27924459
BRAF V600E melanoma sensitive PLX4720 + Vorinostat Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PLX4720 and Zolinza (vorinostat) resulted in antitumor efficacy in a melanoma cell line harboring BRAF V600E, demonstrating decreased cell survival and increased apoptotic activity in xenograft models, and decreased Rb phosphorylation in culture (PMID: 27924459). 27924459
BRAF V600E melanoma sensitive Doxorubicin + PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PLX4720 and Adriamycin (doxorubicin) resulted in antitumor efficacy in a melanoma cell line harboring BRAF V600E, demonstrating decreased cell survival and increased apoptotic activity in xenograft models, and inhibition of cell growth in culture (PMID: 27924459). 27924459
BRAF V600E melanoma sensitive Erlotinib + PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PLX4720 and Tarceva (erlotinib) resulted in antitumor efficacy in a melanoma cell line harboring BRAF V600E, demonstrating decreased cell survival and increased apoptotic activity in xenograft models and culture (PMID: 27924459). 27924459
BRAF V600E colon cancer resistant Panitumumab Guideline Actionable Vectibix (panitumumab), as a monotherapy, is not indicated for use in colon cancer patients with BRAF V600E (NCCN.org). detail...
BRAF V600E glioblastoma conflicting PD-0325901 Preclinical - Cell culture Actionable In a preclinical study, a glioblastoma cell line harboring BRAF V600E demonstrated a decreased response to treatment with PD-0325901, demonstrating increased viability of CD133 positive cells in culture (PMID: 26573800). 26573800
BRAF V600E glioblastoma conflicting PD-0325901 Preclinical - Cell culture Actionable In a preclinical study, PD-0325901 inhibited growth of glioblastoma cell lines harboring BRAF V600E in culture (PMID: 38714355). 38714355
BRAF V600E glioblastoma decreased response PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, a glioblastoma cell line harboring BRAF V600E demonstrated a decreased response to treatment with PLX4720, demonstrating increased viability of CD133 positive cells in culture and in xenograft models (PMID: 26573800). 26573800
BRAF V600E glioblastoma sensitive BI2536 Preclinical - Cell culture Actionable In a preclinical study, a glioblastoma cell line harboring BRAF V600E demonstrated sensitivity to BI2536 in culture (PMID: 26573800). 26573800
BRAF V600E glioblastoma sensitive BI2536 + PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of BI 2536 and PLX4720 resulted in suppression of downstream signaling, increased apoptotic activity, inhibition of cell proliferation, and tumor growth suppression in glioblastoma cell line xenograft models harboring BRAF V600E (PMID: 26573800). 26573800
BRAF V600E Advanced Solid Tumor predicted - sensitive PF-00477736 + PF3644022 Preclinical - Cell culture Actionable In a preclinical study, Chk1 inhibitor PF-477736 and MK2 inhibitor PF3644022 synergistically inhibited growth of transformed cells over expressing BRAF V600E in culture, while single agent inhibition had no effect (PMID: 26140595). 26140595
BRAF V600E melanoma sensitive ASN003 Preclinical - Cell line xenograft Actionable In a preclinical study, a melanoma xenograft model harboring BRAF V600E demonstrated tumor regression when treated with ASN003 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B100). detail...
BRAF V600E lung non-small cell carcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Combination of Mekinist (trametinib) and Tafinlar (dabrafenib) is included in guidelines as a first-line or subsequent therapy for advanced or metastatic non-small cell lung cancer patients harboring BRAF V600E mutations (NCCN.org). detail...
BRAF V600E lung non-small cell carcinoma sensitive Dabrafenib + Trametinib FDA approved - On Companion Diagnostic Actionable In a Phase II trial that supported FDA approval, treatment with the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) in patients with non-small cell lung cancer harboring BRAF V600E resulted in an overall response rate of 66.7% (38/57) in previously treated patients and 64% (23/36) in untreated patients, versus 33% (26/78) treated with Tafinlar (dabrafenib) alone (PMID: 27080216, PMID: 27283860, PMID: 28919011; NCT01336634). 27283860 detail... 27080216 detail... detail... 28919011
BRAF V600E melanoma sensitive Encorafenib + Ribociclib Phase Ib/II Actionable In a Phase Ib/II trial, Encorafenib (LGX818) and Kisqali (ribociclib) combination treatment resulted in confirmed partial response in 7.1% (2/28), unconfirmed partial response in 10.7% (3/28), and stable disease in 35.7% (10/28) of patients with melanoma harboring BRAF V600E (PMID: 28351928). 28351928
BRAF V600E pilocytic astrocytoma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) treatment resulted in a near complete response and resolution of leptomeningeal dissemination in a patient with pilocytic astrocytoma harboring BRAF V600E (PMID: 28784858). 28784858
BRAF V600E colorectal cancer sensitive Cetuximab + PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PLX4720 and Erbitux (cetuximab) inhibited tumor growth in colorectal cancer cell line xenograft models harboring BRAF V600E (PMID: 22281684). 22281684
BRAF V600E colon cancer predicted - sensitive Cetuximab + Sorafenib Case Reports/Case Series Actionable In a clinical case study, a patient with metastatic colon cancer harboring BRAF V600E demonstrated mixed radiographic response with slight progression in some locations and stable disease in other locations for 7 months following treatment with the combination of Nexavar (sorafenib) and Erbitux (cetuximab) (PMID: 23792568). 23792568
BRAF V600E colorectal cancer sensitive Cobimetinib + Vemurafenib Phase II Actionable In a Phase II trial (TAPUR), the combination of Cotellic (cobimetinib) and Zelboraf (vemurafenib) resulted in an objective response rate of 30% (8/27; all partial responses), a disease control rate of 52% (14/27), a median progression-free survival of 15.7 weeks, and a median overall survival of 38.9 weeks in patients with colorectal cancer harboring BRAF V600E (n=26) or K601E (n=1) (PMID: 36409971; NCT02693535). 36409971
BRAF V600E colorectal cancer sensitive Cobimetinib + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Cotellic (cobimetinib) and Zelboraf (vemurafenib) inhibited tumor growth in a cell line xenograft model of colorectal cancer harboring BRAF V600E (PMID: 28649441). 28649441
BRAF V600E melanoma sensitive INU-152 Preclinical - Cell line xenograft Actionable In a preclinical study, INU-152 reduced MEK and ERK phosphorylation and growth of a melanona cell line harboring BRAF V600E in culture, and inhibited tumor growth in xenograft models (PMID: 28645859). 28645859
BRAF V600E colorectal cancer sensitive INU-152 Preclinical - Cell line xenograft Actionable In a preclinical study, INU-152 inhibited growth of colorectal cancer cell lines harboring BRAF V600E in culture, and reduced tumor growth in BRAF V600E-mutant colorectal cancer cell line xenograft models (PMID: 28645859). 28645859
BRAF V600E melanoma predicted - sensitive Cobimetinib Case Reports/Case Series Actionable In a Phase I trial, Cotellic (cobimetinib) treatment resulted in a confirmed partial response in six melanoma patients harboring BRAF V600E (PMID: 27424159). 27424159
BRAF V600E hairy cell leukemia not applicable N/A Guideline Diagnostic BRAF V600E is diagnostic and aids in distinguishing classic hairy cell leukemia (cHCL) from variant hairy cell leukemia (HCLv) and other B-cell lymphomas and leukemias (PMID: 29118233, NCCN.org). detail... 29118233
BRAF V600E colorectal cancer predicted - sensitive LSN3074753 Preclinical - Pdx Actionable In a preclinical study, LSN3074753 demonstrated modest efficacy in patient-derived xenograft models of colorectal cancer harboring BRAF V600E, resulted in tumor growth inhibition or regression, but relapse upon discontinuation of treatment (PMID: 28611205). 28611205
BRAF V600E melanoma sensitive EBI-907 Preclinical - Cell line xenograft Actionable In a preclinical study, EBI-907 inhibited BRAF and ERK signaling, resulted in growth inhibition of melanoma cells harboring BRAF V600E in culture and in cell line xenograft models (PMID: 26810733). 26810733
BRAF V600E colorectal cancer sensitive EBI-907 Preclinical - Cell line xenograft Actionable In a preclinical study, EBI-907 inhibited BRAF and ERK signaling, resulted in growth inhibition of colorectal cancer cells harboring BRAF V600E in culture and in cell line xenograft models (PMID: 26810733). 26810733
BRAF V600E melanoma sensitive Cobimetinib + Vemurafenib FDA approved - On Companion Diagnostic Actionable In a Phase III trial (coBRIM) that supported FDA approval, treatment with the combination of Zelboraf (vemurafenib) and Cotellic (cobimetinib) resulted in an improved progression-free survival of 12.3 months, compared to 7.2 months with Zelboraf (vemurafenib) plus placebo, among patients with BRAF V600-mutated metastatic melanoma, and BRAF V600E and BRAF V600K are on the companion diagnostic (PMID: 27480103; NCT01689519). 27480103 detail... detail...
BRAF V600E melanoma decreased response Dabrafenib + SCH772984 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, combination of Tafinlar (dabrafenib), SCH772984, and Mekinist (trametinib) resulted in durable inhibition of Erk signaling and proliferation of melanoma cells overexpressing BRAF V600E in culture (PMID: 28714990). 28714990
BRAF V600E melanoma predicted - sensitive RAF265 Case Reports/Case Series Actionable In a Phase I trial, treatment with RAF265 in melanoma patients resulted in an objective response rate of 12.1% (8/66), including a partial response in four patients harboring BRAF V600E, two partial responses and one complete response in patients with wild-type BRAF, and one complete response in a patient with unknown mutational status (PMID: 28719152). 28719152
BRAF V600E lung cancer sensitive Ulixertinib Case Reports/Case Series Actionable In a Phase I trial, treatment with Ulixertinib (BVD-523) resulted in a partial response in two patients with lung cancer each harboring BRAF V600E (PMID: 29247021; NCT01781429). 29247021
BRAF V600E glioblastoma sensitive Ulixertinib Case Reports/Case Series Actionable In a Phase I trial, treatment with Ulixertinib (BVD-523) resulted in a partial response in a patient with glioblastoma harboring BRAF V600E (PMID: 29247021; NCT01781429). 29247021
BRAF V600E glioblastoma sensitive Ulixertinib Case Reports/Case Series Actionable In an expanded access program (ULI-EAP-100), Ulixertinib (BVD-523) treatment resulted in clinical benefit in a patient with glioblastoma harboring BRAF V600E who stayed on treatment for 128 days (J Clin Oncol 40, no. 16_suppl (June 01, 2022) e15101; NCT04566393). detail...
BRAF V600E melanoma sensitive Trametinib FDA approved - On Companion Diagnostic Actionable In a Phase III trial (METRIC) that supported FDA approval, Mekinist (trametinib) treatment, as compared to Deticine (dacarbazine) or Taxol (paclitaxel) treatment, resulted in improved progression-free survival of 4.8 months versus 1.5 months and an overall six month survival rate of 81% versus 67% in patients with BRAF V600E/K-positive metastatic melanoma (PMID: 22663011; NCT01245062). 22663011 detail... detail...
BRAF V600E rectum cancer resistant Cetuximab Guideline Actionable Erbitux (cetuximab), as a monotherapy, is not indicated for use in rectum cancer patients with BRAF V600E (NCCN.org). detail...
BRAF V600E rectum cancer resistant Panitumumab Guideline Actionable Vectibix (panitumumab), as a monotherapy, is not indicated for use in rectum cancer patients with BRAF V600E (NCCN.org). detail...
BRAF V600E colorectal cancer predicted - sensitive Dabrafenib + Panitumumab + Trametinib Phase I Actionable In a Phase I trial, combination therapy consisting of Tafinlar (dabrafenib), Vectibix (panitumumab), and Mekinist (trametinib) resulted in an overall response rate of 21% (19/91, 1 complete response, 18 partial response), stable disease in 65% (59/91), and a median progression-free survival of 4.2 months in patients with BRAF V600E colorectal cancer (PMID: 29431699; NCT01750918). 29431699
BRAF V600E colorectal cancer no benefit Panitumumab + Trametinib Phase I Actionable In a Phase I trial, combination therapy consisting of Vectibix (panitumumab) and Mekinist (trametinib) resulted in an overall response rate of 0% (0/31), stable disease in 55% (17/31), and a median progression-free survival of 2.6 months in patients with BRAF V600E colorectal cancer (PMID: 29431699; NCT01750918). 29431699
BRAF V600E pleomorphic xanthoastrocytoma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Mekinist (trametinib) and Tafinlar (dabrafenib) combination treatment resulted in clinical benefit that lasted for more than 14 months in a patient with pleomorphic xanthoastrocytoma harboring BRAF V600E (PMID: 29632053). 29632053
BRAF V600E pleomorphic xanthoastrocytoma sensitive Dabrafenib + Trametinib Guideline Actionable Mekinist (trametinib) and Tafinlar (dabrafenib) combination therapy is included in guidelines as an adjuvant treatment for patients with pleomorphic xanthoastrocytoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E glioblastoma sensitive Dabrafenib + Trametinib Guideline Actionable Combination of Mekinist (trametinib) and Tafinlar (dabrafenib) is included in guidelines for patients with recurrent glioblastoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E glioblastoma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, an epithelioid glioblastoma patient harboring BRAF V600E treated with Mekinist (trametinib) and Tafinlar (dabrafenib) combination therapy resulted in stable disease, and the patient continued to demonstrate stable disease at least 16 months after initiation of therapy (PMID: 29632053). 29632053
BRAF V600E glioblastoma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, combination therapy of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in improved clinical symptoms in a patient with epithelioid glioblastoma harboring BRAF V600E, and treatment of the patient's cells resulted in decreased cell viability, reduced phosphorylation of Mek and Erk, increased apoptotic activity compared to either agent alone, and cell cycle arrest in culture, and led to tumor growth suppression in the patient-derived xenograft (PDX) model (PMID: 31345255). 31345255
BRAF V600E glioblastoma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Mekinist (trametinib) and Tafinlar (dabrafenib) combination treatment resulted in a complete resolution of symptoms and radiographic partial response after the first treatment in a patient with glioblastoma harboring BRAF V600E whose disease progressed on PLX8394 treatment, and a complete response after 11 months of treatment, CDKN2A/B loss and CHEK2 T367fs were also identified in the tumor (PMID: 32923904). 32923904
BRAF V600E glioblastoma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Mekinist (trametinib) and Tafinlar (dabrafenib) combination treatment resulted in significant clinical improvement in a patient with epithelioid glioblastoma harboring BRAF V600E, however, her disease progressed after 3 months of therapy (PMID: 31217909). 31217909
BRAF V600E glioblastoma sensitive Dabrafenib + Trametinib Phase II Actionable In a Phase II trial (ROAR), Tafinlar (dabrafenib) and Mekinist (trametinib) combination treatment resulted in an objective response in 32% (10/31, 2 complete responses, 8 partial responses) and stable disease in 19% (6/31) of patients with glioblastoma harboring BRAF V600E (PMID: 34838156; NCT02034110). 34838156
BRAF V600E melanoma sensitive RAF709 Preclinical - Cell culture Actionable In a preclinical study, RAF709 inhibited Erk signaling and proliferation of melanoma cells harboring BRAF V600E in culture (PMID: 29343524). 29343524
BRAF V600E colorectal cancer sensitive Binimetinib + Cetuximab + Encorafenib Phase II Actionable In a Phase II (ANCHOR CRC) trial, Braftovi (encorafenib), Mektovi (binimetinib), and Erbitux (cetuximab) combination treatment (n=95) demonstrated an acceptable safety profile and resulted in a confirmed objective response rate of 47.8% (44/92), a disease control rate of 88% (81/92), a median progression-free survival of 5.8 months, and a median overall survival of 18.3 months in patients with metastatic colorectal cancer harboring BRAF V600E (PMID: 36763936; NCT03693170). 36763936
BRAF V600E colorectal cancer sensitive Binimetinib + Cetuximab + Encorafenib Phase III Actionable In a Phase III (BEACON CRC) trial, Braftovi (encorafenib), Mektovi (binimetinib), and Erbitux (cetuximab) combination treatment (n=111) resulted in improved median overall survival (9.0 vs 5.4 months, HR=0.52, p<0.001), confirmed response rate (26% vs 2%, p<0.001), and median progression-free survival (4.3 vs 1.5 months, HR=0.38, p<0.001) compared to control (n=107) in patients with metastatic colorectal cancer harboring BRAF V600E (PMID: 31566309; NCT02928224). 31566309
BRAF V600E colorectal cancer sensitive Binimetinib + Cetuximab + Encorafenib Clinical Study Actionable In a retrospective analysis, real-world treatment with Erbitux (cetuximab) and Braftovi (encorafenib) with or without Mektovi (binimetinib) led to an objective response rate (ORR) of 32.2% (57/201, 2 complete responses (CR)), disease control rate of 71.2% (126/201), median progression-free survival of 4.5 months, and median overall survival of 9.2 months in metastatic colorectal cancer patients with BRAF V600E, with an ORR of 33.3% (7/21, 1 CR) in patients treated with triplet therapy (PMID: 39255538). 39255538
BRAF V600E colorectal cancer sensitive Binimetinib + Cetuximab + Encorafenib Clinical Study Actionable In a retrospective study, combination treatment with Mektovi (binimetinib), Erbitux (cetuximab), and Braftovi (encorafenib) resulted in an objective response rate of 31%, a disease control rate of 78%, a median progression-free survival of 4.2 mo, and a median overall survival of 7.1 mo in patients with metastatic colorectal cancer harboring BRAF V600E (PMID: 35696748). 35696748
BRAF V600E ganglioglioma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) treatment resulted in a partial response in two pediatric patients with ganglioglioma (1 suprasellar ganglioglioma, 1 cervicomedullary ganglioglioma) harboring BRAF V600E (PMID: 39399174). 39399174
BRAF V600E ganglioglioma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) treatment resulted in partial response 8 weeks after therapy initiation in a pediatric patient with anaplastic ganglioglioma harboring BRAF V600E, but disease progression occurred at 40 weeks due to acquired resistance (PMID: 29880583). 29880583
BRAF V600E ganglioglioma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical study, Tafinlar (dabrafenib) treatment resulted in symptomatic improvement and clinical efficacy in three pediatric patients with brainstem ganglioglioma harboring BRAF V600E, with decreased tumor volume and treatment continuing for at least 5.5 years in one patient, a significant partial response in another patient, and a partial response in a third patient with tumor regression and ongoing response for at least 2 years (PMID: 31502039). 31502039
BRAF V600E melanoma sensitive Ulixertinib Preclinical - Cell line xenograft Actionable In a preclinical study, Ulixertinib (BVD-523) inhibited Erk signaling in melanoma cells harboring BRAF V600E, resulted in cell cycle arrest in culture and tumor growth inhibition in cell line xenograft models (PMID: 28939558). 28939558
BRAF V600E colorectal cancer sensitive Ulixertinib Preclinical - Cell line xenograft Actionable In a preclinical study, Ulixertinib (BVD-523) inhibited Erk signaling in colorectal cancer cells harboring BRAF V600E, resulted in cell cycle arrest in culture and tumor growth inhibition in cell line xenograft models (PMID: 28939558). 28939558
BRAF V600E melanoma decreased response Nivolumab Clinical Study - Cohort Actionable In a retrospective analysis, patients with melanoma harboring BRAF V600E (n=84) had decreased response rates (29% vs. 53%, p=0.059), progression-free survival (2.7 vs. 19 months, p=0.049), and overall survival (11.7 vs. 20.4 months, p=0.081) relative to patients with BRAF V600K (n=19) when treated with Keytruda (pembrolizumab) (n=62 and 17 for BRAF V600E and V600K, respectively) or Opdivo (nivolumab) (n=22 and 2 for BRAF V600E and V600K, respectively) (PMID: 30630828). 30630828
BRAF V600E melanoma decreased response Pembrolizumab Clinical Study Actionable In a retrospective analysis, patients with melanoma harboring BRAF V600E (n=84) had decreased response rates (29% vs. 53%, p=0.059), progression-free survival (2.7 vs. 19 months, p=0.049), and overall survival (11.7 vs. 20.4 months, p=0.081) relative to patients with BRAF V600K (n=19) when treated with Keytruda (pembrolizumab) (n=62 and 17 for BRAF V600E and V600K, respectively) or Opdivo (nivolumab) (n=22 and 2 for BRAF V600E and V600K, respectively) (PMID: 30630828). 30630828
BRAF V600E melanoma decreased response Pembrolizumab Clinical Study Actionable In a retrospective analysis, real-world treatment with either Keytruda (pembrolizumab) or Toripalimab (JS001) resulted in a median disease-free survival of 17 months in melanoma patients harboring BRAF V600E compared to 32 months in patients with wild-type BRAF, NRAS, and KIT (p=0.022) (PMID: 37403699). 37403699
BRAF V600E high grade glioma sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), responses were seen in glioma patients harboring BRAF V600E (n=24) when treated with Zelboraf (vemurafenib), including 1 patient with a complete response and 5 patients with a partial response (PMID: 32029534; NCT01524978). 32029534
BRAF V600E high grade glioma sensitive Vemurafenib Phase II Actionable In a Phase II trial (VE-BASKET), Zelboraf (vemurafenib) treatment resulted in an objective response rate of 25% (6/24, 1 complete response, 5 partial responses) in patients with gliomas harboring BRAF V600E, with a median progression free survival of 5.5-months, a median overall survival of 28.2 months (PMID: 30351999; NCT01524978). 30351999
BRAF V600E high grade glioma sensitive Vemurafenib Guideline Actionable Zelboraf (vemurafenib) is included in guidelines as adjuvant therapy for pediatric patients with diffuse high-grade gliomas harboring BRAF V600E, or as a preferred regimen for patients with recurrent or progressive disease (NCCN.org). detail...
BRAF V600E pleomorphic xanthoastrocytoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in a complete intracranial response 2 months after treatment in a patient with anaplastic pleomorphic xanthoastrocytoma harboring BRAF V600E, although the disease progressed 1 month later (PMID: 31386052). 31386052
BRAF V600E pleomorphic xanthoastrocytoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), Zelboraf (vemurafenib) treatment resulted in an objective response rate of 42.9% (3/7, 1 complete response, 2 partial responses) and a clinical benefit rate of 57% in patients with pleomorphic xanthoastrocytoma harboring BRAF V600E, with a median progression-free survival of 5.7 months, and median overall survival not reached (PMID: 30351999; NCT01524978). 30351999
BRAF V600E melanoma resistant RMC-4550 Preclinical - Cell culture Actionable In a preclinical study, RMC-4550 did not inhibit Erk phosphorylation or proliferation of melanoma cells harboring BRAF V600E in culture (PMID: 30104724). 30104724
BRAF V600E Advanced Solid Tumor sensitive Vemurafenib Phase II Actionable In a Phase II trial (MyPathway), Zelboraf (vemurafenib) treatment resulted in an objective response of 46% (12/26, 2 complete response, 10 partial response) in patients with advanced solid tumors harboring BRAF V600E, but only 4% (1/23, 1 partial response) in patients harboring non-V600 BRAF mutations (PMID: 29320312; NCT02091141). 29320312
BRAF V600E Advanced Solid Tumor sensitive Vemurafenib Phase II Actionable In a Phase II trial (VE-BASKET), Zelboraf (vemurafenib) treatment resulted in an objective response rate of 33% (56/172) in patients with advanced solid tumors harboring BRAF V600E, including 5 patients with a complete response and 51 patients with a partial response, and led a duration of response of 13.1 months, a progression-free survival of 5.8 months, and an overall survival of 17.6 months (PMID: 32029534; NCT01524978). 32029534
BRAF V600E ovarian cancer predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (MyPathway), Zelboraf (vemurafenib) treatment resulted in an objective response of 50% (2/4, 2 partial response) in patients with ovarian cancer harboring BRAF V600E, and stable disease lasting more than 120 days in 1 patient (PMID: 29320312; NCT02091141). 29320312
BRAF V600E ovarian cancer predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), responses were seen in ovarian cancer patients harboring BRAF V600E (n=4) when treated with Zelboraf (vemurafenib), including 2 patients with a partial response (PMID: 32029534; NCT01524978). 32029534
BRAF V600E larynx cancer predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (MyPathway), Zelboraf (vemurafenib) treatment resulted in complete response in a patient with larynx cancer harboring BRAF V600E (PMID: 29320312; NCT02091141). 29320312
BRAF V600E colon neuroendocrine neoplasm predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) treatment of a patient with recurrent neuroendocrine carcinoma of the colon harboring a BRAF V600E mutation resulted in stable disease for 6 months before disease progression (PMID: 30181415). 30181415
BRAF V600E colon neuroendocrine neoplasm no benefit Binimetinib Case Reports/Case Series Actionable In Phase II trial, Mektovi (binimetinib) therapy in a patient with recurrent neuroendocrine carcinoma of the colon harboring a BRAF V600E mutation who had previously progressed on Tafinlar (dabrafenib) resulted in disease progression after two cycles (PMID: 30181415; NCT01885195). 30181415
BRAF V600E salivary gland carcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case report, combined Tafinlar (dabrafenib) and Mekinist (trametinib) treatment of a patient with salivary duct carcinoma harboring BRAF V600E resulted in a reduction of metastatic lesions and stable disease lasting 13 months followed by disease progression (PMID: 30323086). 30323086
BRAF V600E lymphatic system cancer predicted - sensitive Cobimetinib Case Reports/Case Series Actionable In a Phase II trial, treatment with Cotellic (cobimetinib) in patients with histiocytic neoplasms resulted in a PET overall response rate of 89% (16/18), with complete response in 72% (13/18) and partial response in 17% (3/18), and resulted in stable disease in 6% (1/18) of patients, including 1 partial response and 3 complete responses in 4 patients with Erdheim-Chester disease harboring BRAF V600E (PMID: 30867592; NCT01953926). 30867592
BRAF V600E melanoma sensitive Binimetinib + Encorafenib FDA approved - On Companion Diagnostic Actionable In a Phase III (COLUMBUS) trial that supported FDA approval, Braftovi (encorafenib) in combination with Mektovi (binimetinib) demonstrated improved tolerability profile and efficacy, resulted in a progression-free survival of 14.9 months in patients with advanced melanoma harboring BRAF V600E/K mutations, comparing to 7.3 months in the Zelboraf (vemurafenib) group (HR=0.54, p<0.0001) (PMID: 29573941; NCT01909453), and both BRAF V600E and V600K are on the companion diagnostic. 29573941 detail... detail... detail...
BRAF V600E melanoma sensitive Binimetinib + Encorafenib FDA approved - On Companion Diagnostic Actionable In a Phase III (COLUMBUS) trial that supported FDA approval, Braftovi (encorafenib) in combination with Mektovi (binimetinib) resulted in a median overall survival (OS) of 33.6 months, a 1-year OS rate of 77.5%, and a 2-year OS rate of 57.7% in patients with advanced melanoma harboring BRAF V600E/K mutations compared to a median OS of 16.9 months and 1- and 2-year OS rates of 63.1% and 43.2%, respectively, in the Zelboraf (vemurafenib) treated group (PMID: 30219628; NCT01909453). detail... 30219628 detail... detail...
BRAF V600E glioblastoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in a partial response 1 week after treatment in a patient with epithelioid type glioblastoma harboring BRAF V600E, although the patient soon passed due to complications (PMID: 31386052). 31386052
BRAF V600E glioblastoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in tumor regression as confirmed by MRI after 3-weeks of treatment in a patient with epithelioid glioblastoma harboring BRAF V600E (PMID: 31217909). 31217909
BRAF V600E glioblastoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), Zelboraf (vemurafenib) treatment resulted in stable disease as best response in 3 of 6 patients with glioblastoma harboring BRAF V600E, with stable disease lasting 3.6, 3.7, and 12.9 months, respectively (PMID: 30351999; NCT01524978). 30351999
BRAF V600E glioblastoma sensitive Cobimetinib + Vemurafenib Guideline Actionable Combination of Zelboraf (vemurafenib) and Cotellic (cobimetinib) is included in guidelines for patients with recurrent glioblastoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E glioblastoma sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a clinical case study, a patient with epithelioid glioblastoma harboring BRAF V600E continued to response as Cotellic (cobimetinib) was added to Zelboraf (vemurafenib) treatment (PMID: 31217909). 31217909
BRAF V600E papillary thyroid carcinoma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, a papillary thyroid carcinoma patient who progressed on Lenvima (lenvatinib) was found to harbor BRAF V600E and was treated with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) on a clinical trial, resulting in partial response in the thyroid bed, cervical and intrathoracic lymph nodes, and pulmonary lesions, with a decrease in target lesion size of 67%, and the patient remained on treatment for 18 months before stopping due to progression (PMID: 31085763). 31085763
BRAF V600E papillary thyroid carcinoma sensitive Dabrafenib + Trametinib Guideline Actionable The combination of Tafinlar (dabrafenib) and Mekinist (trametinib) is included in guidelines for patients with papillary thyroid carcinoma harboring BRAF V600E who have progressed on therapy and have no further treatment options (NCCN.org). detail...
BRAF V600E childhood pilocytic astrocytoma predicted - sensitive Selumetinib Phase I Actionable In a Phase I trial, Koselugo (selumetinib) treatment resulted in a sustained partial response (PR) in 36% (9/25) and stable disease in 36% (9/25) of pediatric patients with pilocytic astrocytoma harboring KIAA1549-BRAF (n=18) or BRAF V600E (n=7), with a 2-year progression-free survival of 70%, and 29% (2/7) of the patients harboring BRAF V600E achieved PR (PMID: 31151904; NCT01089101). 31151904
BRAF V600E pancreatic endocrine carcinoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, a patient with pancreatic endocrine carcinoma found to harbor BRAF V600E demonstrated stable disease and some tumor shrinkage when treated with Zelboraf (vemurafenib) (PMID: 31158244). 31158244
BRAF V600E skin melanoma sensitive Dabrafenib Guideline Actionable Tafinlar (dabrafenib) therapy is included in guidelines for cutaneous melanoma patients with unresectable or metastatic disease harboring BRAF V600 activating mutations, such as BRAF V600E, in cases where BRAF/MEK inhibitor combination therapy is contraindicated (NCCN.org). detail...
BRAF V600E skin melanoma sensitive Vemurafenib Guideline Actionable Zelboraf (vemurafenib) therapy is included in guidelines for cutaneous melanoma patients with unresectable or metastatic disease harboring BRAF V600 activating mutations, such as BRAF V600E, in cases where BRAF/MEK inhibitor combination therapy is contraindicated (NCCN.org). detail...
BRAF V600E skin melanoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines for cutaneous melanoma patients harboring a BRAF V600 mutation, such as BRAF V600E, as neoadjuvant or adjuvant therapy for stage III disease and as systemic therapy for patients with unresectable or metastatic disease (NCCN.org). detail...
BRAF V600E skin melanoma sensitive Cobimetinib + Vemurafenib Guideline Actionable Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy is included in cutaneous melanoma guidelines for patients with metastatic or unresectable disease harboring a BRAF V600 activating mutation (NCCN.org). detail...
BRAF V600E skin melanoma sensitive Binimetinib + Encorafenib Guideline Actionable Braftovi (encorafenib) in combination with Mektovi (binimetinib) is included in guidelines as first-line and second-line therapy for patients with metastatic or unresectable cutaneous melanoma harboring BRAF V600E or V600K mutations (PMID: 30959471; NCCN.org). detail... 30959471
BRAF V600E salivary gland carcinoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II (MyPathway) trial, Zelboraf (vemurafenib) treatment resulted in a partial response in a patient with advanced salivary gland carcinoma harboring BRAF V600E, with a progression-free survival of 18.5 months (PMID: 32067683; NCT02091141). 32067683
BRAF V600E ganglioglioma sensitive Dabrafenib + Trametinib Guideline Actionable Combination of Mekinist (trametinib) and Tafinlar (dabrafenib) is included in guidelines as an adjuvant treatment for patients with ganglioglioma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E pilocytic astrocytoma sensitive Dabrafenib + Trametinib Guideline Actionable Combination of Mekinist (trametinib) and Tafinlar (dabrafenib) is included in guidelines as an adjuvant treatment for patients with pilocytic astrocytoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E pilocytic astrocytoma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, the addition of Mekinist (trametinib) to treatment with Tafinlar (dabrafenib) resulted in a partial response in 3 pediatric patients with suprasellar pilocytic astrocytoma harboring BRAF V600E who all previously progressed on Tafinlar (dabrafenib) treatment alone (PMID: 39399174). 39399174
BRAF V600E ganglioglioma sensitive Cobimetinib + Vemurafenib Guideline Actionable Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy is included in guidelines as an adjuvant treatment for patients with ganglioglioma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E pleomorphic xanthoastrocytoma sensitive Cobimetinib + Vemurafenib Guideline Actionable Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy is included in guidelines as an adjuvant treatment for patients with pleomorphic xanthoastrocytoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E pilocytic astrocytoma sensitive Cobimetinib + Vemurafenib Guideline Actionable Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy is included in guidelines as an adjuvant treatment for patients with pilocytic astrocytoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E melanoma predicted - sensitive Dabrafenib + Pembrolizumab + Trametinib Phase II Actionable In a Phase II trial (IMPemBra), Keytruda (pembrolizumab) in combination with short-term or intermittent Tafinlar (dabrafenib) plus Mekinist (trametinib) resulted in improved median progression-free survival compared to Keytruda (pembrolizumab) monotherapy (27.0 vs 10.6 mo, p=0.13) in patients with treatment-naive advanced melanoma harboring BRAF V600E (n=26) or V600K (n=6) mutations (J Clin Oncol 38: 2020 (suppl; abstr 10021); NCT02625337). detail...
BRAF V600E anaplastic thyroid carcinoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, first-line adjuvant therapy with Zelboraf (vemurafenib) resulted in a partial remission of the tumor and lymphatic and pulmonary metastases after 36 days of treatment in a patient with analplastic thyroid carcinoma harboring BRAF V600E, with a near-complete regression observed within 5 months of treatment initiation (PMID: 36855200). 36855200
BRAF V600E anaplastic thyroid carcinoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), Zelboraf (vemurafenib) treatment resulted in a response in patients with anaplastic thyroid carcinoma harboring BRAF V600E (n=12), including 1 patient with a complete response and 2 patients with a partial response (PMID: 32029534; NCT01524978). 32029534
BRAF V600E cholangiocarcinoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), responses were seen in patients with cholangiocarcinoma harboring BRAF V600E (n=9) when treated with Zelboraf (vemurafenib), including 2 patients with a partial response (PMID: 32029534; NCT01524978). 32029534
BRAF V600E sarcoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), responses were seen in sarcoma patients harboring BRAF V600E (n=6) when treated with Zelboraf (vemurafenib), including 1 patient with a complete response and 1 patient with a partial response (PMID: 32029534; NCT01524978). 32029534
BRAF V600E neuroendocrine carcinoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), responses were seen in patients with neuroendocrine carcinoma harboring BRAF V600E (n=3) when treated with Zelboraf (vemurafenib), including 1 patient with a partial response (PMID: 32029534; NCT01524978). 32029534
BRAF V600E salivary gland cancer predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), a patient with salivary ductal carcinoma harboring BRAF V600E demonstrated a partial response when treated with Zelboraf (vemurafenib) (PMID: 32029534; NCT01524978). 32029534
BRAF V600E histiocytic and dendritic cell cancer predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), responses were seen in patients with histiocytic neoplasms harboring BRAF V600E (n=27) when treated with Zelboraf (vemurafenib), including 15 patients with a partial response and 2 patients with a complete response (PMID: 32029534; NCT01524978). 32029534
BRAF V600E thyroid cancer sensitive PLX4720 + Ponatinib Preclinical - Cell line xenograft Actionable In a preclinical study, Iclusig (ponatinib) and PLX4720 treatment demonstrated synergy, and inhibited Erk, Mek, and c-jun phosphorylation, reduced cell proliferation, colony formation, and migration, and induced apoptosis in thyroid cancer cells harboring BRAF V600E and in PLX4720-resistant cells harboring BRAF V600E in culture, and inhibited tumor growth, reduced lung and liver metastases, and increased survival in cell line xenograft models (PMID: 31937621). 31937621
BRAF V600E thyroid cancer sensitive Ponatinib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, Iclusig (ponatinib) and Zelboraf (vemurafenib) treatment synergistically inhibited proliferation of thyroid cancer cells harboring BRAF V600E in culture (PMID: 31937621). 31937621
BRAF V600E ovarian serous carcinoma predicted - sensitive Lifirafenib Case Reports/Case Series Actionable In a Phase I trial, Lifirafenib (BGB-283) treatment demonstrated safety and resulted in partial response (PR) in 15.1% (8/53) of advanced solid tumor patients harboring a BRAF mutation, including 1 patient with BRAF V600E-mutant low-grade serous ovarian cancer (PMID: 32182156; NCT02610361). 32182156
BRAF V600E biliary tract cancer sensitive Dabrafenib + Trametinib Phase II Actionable In a Phase II trial (ROAR), Tafinlar (dabrafenib) in combination with Mekinist (trametinib) demonstrated a manageable safety profile and resulted in an overall response rate of 51% (22/43, all partial responses) in patients with biliary tract cancer harboring BRAF V600E, with a median duration of response of 9 months, a median progression-free survival of 9 months, and a median overall survival of 14 months (PMID: 32818466; NCT02034110). 32818466
BRAF V600E biliary tract cancer sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) in combination with Mekinist (trametinib) is included in guidelines as second or later-line therapy for patients with biliary tract cancer harboring BRAF V600E (PMID: 36372281; ESMO.org). 36372281 detail...
BRAF V600E biliary tract cancer sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) in combination with Mekinist (trametinib) is included in guidelines as subsequent-line therapy for patients with biliary tract cancer harboring BRAF V600E (NCCN.org). detail...
BRAF V600E colorectal cancer predicted - sensitive Ravoxertinib Case Reports/Case Series Actionable In a Phase I trial, two colorectal cancer patients harboring BRAF V600E achieved partial responses lasting 21 and 73 weeks following treatment with Ravoxertinib (GDC-0994) (PMID: 31848189; NCT01875705). 31848189
BRAF V600E papillary thyroid carcinoma sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in a partial response with approximately 90% tumor shrinkage and a progression-free survival of 10 months in a patient with metastatic papillary thyroid carcinoma harboring BRAF V600E (PMID: 38489728). 38489728
BRAF V600E papillary thyroid carcinoma sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase I trial, Zelboraf (vemurafenib) treatment resulted in a complete or partial response in three papillary thyroid carcinoma patients harboring BRAF V600E, with one patient having a response that lasted for 8 months who remained progression-free for 12 months, and another two patients having a stable disease that lasted for 11 and 13 months, respectively (PMID: 20818844; NCT00215605). 20818844
BRAF V600E papillary thyroid carcinoma sensitive Vemurafenib Phase II Actionable In a Phase II trial, Zelboraf (vemurafenib) treatment resulted in a partial response in 38.5% (10/26) of patients with metastatic or unresectable, radioactive iodine-refractory papillary thyroid carcinoma harboring BRAF V600E that were multikinase inhibitor-naive, with a disease control rate of 73%, and a median progression-free survival of 18.2 months (PMID: 27460442; NCT01286753). 27460442
BRAF V600E papillary thyroid carcinoma sensitive Vemurafenib Guideline Actionable Zelboraf (vemurafenib) is included in guidelines for patients with recurrent, advanced, or metastatic thyroid papillary carcinoma harboring BRAF V600E for whom clinical trials are not available or appropriate (NCCN.org). detail...
BRAF V600E colorectal cancer sensitive Capecitabine + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Zelboraf (vemurafenib) treatment in combination with Xeloda (capecitabine) enhanced tumor growth inhibition and increased survival in a cell line xenograft model of colorectal cancer harboring BRAF V600E (PMID: 22180495). 22180495
BRAF V600E colorectal cancer sensitive Bevacizumab + Capecitabine + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Zelboraf (vemurafenib) treatment when combined with Xeloda (capecitabine) and Avastin (bevacizumab) enhanced tumor growth inhibition and increased survival in a cell line xenograft model of colorectal cancer harboring BRAF V600E (PMID: 22180495). 22180495
BRAF V600E colorectal cancer sensitive Irinotecan + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Zelboraf (vemurafenib) treatment in combination with Camptosar (irinotecan) enhanced tumor growth inhibition and increased survival in a cell line xenograft model of colorectal cancer harboring BRAF V600E (PMID: 22180495). 22180495
BRAF V600E colorectal cancer sensitive Cetuximab + Irinotecan + Vemurafenib Phase II Actionable In a Phase II trial (SWOG1406), addition of Zelboraf (vemurafenib) to Camptosar (irinotecan) plus Erbitux (cetuximab) improved progression-free survival (4.4 vs. 2.0 mo, HR 0.50, p=0.001), response rate (17% vs 4%, p=0.05), and disease control rate (67% vs. 22%, p<0.001) in patients with metastatic colorectal cancer harboring BRAF V600E (PMID: 33356422; NCT02164916). 33356422
BRAF V600E colorectal cancer sensitive Cetuximab + Irinotecan + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Zelboraf (vemurafenib) treatment in combination with Erbitux (cetuximab) and Camptosar (irinotecan) enhanced tumor growth inhibition and increased survival in a cell line xenograft model of colorectal cancer harboring BRAF V600E (PMID: 22180495). 22180495
BRAF V600E melanoma predicted - sensitive U0126 Preclinical - Cell culture Actionable In a preclinical study, U0126 treatment inhibited Erk phosphorylation and reduced growth of melanoma cells harboring BRAF V600E in culture (PMID: 18794803). 18794803
BRAF V600E melanoma sensitive C6-ceramide nanoliposome + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, Nexavar (sorafenib) treatment in combination with C6-ceramide nanoliposome inhibited Mek and Akt phosphorylation, led to enhanced apoptosis and inhibition of proliferation, and synergistically reduced viability of melanoma cells harboring BRAF V600E in culture, and enhanced inhibition of tumor growth in a cell line xenograft model (PMID: 18519791). 18519791
BRAF V600E melanoma sensitive BGB659 Preclinical - Cell culture Actionable In a preclinical study, BGB659 treatment inhibited Erk phosphorylation and reduced proliferation of a melanoma cells harboring either monomeric BRAF V600E or dimeric isoform (p61) of V600E in culture (PMID: 30559419). 30559419
BRAF V600E melanoma sensitive PLX8394 Preclinical - Cell culture Actionable In a preclinical study, PLX8394 treatment inhibited Erk phosphorylation and reduced proliferation of melanoma cells harboring either monomeric BRAF V600E or dimeric isoform (p61) of V600E in culture (PMID: 30559419). 30559419
BRAF V600E colorectal cancer predicted - resistant BI-3406 Preclinical - Cell culture Actionable In a preclinical study, BI-3406 treatment failed to inhibit growth of colorectal cancer cells harboring BRAF V600E in culture (PMID: 32816843). 32816843
BRAF V600E melanoma predicted - resistant BI-3406 Preclinical - Cell line xenograft Actionable In a preclinical study, BI-3406 treatment failed to inhibit growth of KRAS wild-type melanoma cells harboring BRAF V600E in culture and in cell line xenograft models (PMID: 32816843). 32816843
BRAF V600E colon cancer sensitive Cetuximab + Encorafenib Guideline Actionable Braftovi (encorafenib) in combination with Erbitux (cetuximab) is included in guidelines as primary or subsequent therapy for patients with colon cancer harboring BRAF V600E (NCCN.org). detail...
BRAF V600E colon cancer sensitive Encorafenib + Panitumumab Guideline Actionable Braftovi (encorafenib) in combination with Vectibix (panitumumab) is included in guidelines as primary or subsequent therapy for patients with colon cancer harboring BRAF V600E (NCCN.org). detail...
BRAF V600E rectum cancer sensitive Encorafenib + Panitumumab Guideline Actionable Braftovi (encorafenib) in combination with Vectibix (panitumumab) is included in guidelines as primary or subsequent therapy for patients with rectal cancer harboring BRAF V600E (NCCN.org). detail...
BRAF V600E rectum cancer sensitive Cetuximab + Encorafenib Guideline Actionable Braftovi (encorafenib) in combination with Erbitux (cetuximab) is included in guidelines as primary or subsequent therapy for patients with rectal cancer harboring BRAF V600E (NCCN.org). detail...
BRAF V600E lung non-small cell carcinoma not predictive Pembrolizumab Clinical Study - Cohort Actionable In a retrospective analysis, non-small cell lung cancer patients harboring BRAF V600E did not demonstrate a significantly different response to treatment with either Keytruda (pembrolizumab), Opdivo (nivolumab), or Tecentriq (atezolizumab), compared to patients with BRAF non-V600E mutations, demonstrating an objective response rate of 25% (3/12) vs 33% (3/9) (p=1.0) and median progression-free survival of 3.7 months vs 4.1 months (p=0.37) (PMID: 29723688). 29723688
BRAF V600E lung non-small cell carcinoma not predictive Nivolumab Clinical Study - Cohort Actionable In a retrospective analysis, non-small cell lung cancer patients harboring BRAF V600E did not demonstrate a significantly different response to treatment with either Keytruda (pembrolizumab), Opdivo (nivolumab), or Tecentriq (atezolizumab), compared to patients with BRAF non-V600E mutations, demonstrating an objective response rate of 25% (3/12) vs 33% (3/9) (p=1.0) and median progression-free survival of 3.7 months vs 4.1 months (p=0.37) (PMID: 29723688). 29723688
BRAF V600E lung non-small cell carcinoma not predictive Atezolizumab Clinical Study - Cohort Actionable In a retrospective analysis, non-small cell lung cancer patients harboring BRAF V600E did not demonstrate a significantly different response to treatment with either Keytruda (pembrolizumab), Opdivo (nivolumab), or Tecentriq (atezolizumab) compared to patients with BRAF non-V600E mutations, demonstrating an objective response rate of 25% (3/12) vs 33% (3/9) (p=1.0) and median progression-free survival of 3.7 months vs 4.1 months (p=0.37) (PMID: 29723688). 29723688
BRAF V600E melanoma sensitive AZD0364 Preclinical - Cell line xenograft Actionable In a preclinical study, AZD0364 (ATG-017) inhibited cell growth, decreased phosphorylation of Erk target genes, and increased expression of apoptosis markers in melanoma cells harboring BRAF V600E in culture, and resulted in tumor regression in cell line xenograft models (PMID: 33273059). 33273059
BRAF V600E lung non-small cell carcinoma unknown unspecified immune checkpoint inhibitor Clinical Study - Cohort Actionable In a retrospective clinical study, patients with non-small cell lung cancer harboring rare targetable drivers (RTD) (BRAF, ERBB2/3, RET, MET, ROS1, NTRK) who received immune checkpoint inhibitors (ICI) achieved longer median overall survival (mOS) (32 vs 13 mo, p=0.01) compared to those who did not receive ICI, mOS was not reached in patients harboring BRAF V600E (n=5) mutations, although RTD type was not associated with OS in a univariate analysis (PMID: 30268448). 30268448
BRAF V600E Advanced Solid Tumor predicted - sensitive Trametinib Phase II Actionable In a Phase II trial (NCI-MATCH), Mekinist (trametinib) treatment resulted in an objective response rate of 37.9% (11/29) in patients with BRAF V600E positive advanced solid tumors other than melanoma, thyroid cancer, or colorectal cancer, with a median duration of response of 25.1 months, and a disease control rate of 75.9% (22/29) (PMID: 32758030; NCT02465060). 32758030
BRAF V600E colorectal cancer sensitive Chloroquine + Trametinib Preclinical - Pdx Actionable In a preclinical study, combination treatment with Mekinist (trametinib) and Chloroquine resulted in tumor regression in a colorectal cancer patient-derived xenograft (PDX) model harboring BRAF V600E (PMID: 30833748). 30833748
BRAF V600E ovarian serous carcinoma predicted - sensitive Trametinib Case Reports/Case Series Actionable In a Phase II trial (NCI-MATCH), Mekinist (trametinib) treatment resulted in an objective response rate of 37.9% (11/29) in patients with advanced solid tumors harboring BRAF V600E, of 5 patients with low grade serous ovarian carcinoma, 4 achieved a partial response (PR), 1 had stable disease, 3 of the PRs lasted over 12 months (PMID: 32758030; NCT02465060). 32758030
BRAF V600E intrahepatic cholangiocarcinoma predicted - sensitive Trametinib Case Reports/Case Series Actionable In a Phase II trial (NCI-MATCH), Mekinist (trametinib) treatment resulted in an objective response rate of 37.9% (11/29) in patients with advanced solid tumors harboring BRAF V600E, of 4 patients with intrahepatic cholangiocarcinoma, 3 achieved a partial response, with individual progression-free survival of 12.8, 9.1, and 29.4 months (PMID: 32758030; NCT02465060). 32758030
BRAF V600E lung adenocarcinoma predicted - sensitive Trametinib Case Reports/Case Series Actionable In a Phase II trial (NCI-MATCH), Mekinist (trametinib) treatment resulted in an objective response rate of 37.9% (11/29) in patients with advanced solid tumors harboring BRAF V600E, of 4 patients with lung adenocarcinoma, 1 achieved a partial response with an ongoing progression-free survival (PFS) at 32.5 mo, 3 patients had stable disease for 15.6, 6.6, and 3.6 mo, and an additional unevaluable patient achieved an 81% reduction of measured lesions and a PFS of 12.7 mo (PMID: 32758030; NCT02465060). 32758030
BRAF V600E pleomorphic xanthoastrocytoma predicted - sensitive Trametinib Case Reports/Case Series Actionable In a Phase II trial (NCI-MATCH), Mekinist (trametinib) treatment resulted in an objective response rate of 37.9% (11/29) in patients with advanced solid tumors harboring BRAF V600E, 1 patient with pleomorphic xanthoastrocytoma achieved a partial response lasting 7.2 months (PMID: 32758030; NCT02465060). 32758030
BRAF V600E histiocytic sarcoma predicted - sensitive Trametinib Case Reports/Case Series Actionable In a Phase II trial (NCI-MATCH), Mekinist (trametinib) treatment resulted in an objective response rate of 37.9% (11/29) in patients with advanced solid tumors harboring BRAF V600E, 1 patient with histiocytic sarcoma of the brain achieved a partial response with an ongoing progression-free survival at 20.9 months (PMID: 32758030; NCT02465060). 32758030
BRAF V600E peritoneal serous papillary adenocarcinoma predicted - sensitive Trametinib Case Reports/Case Series Actionable In a Phase II trial (NCI-MATCH), Mekinist (trametinib) treatment resulted in an objective response rate of 37.9% (11/29) in patients with advanced solid tumors harboring BRAF V600E, 1 patient with mucinous-papillary serous adenocarcinoma of the peritoneum achieved a partial response (PMID: 32758030; NCT02465060). 32758030
BRAF V600E glioblastoma predicted - sensitive PLX8394 Case Reports/Case Series Actionable In a clinical case study, PLX8394 treatment resulted in a radiographic partial response and complete resolution of symptoms for 7 months in a patient with glioblastoma harboring BRAF V600E, CDKN2A/B loss and CHEK2 T367fs were also identified in the tumor (PMID: 32923904; NCT02428712). 32923904
BRAF V600E brain glioblastoma multiforme predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy resulted in tumor shrinkage after 2 months in a patient with epithelioid glioblastoma multiforme harboring BRAF V600E, who had progressed after resection, radiotherapy, and chemotherapy, and following an interruption in therapy due to toxicity demonstrated a partial response and remained progression-free for 19 months, prior to progressing 29 months after initiation of therapy (PMID: 33461766). 33461766
BRAF V600E uveal melanoma no benefit YM-254890 Preclinical - Cell culture Actionable In a preclinical study, transformed mouse melanocytes expressing BRAF V600E were insensitive to treatment with YM-254890 (PMID: 33229459). 33229459
BRAF V600E skin melanoma no benefit Trametinib + YM-254890 Preclinical - Cell culture Actionable In a preclinical study, combination treatment with YM-254890 and Mekinist (trametinib) did not result in synergistic inhibition of cell viability of a cutaneous melanoma cell line harboring BRAF V600E in culture (PMID: 33229459). 33229459
BRAF V600E skin melanoma no benefit YM-254890 Preclinical - Cell culture Actionable In a preclinical study, YM-254890 treatment did not inhibit cell viability of a cutaneous melanoma cell line harboring BRAF V600E in culture (PMID: 33229459). 33229459
BRAF V600E colorectal cancer decreased response unspecified immune checkpoint inhibitor Clinical Study - Cohort Actionable In a retrospective analysis, treatment with Keytruda (pembrolizumab), Opdivo (nivolumab), or combination treatment with Opdivo (nivolumab) and Yervoy (ipilimumab) in patients with mismatch repair-deficient colorectal cancer harboring BRAF V600E vs. patients with wild-type BRAF resulted in a lower objective response rate (44.4% vs. 74.2%, p = 0.12) and shorter progression-free survival (PFS) rates (1-year PFS 40% vs. 73.3%, 2-year PFS 26.7% vs. 73.3%) (PMID: 33631043). 33631043
BRAF V600E melanoma sensitive Lifirafenib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination treatment of Mekinist (trametinib) and Lifirafenib (BGB-283) led to greater inhibition of growth in melanoma cell lines harboring BRAF V600E in culture compared to either treatment alone (PMID: 33318037). 33318037
BRAF V600E colorectal cancer sensitive Lifirafenib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination treatment of Mekinist (trametinib) and Lifirafenib (BGB-283) led to greater inhibition of growth in a colorectal cancer cell line harboring BRAF V600E in culture compared to either treatment alone (PMID: 33318037). 33318037
BRAF V600E Erdheim-Chester disease sensitive Vemurafenib Guideline Actionable Zelboraf (vemurafenib) is included in guidelines as preferred first-line or subsequent-line therapy for patients with Erdheim-Chester disease harboring BRAF V600E (NCCN.org). detail...
BRAF V600E Erdheim-Chester disease sensitive Dabrafenib Guideline Actionable Tafinlar (dabrafenib) is included in guidelines as first-line or subsequent-line therapy for patients with Erdheim-Chester disease harboring BRAF V600E (NCCN.org). detail...
BRAF V600E colorectal cancer sensitive Dabrafenib + Regorafenib + Trametinib Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment with Stivarga (regorafenib), Tafinlar (dabrafenib), and Mekinist (trametinib) in colorectal cancer cell lines harboring BRAF V600E resulted in suppression of colony formation in culture, and inhibition of tumor growth in a cell line xenograft model (PMID: 33568355). 33568355
BRAF V600E colorectal cancer sensitive Dabrafenib + RAF709 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, combination treatment with RAF709, Tafinlar (dabrafenib), and Mekinist (trametinib) in colorectal cancer cell lines harboring BRAF V600E resulted in suppression of colony formation in culture (PMID: 33568355). 33568355
BRAF V600E colorectal cancer sensitive Dabrafenib + LXH 254 + Trametinib Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment with LXH 254, Tafinlar (dabrafenib), and Mekinist (trametinib) in colorectal cancer cell lines harboring BRAF V600E resulted in suppression of colony formation in culture, and inhibition of tumor growth in a cell line xenograft model (PMID: 33568355). 33568355
BRAF V600E melanoma sensitive Dabrafenib + Regorafenib + Trametinib Preclinical - Pdx & cell culture Actionable In a preclinical study, combination treatment with Stivarga (regorafenib), Tafinlar (dabrafenib), and Mekinist (trametinib) in a melanoma cell line harboring BRAF V600E resulted in suppression of colony formation in culture, and suppressed tumor growth in patient-derived xenograft (PDX) models (PMID: 33568355). 33568355
BRAF V600E melanoma sensitive Dabrafenib + LXH 254 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, combination treatment with LXH 254, Tafinlar (dabrafenib), and Mekinist (trametinib) in a melanoma cell line harboring BRAF V600E resulted in suppression of colony formation in culture (PMID: 33568355). 33568355
BRAF V600E melanoma sensitive Dabrafenib + RAF709 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, combination treatment with RAF709, Tafinlar (dabrafenib), and Mekinist (trametinib) in melanoma cell line harboring BRAF V600E resulted in suppression of colony formation in culture (PMID: 33568355). 33568355
BRAF V600E colon adenocarcinoma predicted - sensitive Dabrafenib + Regorafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, combination treatment with Stivarga (regorafenib), Tafinlar (dabrafenib), and Mekinist (trametinib) in a patient with colon adenocarcinoma harboring BRAF V600E led to stable disease, and treatment continued for eight months, at which time some disease progression was observed (PMID: 33568355). 33568355
BRAF V600E thyroid cancer sensitive Dabrafenib + SCH772984 Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment with Tafinlar (dabrafenib) and SCH772984 synergistically inhibited cell growth of thyroid cancer cell lines harboring BRAF V600E in culture, and inhibited tumor growth in a cell line xenograft model (PMID: 33595872). 33595872
BRAF V600E melanoma predicted - sensitive LXH 254 Preclinical - Cell line xenograft Actionable In a preclinical study, a melanoma cell line harboring BRAF V600E was sensitive to treatment with LXH254, demonstrating inhibition of cell proliferation in culture, and inhibition of tumor growth and tumor regression in a cell line xenograft model of melanoma (PMID: 33355204). 33355204
BRAF V600E cholangiocarcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, combination treatment with Tafinlar (dabrafenib) and Mekinist (trametinib) in a patient with metastatic cholangiocarcinoma harboring BRAF V600E who progressed on chemotherapy led to a sustained metabolic response for six months (PMID: 33537843). 33537843
BRAF V600E intrahepatic cholangiocarcinoma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, combination treatment with Tafinlar (dabrafenib) and Mekinist (trametinib) in a patient with intrahepatic cholangiocarcinoma harboring BRAF V600E who progressed on chemotherapy led to stable disease at 6 weeks, improvement of lung and liver lesions at 12 weeks after treatment, and response was maintained until disease progression in the liver was observed at 10 months (PMID: 33537843). 33537843
BRAF V600E intrahepatic cholangiocarcinoma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a Phase II trial (NCI-MATCH), treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in an objective response rate of 38% in patients with advanced solid tumors harboring BRAF V600E, with a significantly greater tumor shrinkage observed in patients (n=4) with intrahepatic cholangiocarcinoma compared to the trial average ( -60% vs -35%, P=0.016) (PMID: 38109210). 38109210
BRAF V600E intrahepatic cholangiocarcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) in combination with Mekinist (trametinib) is included in guidelines as subsequent-line therapy for patients with biliary cancer harboring BRAF V600E, including intrahepatic cholangiocarcinoma (NCCN.org). detail...
BRAF V600E pancreatic acinar cell adenocarcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, combination treatment with Tafinlar (dabrafenib) and Mekinist (trametinib) in a patient with pancreatic acinar cell carcinoma harboring BRAF V600E who had previously progressed on chemotherapy led to a response at 8 weeks, and ongoing clinical and radiological response was still observed at 32 months of treatment (PMID: 33537843). 33537843
BRAF V600E pancreatic acinar cell adenocarcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in near complete remission allowing for debulking surgery and disease control for 12 months in a patient with metastatic pancreatic acinar cell carcinoma harboring BRAF V600E, along with germline PALB2 R414* (PMID: 32843432). 32843432
BRAF V600E melanoma predicted - sensitive JSI-1187 Preclinical - Cell line xenograft Actionable In a preclinical study, JSI-1187 treatment in a melanoma cell line harboring BRAF V600E led to inhibition of cell proliferation in culture, and tumor growth inhibition in a cell line xenograft model (Cancer Res 2020;80(16 Suppl):Abstract nr 4188). detail...
BRAF V600E colorectal cancer predicted - sensitive JSI-1187 Preclinical - Cell line xenograft Actionable In a preclinical study, JSI-1187 treatment in a colorectal cancer cell line harboring BRAF V600E led to inhibition of cell proliferation in culture, and tumor regression and dose-dependent tumor growth inhibition in a cell line xenograft model (Cancer Res 2020;80(16 Suppl):Abstract nr 4188). detail...
BRAF V600E colorectal cancer predicted - sensitive Dabrafenib + JSI-1187 Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment with Tafinlar (dabrafenib) and JSI-1187 led to synergistic inhibition of tumor growth in a colorectal cancer cell line xenograft model harboring BRAF V600E (Cancer Res 2020;80(16 Suppl):Abstract nr 4188). detail...
BRAF V600E melanoma no benefit RM-018 Preclinical - Cell culture Actionable In a preclinical study, RM-018 did not inhibit growth of melanoma cells harboring BRAF V600E in culture (PMID: 33824136). 33824136
BRAF V600E melanoma no benefit Cobimetinib + Pembrolizumab + Vemurafenib Phase I Actionable In a Phase I trial, combination of Cotellic (cobimetinib), Zelboraf (vemurafenib), and Keytruda (pembrolizumab) resulted in unreached median progression-free survival and overall survival in patients with advanced melanoma harboring BRAF V600E or V600K mutations, however, the trial was closed due to high incidence of dose-limiting toxicity and decreased health utility at 1 year (J Clin Oncol 39, no. 15_suppl, abstract e21506; NCT02818023). detail...
BRAF V600E Advanced Solid Tumor no benefit Selumetinib Case Reports/Case Series Actionable In a Phase II trial (Pediatric MATCH), Koselugo (selumetinib) treatment was tolerated but did not result in an objective response in pediatric patients with advanced solid tumors including high-grade glioma (n=8) and rhabdomyosarcoma (n=7) harboring MAPK pathway alterations including BRAF V600E (n=2), activating KRAS (n=8)/HRAS (n=1)/NRAS (n=3) or inactivating NF1 (n=7) mutations, with a 6-month progression-free survival of 15% (3/20) and 3 stable disease as best response (PMID: 35363510; NCT03213691). 35363510
BRAF V600E low grade glioma sensitive Dabrafenib + Trametinib Guideline Actionable Combination of Mekinist (trametinib) and Tafinlar (dabrafenib) is included in guidelines for patients with recurrent or progressive low grade glioma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E low grade glioma sensitive Dabrafenib + Trametinib Phase Ib/II Actionable In a Phase I/II trial (Study X2101), Tafinlar (dabrafenib) and Mekinist (trametinib) combination treatment demonstrated manageable toxicity and resulted in an objective response rate of 25% (9/36, 1 complete response, 8 partial responses) and stable disease in 67% (24/36) of pediatric patients with pretreated low-grade glioma harboring BRAF V600E (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 10506; NCT02124772). detail...
BRAF V600E low grade glioma sensitive Dabrafenib + Trametinib Phase II Actionable In a Phase II trial (ROAR), Tafinlar (dabrafenib) and Mekinist (trametinib) combination treatment resulted in an objective response rate of 69% (9/13, 1 complete response, 6 partial responses, 2 minor responses) in patients with low-grade glioma harboring BRAF V600E, with median duration of response, median progression-free survival, and overall survival time unreached (PMID: 34838156; NCT02034110). 34838156
BRAF V600E low grade glioma sensitive Dabrafenib + Trametinib FDA approved Actionable In a Phase II trial that supported FDA approval, Mekinist (trametinib) plus Tafinlar (dabrafenib) significantly improved overall response rate (47% vs 11%, risk ratio 4.31, p<0.001), clinical benefit rate (86% vs 46%), median progression-free survival (PFS, 20.1 vs 7.4 months, p<0.001, HR 0.31), and 12-month PFS rate (67% vs 26%) compared to standard chemotherapy in pediatric patients of 1 year and older with low-grade glioma harboring BRAF V600E (PMID: 37733309; NCT02684058). detail... detail... 37733309
BRAF V600E low grade glioma sensitive Cobimetinib + Vemurafenib Guideline Actionable Combination of Zelboraf (vemurafenib) and Cotellic (cobimetinib) is included in guidelines for patients with recurrent or progressive low grade glioma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E pilocytic astrocytoma sensitive Selumetinib Guideline Actionable Koselugo (selumetinib) is included in guidelines for patients with recurrent or progressive pilocytic astrocytoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E anaplastic astrocytoma sensitive Dabrafenib + Trametinib Guideline Actionable Combination of Mekinist (trametinib) and Tafinlar (dabrafenib) is included in guidelines for patients with recurrent anaplastic gliomas harboring BRAF V600E, including anaplastic astrocytoma (NCCN.org). detail...
BRAF V600E anaplastic oligodendroglioma sensitive Dabrafenib + Trametinib Guideline Actionable Combination of Mekinist (trametinib) and Tafinlar (dabrafenib) is included in guidelines for patients with recurrent anaplastic gliomas harboring BRAF V600E, including anaplastic oligodendroglioma (NCCN.org). detail...
BRAF V600E anaplastic oligodendroglioma sensitive Cobimetinib + Vemurafenib Guideline Actionable Combination of Zelboraf (vemurafenib) and Cotellic (cobimetinib) is included in guidelines for patients with recurrent anaplastic gliomas harboring BRAF V600E, including anaplastic oligodendroglioma (NCCN.org). detail...
BRAF V600E anaplastic astrocytoma sensitive Cobimetinib + Vemurafenib Guideline Actionable Combination of Zelboraf (vemurafenib) and Cotellic (cobimetinib) is included in guidelines for patients with recurrent anaplastic gliomas harboring BRAF V600E, including anaplastic astrocytoma (NCCN.org). detail...
BRAF V600E hairy cell leukemia predicted - sensitive Ruxolitinib + Vemurafenib Phase II Actionable In a Phase II trial (HCL-PG03), combined Zelboraf (vemurafenib) and Jakafi (ruxolitinib) therapy in relapsed or refractory hairy cell leukemia patients with BRAF V600E demonstrated safety, and led to complete response (CR) in 87% (26/30) of patients, including 17 (65%) with negative minimal residual disease, a progression-free survival rate of 78% at a median follow-up of 37 months, and a relapse-free survival rate of 85% in the 26 patients with a CR at a median follow-up of 34 months (PMID: 33979489). 33979489
BRAF V600E melanoma predicted - sensitive Belvarafenib Preclinical - Cell culture Actionable In a preclinical study, Belvarafenib (HM95573) treatment led to inhibition of tumor growth in a melanoma cell line xenograft model harboring BRAF V600E (PMID: 33953400). 33953400
BRAF V600E colorectal cancer predicted - sensitive Belvarafenib Case Reports/Case Series Actionable In a Phase I trial, Belvarafenib (HM95573) treatment in a colorectal cancer patient harboring BRAF V600E led to a tumor reduction of 39% after 8 weeks of treatment and a confirmed partial response at 12 weeks, and response to treatment was maintained for 8 weeks (PMID: 33953400; NCT03118817). 33953400
BRAF V600E anaplastic pleomorphic xanthoastrocytoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Mekinist (trametinib) and Tafinlar (dabrafenib) combination treatment resulted in an ongoing partial response 8 months after initiation of treatment in a patient with anaplastic pleomorphic xanthoastrocytoma harboring BRAF V600E, and a near complete response after 3 months of treatment in another patient with relapsed disease (PMID: 28984141). 28984141
BRAF V600E anaplastic astrocytoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), Zelboraf (vemurafenib) treatment resulted in a partial response in 1 of 5 patients with anaplastic astrrocytoma harboring BRAF V600E, with 2 other patients achieved stable disease lasting 14.9, and 5.6 months, respectively (PMID: 30351999; NCT01524978). 30351999
BRAF V600E pilocytic astrocytoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), Zelboraf (vemurafenib) treatment resulted in a partial response in a patient with pilocytic astrrocytoma harboring BRAF V600E who stayed on treatment for 15.3 months (PMID: 30351999; NCT01524978). 30351999
BRAF V600E ganglioglioma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (VE-BASKET), Zelboraf (vemurafenib) treatment resulted in a partial response in a patient with anaplastic ganglioglioma harboring BRAF V600E who stayed on treatment for 13.8 months (PMID: 30351999; NCT01524978). 30351999
BRAF V600E melanoma sensitive LY3214996 Preclinical - Cell line xenograft Actionable In a preclinical study, LY3214996 treatment in a melanoma cell line harboring BRAF V600E led to decreased cell proliferation in culture, and inhibition of tumor growth in a cell line xenograft model (PMID: 31744895). 31744895
BRAF V600E endometrial adenocarcinoma predicted - sensitive Dabrafenib + Rabeprazole + Rifampin Case Reports/Case Series Actionable In a Phase I trial, combination treatment with Tafinlar (dabrafenib), Rabeprazole, and Rifampin in a patient with metastatic endometrial adenocarcinoma harboring BRAF V600E, that recurred 11 years after original diagnosis and was refractory to treatment with chemotherapy, led to reduction of lung metastases and pelvic tissue mass at three months, but a slight increase in pelvic mass was observed at six months (PMID: 33537843; NCT01954043). 33537843
BRAF V600E endometrial adenocarcinoma predicted - sensitive Dabrafenib + Rabeprazole + Rifampin + Trametinib Case Reports/Case Series Actionable In a Phase I trial, the addition of Mekinist (trametinib) to combination treatment with Tafinlar (dabrafenib), Rabeprazole, and Rifampin resulted in a maintained radiographic response of lung metastases and stable pelvic mass size in a patient with metastatic endometrial adenocarcinoma harboring BRAF V600E, which lasted for 12 months (PMID: 33537843; NCT01954043). 33537843
BRAF V600E colorectal cancer predicted - sensitive LY3214996 Preclinical - Cell culture Actionable In a preclinical study, a lung cancer cell line harboring BRAF V600E and NF1 T2805I was sensitive to treatment with LY3214996 in culture, demonstrating decreased cell viability (PMID: 31744895). 31744895
BRAF V600E colorectal cancer sensitive ERAS-007 Preclinical - Pdx & cell culture Actionable In a preclinical study, ERAS-007 (ASN007) treatment inhibited cell proliferation and Erk signaling in colorectal cancer cell lines harboring BRAF V600E in culture, and induced tumor regression in two patient-derived xenograft (PDX) models (PMID: 34337566). 34337566
BRAF V600E diffuse large B-cell lymphoma sensitive ERAS-007 Preclinical - Cell culture Actionable In a preclinical study, ERAS-007 (ASN007) treatment inhibited proliferation of diffuse large B-cell lymphoma cells harboring BRAF V600E in culture (PMID: 34337566). 34337566
BRAF V600E melanoma sensitive ERAS-007 Preclinical - Pdx & cell culture Actionable In a preclinical study, ERAS-007 (ASN007) treatment inhibited proliferation and Erk signaling in a melanoma cell line harboring BRAF V600E in culture, and inhibited tumor growth in patient-derived xenograft (PDX) models, including a Zelboraf (vemurafenib)-resistant PDX model (PMID: 34337566). 34337566
BRAF V600E basal cell carcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in stable disease after three months of treatment in a patient with basal cell carcinoma harboring BRAF V600E (PMID: 33537843) 33537843
BRAF V600E glioblastoma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, a previously treated pediatric patient with epithelioid glioblastoma harboring BRAF V600E demonstrated stable disease for 10 months when treated with Tafinlar (dabrafenib) (PMID: 29621181). 29621181
BRAF V600E melanoma predicted - resistant KRT-232 Preclinical - Pdx Actionable In a preclinical study, patient-derived xenograft (PDX) models of melanoma harboring BRAF V600E demonstrated resistance to treatment with KRT-232 (AMG 232) (PMID: 32234759). 32234759
BRAF V600E melanoma predicted - sensitive Dabrafenib + KRT-232 + Trametinib Preclinical - Pdx Actionable In a preclinical study, the addition of KRT-232 (AMG 232) to the combination treatment of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a synergistic effect, leading to a greater decrease in tumor growth and tumor size compared to either KRT-232 (AMG 232) alone or Tafinlar (dabrafenib) plus Mekinist (trametinib) in patient-derived xenograft (PDX) models of melanoma harboring BRAF V600E (PMID: 32234759). 32234759
BRAF V600E melanoma sensitive KRT-232 + Navitoclax Preclinical - Pdx Actionable In a preclinical study, the combination of KRT-232 (AMG 232) and Navitoclax (ABT-263) resulted in a greater response than either drug alone in patient-derived xenograft (PDX) models of melanoma harboring BRAF V600E, leading to tumor regression (PMID: 32234759). 32234759
BRAF V600E papillary thyroid carcinoma sensitive Dabrafenib Clinical Study - Cohort Actionable In a clinical study, Tafinlar (dabrafenib) treatment stimulated radioiodine uptake in 60% (6/10) of patients with metastatic iodine-refractory papillary thyroid cancer harboring BRAF V600E (PMID: 25549723; NCT01534897). 25549723
BRAF V600E papillary thyroid carcinoma sensitive Dabrafenib Guideline Actionable Tafinlar (dabrafenib) is included in guidelines for patients with recurrent, advanced, or metastatic thyroid papillary carcinoma harboring BRAF V600E for whom clinical trials are not available or appropriate (NCCN.org). detail...
BRAF V600E follicular thyroid carcinoma sensitive Dabrafenib Guideline Actionable Tafinlar (dabrafenib) is included in guidelines for patients with recurrent, advanced, or metastatic thyroid follicular carcinoma harboring BRAF V600E for whom clinical trials are not available or appropriate (NCCN.org). detail...
BRAF V600E follicular thyroid carcinoma sensitive Vemurafenib Guideline Actionable Zelboraf (vemurafenib) is included in guidelines for patients with recurrent, advanced, or metastatic thyroid follicular carcinoma harboring BRAF V600E for whom clinical trials are not available or appropriate (NCCN.org). detail...
BRAF V600E oncocytic carcinoma of the thyroid sensitive Vemurafenib Guideline Actionable Zelboraf (vemurafenib) is included in guidelines for patients with recurrent, advanced, or metastatic thyroid Hurthle cell carcinoma harboring BRAF V600E for whom clinical trials are not available or appropriate (NCCN.org). detail...
BRAF V600E thyroid gland carcinoma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a Phase I trial, Tafinlar (dabrafenib) treatment resulted in a partial response in 29% (4/14) and stable disease in 43% (6/14) of patients with thyroid carcinoma harboring BRAF V600E, with 64% (9/14) of patients achieved at least a 10% decrease by RECIST, and a median progression-free survival of 11.3 months among responders (PMID: 25285888; NCT00880321). 25285888
BRAF V600E melanoma sensitive ASTX029 Preclinical - Cell line xenograft Actionable In a preclinical study, ASTX029 treatment reduced Erk and Rsk phosphorylation, induced cell-cycle arrest, and inhibited growth of a melanoma cell line harboring BRAF V600E in culture and inhibited tumor growth in cell line xenograft models, and was also effective against cells with acquired resistance to Zelboraf (vemurafenib) or Koselugo (selumetinib) (PMID: 34330842). 34330842
BRAF V600E melanoma sensitive ASTX029 + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, combination treatment with ASTX029 and Zelboraf (vemurafenib) resulted in decreased viability of melanoma cells harboring BRAF V600E compared to either agent alone in culture (PMID: 34330842). 34330842
BRAF V600E colorectal adenocarcinoma sensitive ASTX029 Preclinical - Cell line xenograft Actionable In a preclinical study, ASTX029 treatment reduced Erk and Rsk phosphorylation and inhibited growth of colorectal adenocarcinoma cell lines harboring BRAF V600E in culture, and inhibited tumor growth and induced tumor regression in cell line xenograft models (PMID: 34330842). 34330842
BRAF V600E pancreatic ductal adenocarcinoma predicted - sensitive Cobimetinib + Gemcitabine + Nab-paclitaxel Case Reports/Case Series Actionable In a clinical case study, Gemzar (gemcitabine), Abraxane (nab-paclitaxel), and Cotellic (cobimetinib) combination treatment resulted in a complete radiologic response within 6 months of initiation lasting at least 16 months in a patient with microsatellite stable, KRAS wild-type pancreatic ductal adenocarcinoma harboring BRAF V600E (PMID: 34667063). 34667063
BRAF V600E high grade glioma predicted - sensitive LY3009120 Preclinical - Patient cell culture Actionable In a preclinical study, LY3009120 treatment led to inhibition of cell viability and growth in a patient-derived glioma cell line harboring BRAF V600E in culture (PMID: 34433654). 34433654
BRAF V600E high grade glioma sensitive LY3009120 + Vemurafenib Preclinical - Patient cell culture Actionable In a preclinical study, combination treatment with Zelboraf (vemurafenib) and LY3009120 treatment led to greater inhibition of cell growth compared to either agent alone in a patient-derived glioma cell line harboring BRAF V600E in culture (PMID: 34433654). 34433654
BRAF V600E high grade glioma predicted - sensitive Belvarafenib Preclinical - Cell culture Actionable In a preclinical study, Belvarafenib (HM95573) treatment led to inhibition of cell viability in a patient-derived glioma cell line harboring BRAF V600E in culture (PMID: 34433654). 34433654
BRAF V600E high grade glioma no benefit ZM336372 Preclinical - Patient cell culture Actionable In a preclinical study, ZM336372 treatment did not lead to inhibition of cell growth in a patient-derived glioma cell line harboring BRAF V600E in culture (PMID: 34433654). 34433654
BRAF V600E high grade glioma predicted - sensitive Vemurafenib + ZM336372 Preclinical - Patient cell culture Actionable In a preclinical study, combination treatment with Zelboraf (vemurafenib) and ZM336372 treatment led to inhibition of cell growth in a patient-derived glioma cell line harboring BRAF V600E in culture (PMID: 34433654). 34433654
BRAF V600E high grade glioma sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a partial response in a pediatric patient with posterior fossa high grade glioma harboring BRAF V600E and deletion of CDKN2A (PMID: 39399174). 39399174
BRAF V600E high grade glioma sensitive Dabrafenib + Trametinib Phase II Actionable In a Phase II trial (ROAR), Tafinlar (dabrafenib) and Mekinist (trametinib) combination treatment resulted in an objective response rate of 33% (15/45, 3 complete responses, 12 partial responses) in patients with high-grade glioma harboring BRAF V600E, with a median duration of response of 36.9 months, a median progression-free survival of 3.8 months, and an overall survival of 17.6 months (PMID: 34838156; NCT02034110). 34838156
BRAF V600E high grade glioma sensitive Dabrafenib + Trametinib Phase II Actionable In a Phase II trial, Tafinlar (dabrafenib) and Mekinist (trametinib) combination treatment resulted in an overall response rate of 56.1% (23/41; 12 complete, 11 partial responses), a clinical benefit rate of 65.9% (27/41), median duration of response of 22.2 months, median progression-free survival of 9.0 months, and median overall survival of 32.8 months in pediatric patients with relapsed or refractory high-grade glioma harboring BRAF V600E (PMID: 37643378; NCT02684058). 37643378
BRAF V600E high grade glioma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) in combination with Mekinist (trametinib) is included in guidelines as adjuvant therapy for pediatric patients with diffuse high-grade gliomas harboring BRAF V600E, or as a preferred regimen for patients with recurrent or progressive disease (NCCN.org). detail...
BRAF V600E triple-receptor negative breast cancer predicted - sensitive Nab-paclitaxel + Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) and Abraxane (nab-paclitaxel) combination treatment resulted in regression of some metastatic pulmonary lesions and a progression-free survival of 4.4 months in a patient with triple-negative breast cancer harboring BRAF V600E, but a biopsy in lesions that progressed showed acquisition of additional mutations in PDGFRB, NF2, GRM3, MLH1, FOXA1, LRP1B, and AR amplification (PMID: 34818649). 34818649
BRAF V600E pancreatic ductal adenocarcinoma predicted - sensitive Trametinib + Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) and Mekinist (trametinib) combination treatment resulted in decreased tumor markers in a patient with pancreatic ductal adenocarcinoma harboring BRAF V600E, as well as a germline ATM mutation, and the patient received 13 months of combination therapy and was followed for 24 months (PMID: 35145907). 35145907
BRAF V600E lung papillary adenocarcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) and Mekinist (trametinib) combination treatment resulted in regression of the lesions in the chest, abdomen and brain in a patient with lung papillary carcinoma harboring BRAF V600E but progression was observed 3 months later (PMID: 34178685). 34178685
BRAF V600E pancreatic ductal adenocarcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, combination treatment with Tafinlar (dabrafenib) and Mekinist (trametinib) led to a partial response after 1 month of treatment in a metastatic pancreatic ductal adenocarcinoma patient harboring BRAF V600E, followed by disease progression, which was detected 12 months later (PMID: 35382161). 35382161
BRAF V600E pancreatic ductal adenocarcinoma predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a clinical case study, combination treatment with Cotellic (cobimetinib) and Zelboraf (vemurafenib) led to a partial response after 1 month of treatment in a microsatellite stable pancreatic ductal adenocarcinoma patient with BRAF V600E and amplification of FGFR1 and NOTCH2, with significant decrease in hepatic metastatic lesions and undetectable lung lesions at 6 months, and a progression-free survival of at least 6 months (PMID: 35382161). 35382161
BRAF V600E IDH-wildtype glioblastoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in disease stability over 15 months in a patient with epithelioid glioblastoma harboring BRAF V600E, who previously progressed on conventional treatment options (PMID: 34232949). 34232949
BRAF V600E lung non-small cell carcinoma predicted - sensitive LTT462 + LXH 254 Case Reports/Case Series Actionable In a Phase Ib trial, LTT462 and LXH 254 combination therapy was well tolerated and resulted in an unconfirmed partial response (PR) in 4% (2/49) and stable disease (SD) in 33% (16/49) of patients with advanced or metastatic KRAS- or BRAF-mutant non-small cell lung cancer, with 1 patient harboring BRAF V600E achieving an unconfirmed PR and 1 achieving SD with over 25% tumor reduction (Ann Oncol 31 (suppl 4):S881-S882; NCT02974725). detail...
BRAF V600E large cell carcinoma predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a clinical study, a patient with poorly differentiated large cell carcinoma harboring BRAF V600E experienced a complete response lasting at least 2 years on combination treatment with Cotellic (cobimetinib) and Zelboraf (vemurafenib) (PMID: 35046062). 35046062
BRAF V600E Advanced Solid Tumor sensitive Dabrafenib + Trametinib FDA approved Actionable In 3 Phase II trials (ROAR, NCI-MATCH, X2101) that supported FDA approval, Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy demonstrated safety and efficacy in adult and pediatric (6 years or older) patients with advanced solid tumors harboring BRAF V600E (PMID: 34838156, PMID: 32818466, J Clin Oncol 37, no. 15_suppl (May 20, 2019) 3002, J Clin Oncol 38, no. 15_suppl (May 20, 2020) 10506; NCT02034110, NCT02465060, NCT02124772). 32818466 detail... detail... 34838156 detail... detail...
BRAF V600E Advanced Solid Tumor sensitive Dabrafenib + Trametinib Phase II Actionable In a Phase II trial (NCI-MATCH), Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy resulted in an objective response rate of 33.3% (11/33) in patients with advanced solid tumors other than melanoma, thyroid, colorectal, and lung cancer harboring BRAF V600E, with a median duration of response of 12 months, median progression-free survival of 9.4 months, and median overall survival not reached (J Clin Oncol 37, no. 15_suppl (May 20, 2019) 3002; NCT02465060). detail...
BRAF V600E hairy cell leukemia sensitive Dabrafenib + Trametinib Phase II Actionable In a Phase II trial (ROAR), treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) demonstrated safety and resulted in an overall response rate of 89.1% (49/55; 36 complete responses, 13 partial responses) in patients with heavily pretreated recurrent/refractory hairy cell leukemia harboring BRAF V600E, with 24-month duration of response, progression-free-survival, and overall survival rates of 97.7%, 94.4% and 94.5%, respectively (PMID: 36108341; NCT02034110). 36108341
BRAF V600E melanoma predicted - sensitive PF-07284890 Preclinical - Cell line xenograft Actionable In a preclinical study, PF-07284890 treatment led to inhibition of tumor growth, tumor regression, and survival benefit in an intracranial model, and tumor growth inhibition in a subcutaneous cell line xenograft model of melanoma harboring BRAF V600E (Cancer Res 2021;81(13_Suppl):Abstract nr 1473). detail...
BRAF V600E colon cancer predicted - sensitive ABM-1310 + Binimetinib Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment with ABM-1310 and Mektovi (binimetinib) led to inhibition of tumor growth in a colon cancer cell line xenograft model harboring BRAF V600E (Cancer Res (2020) 80 (16_Supplement): 4038). detail...
BRAF V600E colon cancer predicted - sensitive ABM-1310 + Cetuximab Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment with ABM-1310 and Erbitux (cetuximab) led to inhibition of tumor growth in a colon cancer cell line xenograft model harboring BRAF V600E (Cancer Res (2020) 80 (16_Supplement): 4038). detail...
BRAF V600E Advanced Solid Tumor sensitive Cobimetinib + Vemurafenib Phase II Actionable In a Phase II trial (TAPUR), treatment with the combination of Zelboraf (vemurafenib) and Cotellic (cobimetinib) resulted in an objective response rate of 57% (16/28, 2 complete responses (CR), 14 partial responses (PR)) and a disease control rate (CR + PR + stable disease at 16 wks (SD16+)) of 68% in patients with advanced solid tumors harboring BRAF mutations, with 2 CR, 13 PR, and 2 SD16+ in a total of 26 patients harboring BRAF V600E (PMID: 38096472; NCT02693535). 38096472
BRAF V600E breast cancer predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (TAPUR), Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy resulted in a complete response that lasted 170 days and 2 partial responses out of 4 patients with advanced breast cancer harboring BRAF V600E (PMID: 38096472; NCT02693535). 38096472
BRAF V600E ovarian cancer predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (TAPUR), Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy resulted in one complete response lasting 5 weeks, 2 partial responses, and 1 with stable disease lasting more than 16 weeks out of 6 patients with advanced ovarian cancer harboring BRAF V600E (PMID: 38096472; NCT02693535). 38096472
BRAF V600E melanoma predicted - sensitive IMM-1-104 Preclinical - Cell line xenograft Actionable In a preclinical study, IMM-1-104 treatment led to tumor growth inhibition in a cell line xenograft model of melanoma harboring BRAF V600E (Mol Cancer Ther 2021;20(12 Suppl):Abstract nr P252). detail...
BRAF V600E ovary epithelial cancer sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines as systemic therapy for patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer harboring BRAF V600E (NCCN.org). detail...
BRAF V600E ovarian serous carcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy resulted in a complete response after 8 months of treatment in a patient with low-grade serous ovarian carcinoma harboring BRAF V600E (PMID: 33043759). 33043759
BRAF V600E ovarian serous carcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) and Mekinist (trametinib) combination treatment resulted in a partial response lasting at least 2.5 years in one patient with low grade serous ovarian carcinoma harboring BRAF V600E, and resulted in a complete metabolic response after 4 months of treatment in a second patient (PMID: 35242981). 35242981
BRAF V600E ovarian serous carcinoma predicted - sensitive Binimetinib + Encorafenib Case Reports/Case Series Actionable In a clinical case study, Braftovi (encorafenib) and Mektovi (binimetinib) combination therapy resulted in durable stable disease lasting at least 3.5 years in a patient with low-grade serous ovarian carcinoma harboring BRAF V600E, who was previously treated with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) (PMID: 33043759). 33043759
BRAF V600E ovary serous adenocarcinoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in a partial response lasting more than 21 months in a patient with low grade serous ovarian adenocarcinoma harboring BRAF V600E (PMID: 26490654). 26490654
BRAF V600E central nervous system cancer predicted - sensitive Ulixertinib Case Reports/Case Series Actionable In a Phase II trial (APEC1621J), Ulixertinib (BVD-523) treatment resulted in a 6-month progression-free survival rate of 37% but no objective response in pediatric and young adult patients with advanced solid tumors harboring MAPK pathway activation, however, a patient with glioneuronal tumor harboring a BRAF V600E achieved prolonged stable disease and remained on treatment for 9 cycles (J Clin Oncol 40, no. 16_suppl (June 01, 2022) 3009; NCT03698994). detail...
BRAF V600E pancreatic ductal adenocarcinoma predicted - sensitive Ulixertinib Case Reports/Case Series Actionable In an expanded access program (ULI-EAP-100), Ulixertinib (BVD-523) treatment resulted in clinical benefit in a patient with pancreatic ductal adenocarcinoma harboring BRAF V600E who stayed on treatment for 413 days (J Clin Oncol 40, no. 16_suppl (June 01, 2022) e15101; NCT04566393). detail...
BRAF V600E colorectal cancer predicted - sensitive Cetuximab + Encorafenib + Ulixertinib Case Reports/Case Series Actionable In an expanded access program (ULI-EAP-100), Ulixertinib (BVD-523), Braftovi (encorafenib), and Erbitux (cetuximab) combination therapy resulted in a complete response in a patient with colorectal cancer harboring BRAF V600E (J Clin Oncol 40, no. 16_suppl (June 01, 2022) e15101; NCT04566393). detail...
BRAF V600E melanoma predicted - sensitive Abemaciclib Preclinical - Cell line xenograft Actionable In a preclinical study, Verzenio (abemaciclib) induced apoptosis in Zelboraf (vemurafenib)-resistant melanoma cells harboring BRAF V600E in culture, and induced tumor regression in xenograft models (PMID: 25122067). 25122067
BRAF V600E spermatic cord cancer predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a regression of the lung metastasis and progression-free survival of 6.5 months in a patient with undifferentiated sarcoma of the spermatic cord harboring BRAF V600E (PMID: 36157689). 36157689
BRAF V600E cholangiocarcinoma predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination treatment resulted in reduction of the pulmonary nodules and hepatic lesions 6 months after treatment initiation in a patient with metastatic cholangiocarcinoma harboring BRAF V600E, who maintained a stable disease and remained on treatment at 20 months (PMID: 33669326). 33669326
BRAF V600E cholangiocarcinoma predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (TAPUR), Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy resulted in a partial response in a patient with cholangiocarcinoma harboring BRAF V600E (PMID: 38096472; NCT02693535). 38096472
BRAF V600E oncocytic carcinoma of the thyroid sensitive Dabrafenib + Trametinib Guideline Actionable The combination of Tafinlar (dabrafenib) and Mekinist (trametinib) is included in guidelines for patients with thryoid Hürthle cell carcinoma harboring BRAF V600E who have progressed on therapy and have no further treatment options (NCCN.org). detail...
BRAF V600E follicular thyroid carcinoma sensitive Dabrafenib + Trametinib Guideline Actionable The combination of Tafinlar (dabrafenib) and Mekinist (trametinib) is included in guidelines for patients with follicular thyroid carcinoma harboring BRAF V600E who have progressed on therapy and have no further treatment options (NCCN.org). detail...
BRAF V600E colorectal adenocarcinoma sensitive RO5126766 Preclinical - Cell line xenograft Actionable In a preclinical study, RO5126766 (VS-6766) inhibited tumor growth in a cell line xenograft model of colorectal adenocarcinoma harboring BRAF V600E (PMID: 23667175). 23667175
BRAF V600E epithelial predominant Wilms' tumor predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy resulted in a complete radiographic response 4 months after treatment initiation that was maintained for at least 12 months in a pediatric patient with metastatic epithelial-predominant Wilms tumor harboring BRAF V600E (PMID: 32238401). 32238401
BRAF V600E gastrointestinal stromal tumor predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) treatment resulted in a 20% decrease in tumor size after 24 weeks in a patient with a gastrointestinal stromal tumor harboring BRAF V600E (PMID: 23470635). 23470635
BRAF V600E gastrointestinal stromal tumor predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, third-line treatment with Tafinlar (dabrafenib) resulted in a partial response lasting 19 months in a patient with gastrointestinal stromal tumor harboring BRAF V600E (PMID: 38756640). 38756640
BRAF V600E ampulla of Vater adenocarcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines as first-line therapy with good (ECOG 0-1; category 3) or poor (category 2B) performance status, or as subsequent therapy, for metastatic ampullary adenocarcinoma patients harboring BRAF V600E (NCCN.org). detail...
BRAF V600E pancreatic adenocarcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines as first-line therapy for patients with locally advanced (category 2A) or metastatic (category 2B) pancreatic adenocarcinoma harboring BRAF V600E, and as subsequent-line therapy for patients with locally advanced, recurrent, or metastatic disease (category 2A) (NCCN.org). detail...
BRAF V600E Her2-receptor negative breast cancer predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a complete response in the liver and bone lesions with a progression-free survival of 9 months in a patient with metastatic ERBB2 (HER2)-negative, hormone receptor-positive breast cancer harboring BRAF V600E (PMID: 36531075). 36531075
BRAF V600E colorectal cancer sensitive Pyrvinium + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment with Zelboraf (vemurafenib) and Pyrvinium resulted in decreased viability in colorectal cancer cell lines harboring BRAF V600E in culture, and led to synergistic inhibition of tumor growth in a cell line xenograft model (PMID: 36198029). 36198029
BRAF V600E colorectal cancer sensitive Axitinib + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment with Zelboraf (vemurafenib) and Inlyta (axitinib) resulted in decreased cell viability in colorectal cancer cell lines harboring BRAF V600E in culture, and led to synergistic inhibition of tumor growth in cell line xenograft models (PMID: 36198029). 36198029
BRAF V600E colorectal cancer sensitive Panobinostat + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Mekinist (trametinib) and Farydak (panobinostat) synergistically inhibited cell viability and induced apoptosis in a colorectal cancer cell line harboring BRAF V600E in culture (PMID: 36343387). 36343387
BRAF V600E colorectal cancer sensitive Trametinib + Vorinostat Preclinical - Cell culture Actionable In a preclinical study, the combination of Mekinist (trametinib) and Zolinza (vorinostat) resulted in greater apoptotic activity compared to either agent alone in a colorectal cancer cell line harboring BRAF V600E in culture (PMID: 36343387). 36343387
BRAF V600E colorectal cancer sensitive Romidepsin + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Mekinist (trametinib) and Istodax (romidepsin) resulted in greater apoptotic activity compared to either agent alone in a colorectal cancer cell line harboring BRAF V600E in culture (PMID: 36343387). 36343387
BRAF V600E colorectal cancer sensitive Mocetinostat + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Mekinist (trametinib) and Mocetinostat (MGCD0103) resulted in greater apoptotic activity compared to either agent alone in a colorectal cancer cell line harboring BRAF V600E in culture (PMID: 36343387). 36343387
BRAF V600E colorectal cancer sensitive Entinostat + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Mekinist (trametinib) and Entinostat (MS-275) resulted in greater apoptotic activity compared to either agent alone in a colorectal cancer cell line harboring BRAF V600E in culture (PMID: 36343387). 36343387
BRAF V600E colorectal cancer sensitive Trametinib + Valproic acid Preclinical - Cell culture Actionable In a preclinical study, the combination of Mekinist (trametinib) and Valproic acid resulted in greater apoptotic activity compared to either agent alone in a colorectal cancer cell line harboring BRAF V600E in culture (PMID: 36343387). 36343387
BRAF V600E colorectal cancer sensitive Panobinostat + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, the combination of Zelboraf (vemurafenib) and Farydak (panobinostat) resulted in greater apoptotic activity compared to either agent alone in a colorectal cancer cell line harboring BRAF V600E in culture (PMID: 36343387). 36343387
BRAF V600E salivary gland cancer sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines for patients with recurrent, unresectable, or metastatic salivary gland tumors harboring BRAF V600E (NCCN.org). detail...
BRAF V600E Advanced Solid Tumor predicted - sensitive Erlotinib + Vemurafenib Case Reports/Case Series Actionable In a Phase Ib/II trial (EVICT), the combination of Zelboraf (vemurafenib) and Tarceva (erlotinib) resulted in an overall response rate of 43% (3/7), a clinical benefit rate of 100%, and a median PFS of 5.5 months in patients with advanced solid tumors other tan colorectal cancer harboring BRAF V600E (PMID: 36638198). 36638198
BRAF V600E pleomorphic xanthoastrocytoma sensitive Binimetinib + Ulixertinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Ulixertinib (BVD-523) and Mektovi (binimetinib) inhibited proliferation and had a synergistic effect on induction of apoptosis and inhibition of MAPK pathway activity in a pleomorphic xanthoastrocytoma cell line harboring BRAF V600E in culture, and improved the progressive disease/partial response ratio compared to either drug alone in a zebrafish xenograft model (PMID: 35882450). 35882450
BRAF V600E pleomorphic xanthoastrocytoma predicted - sensitive Selumetinib + Ulixertinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Ulixertinib (BVD-523) and Koselugo (selumetinib) inhibited proliferation in a pleomorphic xanthoastrocytoma cell line harboring BRAF V600E in culture (PMID: 35882450). 35882450
BRAF V600E pleomorphic xanthoastrocytoma sensitive Ulixertinib + Vinblastine Preclinical - Cell culture Actionable In a preclinical study, the combination of Ulixertinib (BVD-523) and Velban (vinblastine) inhibited proliferation and had a synergistic effect on induction of apoptosis in a pleomorphic xanthoastrocytoma cell line harboring BRAF V600E in culture (PMID: 35882450). 35882450
BRAF V600E pleomorphic xanthoastrocytoma predicted - sensitive Trametinib + Ulixertinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Ulixertinib (BVD-523) and Mekinist (trametinib) had a synergistic effect on the induction of apoptosis in a pleomorphic xanthoastrocytoma cell line harboring BRAF V600E in culture (PMID: 35882450). 35882450
BRAF V600E pleomorphic xanthoastrocytoma sensitive Navitoclax + Ulixertinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Ulixertinib (BVD-523) and Navitoclax (ABT-263) synergistically induced apoptosis in a pleomorphic xanthoastrocytoma cell line harboring BRAF V600E in culture, and improved the partial response rate compared to either drug alone in a zebrafish xenograft model (PMID: 35882450). 35882450
BRAF V600E pleomorphic xanthoastrocytoma sensitive Ulixertinib Preclinical - Cell line xenograft Actionable In a preclinical study, Ulixertinib (BVD-523) reduced metabolic activity and inhibited MAPK pathway activity in a pleomorphic xanthoastrocytoma cell line harboring BRAF V600E in culture, and inhibited tumor growth and improved survival compared to vehicle (48.5 days vs 30 days, respectively) in a xenograft model (PMID: 35882450). 35882450
BRAF V600E thyroid gland carcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Mekinist (trametinib) and Tafinlar (dabrafenib) combination treatment resulted in a partial response after 3 months in a patient with metastatic squamous cell carcinoma of the thyroid harboring BRAF V600E (PMID: 34956922). 34956922
BRAF V600E melanoma predicted - sensitive DS03090629 Preclinical - Cell line xenograft Actionable In a preclinical study, DS03090629 inhibited Erk and Mek phosphorylation and proliferation in a melanoma cell line harboring BRAF V600E in culture and led to tumor stasis in a cell line xenograft model (PMID: 36622773). 36622773
BRAF V600E melanoma sensitive Dabrafenib + DS03090629 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of DS03090629 and Tafinlar (dabrafenib) inhibited Erk phosphorylation and proliferation in a melanoma cell line harboring BRAF V600E in culture and induced tumor regression in a cell line xenograft model (PMID: 36622773). 36622773
BRAF V600E IDH-wildtype glioblastoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy resulted in a partial response lasting 9 months in a patient with metastatic MGMT unmethylated, IDH-wildtype glioblastoma harboring BRAF V600E along with amplification of MYC and TERT and loss of CDKN2A/B (PMID: 36825105). 36825105
BRAF V600E gastroesophageal junction adenocarcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines as second-line or subsequent therapy for patients with locally advanced, recurrent, or metastatic gastroesophageal junction adenocarcinoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E esophagus adenocarcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines as second-line or subsequent therapy for patients with locally advanced, recurrent, or metastatic esophageal adenocarcinoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E esophagus squamous cell carcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines as second-line or subsequent therapy for patients with locally advanced, recurrent, or metastatic esophageal squamous cell carcinoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E ovarian serous cystadenocarcinoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in a complete radiological response that lasted for 3 years in a patient with low-grade serous cystadenocarcinoma of the ovary harboring BRAF V600E (PMID: 36967525). 36967525
BRAF V600E colorectal cancer resistant Cetuximab Guideline Actionable Erbitux (cetuximab), as a monotherapy, is not indicated for use in metastatic colorectal cancer patients with BRAF V600E (PMID: 36307056; ESMO.org). 36307056 detail...
BRAF V600E colorectal cancer resistant Panitumumab Guideline Actionable Vectibix (panitumumab), as a monotherapy, is not indicated for use in metastatic colorectal cancer patients with BRAF V600E (PMID: 36307056; ESMO.org). 36307056 detail...
BRAF V600E colorectal cancer sensitive Dabrafenib + Spartalizumab + Trametinib Phase II Actionable In a Phase II trial, the combination of Spartalizumab (PDR001), Tafinlar (dabrafenib), and Mekinist (trametinib) was well tolerated and resulted in a confirmed objective response rate of 24.3% (9/37, 2 complete responses), disease control rate of 70.3% (26/37), median progression-free survival of 4.3 months, median duration of response of 7.4 months, and median overall survival of 13.6 months in patients with metastatic colorectal cancer harboring BRAF V600E (PMID: 36702949; NCT03668431). 36702949
BRAF V600E gastrointestinal stromal tumor sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) in combination with Mekinist (trametinib) is included in guidelines as neoadjuvant therapy for patients with resectable disease and as first-line systemic therapy for patients with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring BRAF V600E (NCCN.org). detail...
BRAF V600E stomach cancer sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy is included in guidelines as second-line or subsequent therapy for patients with unresectable locally advanced, recurrent, or metastatic gastric cancer harboring BRAF V600E (NCCN.org). detail...
BRAF V600E colorectal cancer sensitive Saracatinib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, the combination of Zelboraf (vemurafenib) and Saracatinib (AZD0530) synergistically inhibited viability in colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Dasatinib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, the combination of Zelboraf (vemurafenib) and Sprycel (dasatinib) synergistically inhibited viability in colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Bosutinib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, the combination of Zelboraf (vemurafenib) and Bosulif (bosutinib) synergistically inhibited viability in colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Dasatinib + Gefitinib + Vemurafenib Preclinical - Pdx & cell culture Actionable In a preclinical study, the combination of Zelboraf (vemurafenib), Iressa (gefitinib), and Sprycel (dasatinib) synergistically inhibited viability in colorectal cancer cell lines harboring BRAF V600E in culture and resulted in greater tumor growth inhibition and tumor regression in patient-derived xenograft (PDX) models to a greater degree than the doublet combinations of Zelboraf (vemurafenib) and Iressa (gefitinib) or Zelboraf (vemurafenib) and Sprycel (dasatinib) (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Dasatinib + Gefitinib + PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PLX4720, Iressa (gefitinib), and Sprycel (dasatinib) inhibited tumor growth in colorectal cancer cell line xenograft models harboring BRAF V600E to a greater degree than the combinations of PLX4720 and Iressa (gefitinib) or PLX4720 and Sprycel (dasatinib) (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Gefitinib + PLX4720 + Saracatinib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PLX4720, Iressa (gefitinib), and Saracatinib (AZD0530) inhibited tumor growth in colorectal cancer cell line xenograft models harboring BRAF V600E to a greater degree than the combinations of PLX4720 and Iressa (gefitinib) or PLX4720 and Saracatinib (AZD0530) (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Celecoxib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, the combination of Zelboraf (vemurafenib) and Celecoxib synergistically inhibited viability in colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Celecoxib + Gefitinib + Trametinib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, the addition of Celecoxib to the combination of Zelboraf (vemurafenib), Mekinist (trametinib), and Iressa (gefitinib) synergistically inhibited viability in colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Celecoxib + Trametinib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, the addition of Celecoxib to the combination of Zelboraf (vemurafenib) and Mekinist (trametinib) synergistically inhibited viability in colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Celecoxib + Dabrafenib + Trametinib Preclinical - Pdx Actionable In a preclinical study, the addition of Celecoxib to the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) inhibited tumor growth and induced tumor regression in colorectal cancer patient-derived xenograft (PDX) models harboring BRAF V600E (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Celecoxib + Dabrafenib + Panitumumab + Trametinib Preclinical - Pdx Actionable In a preclinical study, the addition of Celecoxib to the combination of Tafinlar (dabrafenib), Mekinist (trametinib), and Vectibix (panitumumab) inhibited tumor growth and induced tumor regression in colorectal cancer patient-derived xenograft (PDX) models harboring BRAF V600E (PMID: 36759733). 36759733
BRAF V600E colorectal cancer sensitive Celecoxib + Encorafenib + Panitumumab Preclinical - Pdx Actionable In a preclinical study, the addition of Celecoxib to the combination of Braftovi (encorafenib) and Vectibix (panitumumab) inhibited tumor growth and induced tumor regression in colorectal cancer patient-derived xenograft (PDX) models harboring BRAF V600E to a greater degree than the combination of Braftovi (encorafenib) and Vectibix (panitumumab) (PMID: 36759733). 36759733
BRAF V600E multiple myeloma sensitive Binimetinib + Encorafenib Phase II Actionable In a Phase II trial (BIRMA), Mektovi (binimetinib) plus Braftovi (encorafenib) treatment demonstrated safety and efficacy in relapsed/refractory multiple myeloma patients with BRAF V600E, with an overall response rate of 83% (10/12, 1 complete response (CR), 2 near-CR, 6 very good partial responses (PR), and 1 PR), a median duration of response of 4.8 mo, a median progression-free survival of 5.6 mo, and a median overall survival not reached but 66% at 12 mo and 55% at 24 mo (PMID: 36608320; NCT02834364). 36608320
BRAF V600E melanoma predicted - sensitive PHI-501 Preclinical - Cell line xenograft Actionable In a preclinical study, PHI-501 inhibited growth and migration and induced apoptosis in a melanoma cell line harboring BRAF V600E and induced dose-dependent tumor growth inhibition in a cell line xenograft model (Cancer Res (2023) 83 (7_Supplement): 1627). detail...
BRAF V600E melanoma predicted - sensitive BGB3245 Case Reports/Case Series Actionable In a Phase I trial, BGB3245 treatment demonstrated manageable safety and resulted in a disease control rate of 48% (16/33,1 complete response, 5 confirmed partial responses (PR), 2 unconfirmed PR, and 8 stable disease > 24 weeks) in patients with advanced solid tumors harboring MAPK pathway alterations, including 1 complete response and 1 confirmed partial response in patients with melanoma harboring BRAF V600E (Cancer Res (2023) 83 (8_Supplement): CT031). detail...
BRAF V600E ovarian serous carcinoma predicted - sensitive BGB3245 Case Reports/Case Series Actionable In a Phase I trial, BGB3245 treatment demonstrated manageable safety and resulted in a disease control rate of 48% (16/33,1 complete response, 5 confirmed partial responses (PR), 2 unconfirmed PR, and 8 stable disease > 24 weeks) in patients with advanced solid tumors harboring MAPK pathway alterations, including a partial response in a patient with low grade serous ovarian carcinoma harboring BRAF V600E (Cancer Res (2023) 83 (8_Supplement): CT031). detail...
BRAF V600E cholangiocarcinoma predicted - sensitive BGB3245 Case Reports/Case Series Actionable In a Phase I trial, BGB3245 treatment demonstrated manageable safety and resulted in a disease control rate of 48% (16/33,1 complete response, 5 confirmed partial responses (PR), 2 unconfirmed PR, and 8 stable disease > 24 weeks) in patients with advanced solid tumors harboring MAPK pathway alterations, including a partial response in a patient with cholangiocarcinoma harboring BRAF V600E (Cancer Res (2023) 83 (8_Supplement): CT031). detail...
BRAF V600E lung non-small cell carcinoma predicted - sensitive Lifirafenib + PD-0325901 Phase I Actionable In a Phase Ib trial, Lifirafenib (BGB-283) and PD-0325901 combination treatment demonstrated safety and activity in patients with advanced solid tumors harboring MAPK pathway alterations, resulting in an objective response rate of 27.8% (15/54, 1 complete and 14 partial responses), including an objective response in a patient with non-small cell lung cancer harboring BRAF V600E (Cancer Res (2023) 83 (8_Supplement): CT033). detail...
BRAF V600E lung non-small cell carcinoma sensitive Binimetinib + Encorafenib Guideline Actionable Combination of Braftovi (encorafenib) and Mektovi (binimetinib) is included in guidelines as a first-line or subsequent therapy for advanced or metastatic non-small cell lung cancer patients harboring BRAF V600E mutations (NCCN.org). detail...
BRAF V600E lung non-small cell carcinoma sensitive Binimetinib + Encorafenib FDA approved - On Companion Diagnostic Actionable In a Phase II trial (PHAROS) that supported FDA approval, Braftovi (encorafenib) and Mektovi (binimetinib) combination therapy resulted in an objective response rate (ORR) of 75% (44/59; 9 complete, 35 partial responses) with a duration of response (DOR) lasting 12 or more months in 59% of treatment-naive patients, and an ORR of 46% (18/39) with a DOR lasting 12 or more months in 33% of previously treated patients with metastatic non-small cell lung cancer harboring BRAF V600E (PMID: 37270692; NCT03915951). detail... detail... detail... 37270692
BRAF V600E colorectal cancer sensitive Binimetinib + Encorafenib + PF-07284892 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of PF-07284892, Mektovi (binimetinib), and Braftovi (encorafenib) inhibited Erk phosphorylation in colorectal cancer cells harboring BRAF V600E in culture and induced tumor regression in a cell line xenograft model (PMID: 37269335). 37269335
BRAF V600E colorectal cancer predicted - sensitive Cetuximab + Encorafenib + PF-07284892 Case Reports/Case Series Actionable In a clinical case study, the combination of PF-07284892, Erbitux (cetuximab), and Braftovi (encorafenib) resulted in a 30% tumor reduction in a colorectal cancer patient harboring BRAF V600E, who remained on treatment for 6 months, and the combination inhibited tumor growth in a cell line xenograft model (PMID: 37269335; NCT04800822). 37269335
BRAF V600E colorectal cancer predicted - sensitive Cetuximab + Encorafenib + ERAS-007 Case Reports/Case Series Actionable In a Phase Ib/II trial (HERKULES), combination treatment with ERAS-007, Erbitux (cetuximab), and Braftovi (encorafenib) demonstrated safety and activity in patients with metastatic colorectal cancer harboring BRAF V600E, resulting in 1 confirmed and 1 unconfirmed partial response of 4 efficacy evaluable patients (J Clin Oncol 41, 2023 (suppl 16; abstr 3557); NCT05039177). detail...
BRAF V600E colon adenocarcinoma predicted - sensitive Cetuximab + Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, the combination of Tafinlar (dabrafenib), Mekinist (trametinib), and Erbitux (cetuximab) resulted in a partial response after 2 months followed by a complete response in a patient with microsatellite-stable, metastatic colon adenocarcinoma harboring BRAF V600E, and the patient remained in complete remission 11 months after discontinuation of treatment (PMID: 37213293). 37213293
BRAF V600E Advanced Solid Tumor sensitive IHMT-RAF-128 Preclinical - Cell culture Actionable In a preclinical study, IHMT-RAF-128 inhibited proliferation of a cell line expressing BRAF V600E in culture (PMID: 37164118). 37164118
BRAF V600E melanoma sensitive IHMT-RAF-128 Preclinical - Cell line xenograft Actionable In a preclinical study, IHMT-RAF-128 inhibited proliferation and induced cell cycle arrest and apoptosis in melanoma cell lines harboring BRAF V600E in culture and inhibited tumor growth in a cell line xenograft model (PMID: 37164118). 37164118
BRAF V600E colon cancer sensitive IHMT-RAF-128 Preclinical - Cell culture Actionable In a preclinical study, IHMT-RAF-128 inhibited proliferation in a colon cancer cell line harboring BRAF V600E in culture (PMID: 37164118). 37164118
BRAF V600E thyroid cancer sensitive SHP099 + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Zelboraf (vemurafenib) and SHP099 inhibited proliferation and induced cell cycle arrest in Zelboraf (vemurafenib)-resistant thyroid cancer cell lines harboring BRAF V600E in culture and inhibited tumor growth in a cell line xenograft model (PMID: 37325052). 37325052
BRAF V600E colorectal cancer no benefit Cetuximab + Fluorouracil + Irinotecan + Leucovorin + Oxaliplatin Phase II Actionable In a Phase II trial (FIRE-4.5), the combination of Erbitux (cetuximab) with FOLFOXIRI as first-line therapy did not result in an improved objective response rate (ORR) compared to Avastin (bevacizumab) with FOLFOXIRI, with an ORR of 50.8% (30/59) vs 66.7% (20/30) (P=0.92), a median progression-free survival of 7.6 vs 12.4 months (P=0.003), and a median overall survival of 15.2 vs 22.9 months (P=0.14), respectively, in metastatic colorectal cancer patients harboring BRAF V600E (PMID: 37352476). 37352476
BRAF V600E colorectal cancer sensitive Binimetinib + Trifluridine-tipiracil hydrochloride Preclinical - Cell culture Actionable In a preclinical study, the addition of Mektovi (binimetinib) enhanced the efficacy of Lonsurf (trifluridine/tipiracil hydrochloride) treatment and synergistically inhibited proliferation in colorectal cancer cells harboring BRAF V600E in culture (PMID: 28551618). 28551618
BRAF V600E colorectal carcinoma no benefit Palbociclib + PD-0325901 Preclinical - Cell culture Actionable In a preclinical study, the combination of Ibrance (palbociclib) and PD-0325901 demonstrated antagonism in a colorectal carcinoma cell line harboring BRAF V600E in culture (PMID: 37326340). 37326340
BRAF V600E colorectal carcinoma sensitive Gedatolisib + Palbociclib Preclinical - Cell culture Actionable In a preclinical study, the combination of Ibrance (palbociclib) and Gedatolisib (PF-05212384) synergistically inhibited growth of a colorectal carcinoma cell line harboring BRAF V600E in culture (PMID: 37326340). 37326340
BRAF V600E colorectal cancer no benefit Cetuximab + Fluorouracil + Leucovorin + Vemurafenib Phase II Actionable In a Phase II trial (MODUL), patients with metastatic colorectal cancer harboring BRAF V600E did not demonstrate improved progression-free survival when treated with the combination of Zelboraf (vemurafenib), Erbitux (cetuximab), Adrucil (fluorouracil), and Wellcovorin (leucovorin) compared to the control treatment of Avastin (bevacizumab) with a fluoropyrimidine (HR=0.95 and P=0.872) (PMID: 36921494; NCT02291289). 36921494
BRAF V600E colorectal cancer sensitive SJ-C1044 Preclinical - Cell line xenograft Actionable In a preclinical study, SJ-C1044 decreased growth in a colorectal cancer cell line harboring BRAF V600E in culture and inhibited tumor growth in a cell line xenograft model (PMID: 37504287). 37504287
BRAF V600E synovial sarcoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) resulted in a partial response after 4 months of treatment in a patient with intrathoracic synovial sarcoma harboring SS18-SSX1 (e10:e6) and BRAF V600E (PMID: 37417899). 37417899
BRAF V600E melanoma decreased response Toripalimab-tpzi Clinical Study Actionable In a retrospective analysis, real-world treatment with either Keytruda (pembrolizumab) or Loqtorz (toripalimab-tpzi) resulted in a median disease-free survival of 17 months in melanoma patients harboring BRAF V600E compared to 32 months in patients with wild-type BRAF, NRAS, and KIT (p=0.022) (PMID: 37403699). 37403699
BRAF V600E colorectal cancer sensitive Cetuximab + Encorafenib + Fluorouracil + Leucovorin + Oxaliplatin Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of Erbitux (cetuximab), Braftovi (encorafenib), and FOLFOX resulted in increased tumor growth inhibition and survival compared to Erbitux (cetuximab) plus Braftovi (encorafenib) or FOLFOX alone in colorectal cancer cell line xenograft models harboring BRAF V600E (PMID: 37040395). 37040395
BRAF V600E colorectal cancer sensitive Cetuximab + Encorafenib + Fluorouracil + Irinotecan + Leucovorin Preclinical - Pdx Actionable In a preclinical study, treatment with the combination of Erbitux (cetuximab), Braftovi (encorafenib), and FOLFIRI resulted in increased tumor growth inhibition and tumor regression compared to Erbitux (cetuximab) plus Braftovi (encorafenib) or FOLFIRI alone in patient-derived xenograft (PDX) models of colorectal cancer harboring BRAF V600E and increased survival and tumor growth inhibition in cell line xenograft models (PMID: 37040395). 37040395
BRAF V600E Squamous Cell Carcinoma of Unknown Primary sensitive Dabrafenib + Trametinib Guideline Actionable The combination of Tafinlar (dabrafenib) and Mekinist (trametinib) is included in guidelines for patients with squamous cell carcinoma of unknown primary harboring BRAF V600E (NCCN.org). detail...
BRAF V600E Adenocarcinoma of Unknown Primary sensitive Dabrafenib + Trametinib Guideline Actionable The combination of Tafinlar (dabrafenib) and Mekinist (trametinib) is included in guidelines for patients with adenocarcinoma of unknown primary harboring BRAF V600E (NCCN.org). detail...
BRAF V600E appendix adenocarcinoma sensitive Encorafenib + Panitumumab Guideline Actionable Braftovi (encorafenib) in combination with Vectibix (panitumumab) is included in guidelines for patients with advanced or metastatic appendiceal adenocarcinoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E appendix adenocarcinoma sensitive Cetuximab + Encorafenib Guideline Actionable Braftovi (encorafenib) in combination with Erbitux (cetuximab) is included in guidelines for patients with advanced or metastatic appendiceal adenocarcinoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E low grade glioma predicted - sensitive FCN-159 Phase II Actionable In a Phase II trial, FCN-159 treatment was well tolerated and resulted in 6 partial responses, 9 minor responses, and 7 with stable disease in 22 pediatric patients with low-grade glioma harboring BRAF V600E (12/23), KIAA1549-BRAF (8/23), or NF1 mutations (3/23) (Ann Oncol (2023) 34 (suppl_2): S391-S392). detail...
BRAF V600E melanoma sensitive SIJ777 Preclinical - Cell culture Actionable In a preclinical study, SIJ777 inhibited Mek, Erk, and Akt phosphorylation, proliferation, migration, and invasion, and induced apoptosis in melanoma cell lines harboring BRAF V600E in culture (PMID: 33917428). 33917428
BRAF V600E ameloblastoma sensitive Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, Zelboraf (vemurafenib) inhibited viability of ameloblastoma cell lines harboring BRAF V600E in culture (PMID: 35689405). 35689405
BRAF V600E ameloblastoma sensitive Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, Zelboraf (vemurafenib) inhibited viability of an ameloblastoma cell line harboring BRAF V600E in culture (PMID: 24859340). 24859340
BRAF V600E pancreatic cancer predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a retrospective analysis, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a partial response in two patients with pancreatic cancer harboring BRAF V600E, including one patient with a progression-free survival (PFS) of at least 2 years on third-line therapy and another patient with a PFS of 48 weeks on second-line therapy (PMID: 34476331). 34476331
BRAF V600E melanoma sensitive Encorafenib + Talazoparib Preclinical - Patient cell culture Actionable In a preclinical study, treatment with the combination of Talzenna (talazoparib) and Braftovi (encorafenib) synergistically inhibited viability of patient-derived melanoma spheroids harboring BRAF V600E in culture (PMID: 37729428). 37729428
BRAF V600E melanoma sensitive Binimetinib + Encorafenib + Talazoparib Preclinical - Patient cell culture Actionable In a preclinical study, treatment with the combination of Talzenna (talazoparib), Braftovi (encorafenib), and Mektovi (binimetinib) synergistically inhibited viability of patient-derived melanoma spheroids harboring BRAF V600E in culture (PMID: 37729428). 37729428
BRAF V600E melanoma sensitive AZD3514 + Dabrafenib Preclinical - Cell culture Actionable In a preclinical study, the addition of AZD3514 to Tafinlar (dabrafenib) treatment inhibited colony formation in melanoma cell lines harboring BRAF V600E in culture (PMID: 37838724). 37838724
BRAF V600E melanoma sensitive ARCC4 + Dabrafenib Preclinical - Cell culture Actionable In a preclinical study, the addition of ARCC4 to Tafinlar (dabrafenib) treatment inhibited colony formation in melanoma cell lines harboring BRAF V600E in culture (PMID: 37838724). 37838724
BRAF V600E breast malignant phyllodes tumor predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (TAPUR), Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy resulted in a partial response in a patient with a phyllodes tumor of the breast harboring BRAF V600E (PMID: 38096472; NCT02693535). 38096472
BRAF V600E pancreatic endocrine carcinoma predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (TAPUR), Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy resulted in a partial response in a patient with pancreatic neuroendocrine carcinoma harboring BRAF V600E (PMID: 38096472; NCT02693535). 38096472
BRAF V600E pancreatic cancer predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (TAPUR), Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy resulted in a partial response in 2 patients with pancreatic cancer harboring BRAF V600E (PMID: 38096472; NCT02693535). 38096472
BRAF V600E clear cell sarcoma predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (TAPUR), Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy resulted in a partial response in a patient with clear cell sarcoma harboring BRAF V600E (PMID: 38096472; NCT02693535). 38096472
BRAF V600E colon neuroendocrine neoplasm predicted - sensitive Cobimetinib + Vemurafenib Case Reports/Case Series Actionable In a Phase II trial (TAPUR), Zelboraf (vemurafenib) and Cotellic (cobimetinib) combination therapy resulted in a partial response in a patient with neuroendocrine carcinoma of the colon harboring BRAF V600E (PMID: 38096472; NCT02693535). 38096472
BRAF V600E small intestine adenocarcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) in combination with Mekinist (trametinib) is included in guidelines for patients with advanced or metastatic small bowel adenocarcinoma harboring BRAF V600E (NCCN.org). detail...
BRAF V600E melanoma predicted - sensitive IMM-6-415 Preclinical - Cell line xenograft Actionable In a preclinical study, IMM-6-415 treatment inhibited tumor growth in a cell line xenograft model of melanoma harboring BRAF V600E (Mol Cancer Ther (2023) 22 (12_Supplement): A093). detail...
BRAF V600E colorectal cancer predicted - sensitive IMM-6-415 Preclinical - Cell line xenograft Actionable In a preclinical study, IMM-6-415 treatment inhibited tumor growth in a cell line xenograft model of colorectal cancer harboring BRAF V600E (Mol Cancer Ther (2023) 22 (12_Supplement): A093). detail...
BRAF V600E melanoma predicted - sensitive Encorafenib + IMM-6-415 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of Braftovi (encorafenib) and IMM-6-415 resulted in increased tumor growth inhibition compared to the combination of Mektovi (binimetinib) and Braftovi (encorafenib) in a cell line xenograft model of melanoma harboring BRAF V600E (Mol Cancer Ther (2023) 22 (12_Supplement): A093). detail...
BRAF V600E colorectal cancer predicted - sensitive Encorafenib + IMM-6-415 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of Braftovi (encorafenib) and IMM-6-415 resulted in increased tumor growth inhibition compared to the combination of Mektovi (binimetinib) and Braftovi (encorafenib) in a cell line xenograft model of colorectal cancer harboring BRAF V600E (Mol Cancer Ther (2023) 22 (12_Supplement): A093). detail...
BRAF V600E melanoma sensitive Tovorafenib Preclinical - Cell culture Actionable In a preclinical study, Ojemda (tovorafenib) treatment inhibited viability of a melanoma cell line harboring BRAF V600E in culture (PMID: 31618628). 31618628
BRAF V600E triple-receptor negative breast cancer predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in a partial response in the lung lesion lasting at least 19 months in a patient with metastatic triple-negative breast cancer harboring BRAF V600E (PMID: 33976643). 33976643
BRAF V600E triple-receptor negative breast cancer sensitive Dabrafenib Preclinical - Cell culture Actionable In a preclinical study, Tafinlar (dabrafenib) treatment inhibited Mapk signaling and viability in a triple-negative breast cancer cell line harboring BRAF V600E in culture (PMID: 36011019). 36011019
BRAF V600E triple-receptor negative breast cancer sensitive Trametinib Preclinical - Cell culture Actionable In a preclinical study, Mekinist (trametinib) treatment inhibited Mapk signaling and viability in a triple-negative breast cancer cell line harboring BRAF V600E in culture (PMID: 36011019). 36011019
BRAF V600E triple-receptor negative breast cancer sensitive Binimetinib Preclinical - Cell culture Actionable In a preclinical study, Mektovi (binimetinib) treatment inhibited Mapk signaling and viability in a triple-negative breast cancer cell line harboring BRAF V600E in culture (PMID: 36011019). 36011019
BRAF V600E breast metaplastic carcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a partial response in a patient with metaplastic breast cancer harboring BRAF V600E, however, progression occurred after 8 weeks (PMID: 32206360). 32206360
BRAF V600E skin melanoma sensitive Dabrafenib + Pembrolizumab + Trametinib Guideline Actionable Tafinlar (dabrafenib) plus Mekinist (trametinib) in combination with an immune checkpoint inhibitor, such as Keytruda (pembrolizumab), is included in guidelines as second-line or subsequent therapy (category 2A) for patients with metastatic or unresectable cutaneous melanoma harboring a BRAF V600 mutation, such as BRAF V600E (NCCN.org). detail...
BRAF V600E skin melanoma sensitive Atezolizumab + Cobimetinib + Vemurafenib Guideline Actionable Zelboraf (vemurafenib) plus Cotellic (cobimetinib) in combination with an immune checkpoint inhibitor, such as Tecentriq (atezolizumab), is included in guidelines as second-line or subsequent therapy (category 2A) for patients with metastatic or unresectable cutaneous melanoma harboring a BRAF V600 mutation, such as BRAF V600E (NCCN.org). detail...
BRAF V600E anaplastic thyroid carcinoma sensitive Binimetinib + Encorafenib Case Reports/Case Series Actionable In a Phase II trial, Mektovi (binimetinib) and Braftovi (encorafenib) combination therapy demonstrated safety and activity in patients with thyroid cancer harboring BRAF V600E, resulting in an objective response rate (ORR) of 54.5% (12/22, 12 partial responses (PR)), with an ORR of 80% (4/5, 4 PR) and a disease control rate of 100% (5/5) in patients with anaplastic thyroid cancer, and median duration of response, progression-free survival, and overall survival were not reached (PMID: 38343359). 38343359
BRAF V600E thyroid cancer sensitive Binimetinib + Encorafenib Phase II Actionable In a Phase II trial, treatment with Mektovi (binimetinib) plus Braftovi (encorafenib) demonstrated safety and activity in patients with BRAF V600E-mutant thyroid cancer, regardless of histological subtype, resulting in an objective response rate of 54.5% (12/22, 12 partial responses), disease control rate of 100% (22/22), and 12-month rate of ongoing response of 90.9%, and median duration of response, median progression-free survival, and median overall survival were not reached (PMID: 38343359). 38343359
BRAF V600E high grade glioma sensitive Binimetinib + Encorafenib Case Reports/Case Series Actionable In a Phase II trial, treatment with the combination of Mektovi (binimetinib) and Braftovi (encorafenib) demonstrated safety and activity in patients with high grade glioma harboring BRAF V600E, resulting in a radiographic response rate of 60% (3/5, 1 complete and 2 partial responses), with stable disease in 1 patient, a median duration of response of 10 months, a median progression-free survival of 9.4 months, and a median overall survival of 14.6 months (PMID: 38446982; NCT03973918). 38446982
BRAF V600E papillary thyroid carcinoma sensitive Binimetinib + Encorafenib Phase II Actionable In a Phase II trial, Mektovi (binimetinib) plus Braftovi (encorafenib) demonstrated safety in patients with BRAF V600E-mutant thyroid cancer and resulted in an objective response rate (ORR) of 54.5% (12/22, 12 partial responses (PR)), with an ORR of 47.1% (8/17, 8 PR) and a disease control rate of 100% (17/17) in the differentiated thyroid cancer cohort (all papillary thyroid cancer), and median duration of response, progression-free survival, and overall survival were not reached (PMID: 38343359). 38343359
BRAF V600E colorectal cancer sensitive Dabrafenib + IAG933 Preclinical - Cell culture Actionable In a preclinical study, the combination of IAG933 and Tafinlar (dabrafenib) inhibited proliferation of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 38565920). 38565920
BRAF V600E colorectal cancer sensitive Dabrafenib + IAG933 + LTT462 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of IAG933, Tafinlar (dabrafenib), and LTT462 inhibited proliferation of colorectal cancer cell lines harboring BRAF V600E in culture and resulted in greater tumor growth inhibition compared to either agent alone in a cell line xenograft model (PMID: 38565920). 38565920
BRAF V600E colorectal cancer sensitive Dabrafenib + IAG933 + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the combination of IAG933, Tafinlar (dabrafenib), and Mekinist (trametinib) inhibited proliferation of colorectal cancer cell lines harboring BRAF V600E in culture (PMID: 38565920). 38565920
BRAF V600E melanoma sensitive Tunlametinib + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of Tunlametinib (HL-085) and Zelboraf (vemurafenib) synergistically inhibited proliferation of a melanoma cell line harboring BRAF V600E in culture, and synergistically inhibited tumor growth in a cell line xenograft model (PMID: 37808191). 37808191
BRAF V600E colorectal cancer sensitive Tunlametinib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, treatment with the combination of Tunlametinib (HL-085) and Zelboraf (vemurafenib) synergistically inhibited proliferation of a colorectal cancer cell line harboring BRAF V600E in culture (PMID: 37808191). 37808191
BRAF V600E melanoma sensitive Tunlametinib Preclinical - Cell line xenograft Actionable In a preclinical study, Tunlametinib (HL-085) inhibited viability and induced cell cycle arrest in melanoma cell lines harboring BRAF V600E in culture, and inhibited tumor growth in a cell line xenograft model (PMID: 37808191). 37808191
BRAF V600E colorectal cancer sensitive Tunlametinib Preclinical - Cell line xenograft Actionable In a preclinical study, Tunlametinib (HL-085) inhibited viability of a colorectal cancer cell line harboring BRAF V600E in culture and inhibited tumor growth in a cell line xenograft model (PMID: 37808191). 37808191
BRAF V600E gallbladder cancer sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) in combination with Mekinist (trametinib) is included in guidelines as subsequent-line therapy for patients with biliary cancer harboring BRAF V600E, including gallbladder cancer (NCCN.org). detail...
BRAF V600E extrahepatic bile duct carcinoma sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) in combination with Mekinist (trametinib) is included in guidelines as subsequent-line therapy for patients with biliary cancer harboring BRAF V600E, including extrahepatic cholangiocarcinoma (NCCN.org). detail...
BRAF V600E colon neuroendocrine neoplasm predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) resulted in a clinical improvement and reduction of the primary tumor as well as the metastatic lesions in a cecal neuroendocrine carcinoma patient harboring BRAF V600E (PMID: 30036245). 30036245
BRAF V600E colon neuroendocrine neoplasm predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical study, second-line treatment with the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) resulted in a partial response in two patients with a neuroendocrine carcinoma of the colon harboring BRAF V600E, with progression-free survival of 6 months in one patient and 4 months in the second patient (PMID: 38716076). 38716076
BRAF V600E papillary thyroid carcinoma sensitive Ravoxertinib Preclinical - Cell culture Actionable In a preclinical study, Ravoxertinib (GDC-0994) inhibited viability and colony formation in papillary thyroid carcinoma cell lines harboring BRAF V600E in culture (PMID: 38728872). 38728872
BRAF V600E anaplastic thyroid carcinoma sensitive Ravoxertinib Preclinical - Cell line xenograft Actionable In a preclinical study, Ravoxertinib (GDC-0994) inhibited viability and colony formation and induced cell cycle arrest in an anaplastic thyroid carcinoma cell line harboring BRAF V600E in culture, and inhibited tumor growth in a cell line xenograft model (PMID: 38728872). 38728872
BRAF V600E melanoma sensitive Ravoxertinib Preclinical - Cell culture Actionable In a preclinical study, Ravoxertinib (GDC-0994) inhibited viability and colony formation in melanoma cell lines harboring BRAF V600E in culture (PMID: 38728872). 38728872
BRAF V600E melanoma sensitive Ravoxertinib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, treatment with the combination of Ravoxertinib (GDC-0994) and Zelboraf (vemurafenib) resulted in additive effects, with decreased growth of a melanoma cell line harboring BRAF V600E in culture (PMID: 38728872). 38728872
BRAF V600E lung adenocarcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a partial response ongoing for at least 73 months in an elderly patient with lung adenocarcinoma harboring BRAF V600E (PMID: 39376796). 39376796
BRAF V600E lung adenocarcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a partial response with a decrease in the size of the lung lesion in a patient with metastatic lung adenocarcinoma harboring BRAF V600E and germline BRCA L1908Rfs*2, who had previously progressed on several lines of therapy (PMID: 38715777). 38715777
BRAF V600E melanoma predicted - sensitive Binimetinib + PLX8394 Preclinical - Cell culture Actionable In a preclinical study, treatment with the combination of Mektovi (binimetinib) and PLX8394 inhibited viability of melanoma cells harboring BRAF V600E in culture (Cancer Res (2024) 84 (6_Supplement): 4609). detail...
BRAF V600E colorectal cancer predicted - sensitive Binimetinib + PLX8394 Preclinical - Cell culture Actionable In a preclinical study, treatment with the combination of Mektovi (binimetinib) and PLX8394 inhibited viability of colorectal cancer cells harboring BRAF V600E in culture (Cancer Res (2024) 84 (6_Supplement): 4609). detail...
BRAF V600E colorectal cancer predicted - sensitive PF-07799933 Preclinical - Biochemical Actionable In a preclinical study, PF-07799933 inhibited Erk phosphorylation in colorectal cancer cells harboring BRAF V600E in culture (PMID: 38691346). 38691346
BRAF V600E high grade glioma predicted - sensitive Binimetinib + PF-07799933 Case Reports/Case Series Actionable In a Phase I trial, the addition of Mektovi (binimetinib) to treatment with PF-07799933 resulted in a partial response in a patient with high grade glioma harboring BRAF V600E (PMID: 38691346; NCT05355701). 38691346
BRAF V600E melanoma predicted - sensitive Binimetinib + PF-07799933 Case Reports/Case Series Actionable In a Phase I trial, treatment with the combination of PF-07799933 and Mektovi (binimetinib) resulted in a partial response in 2 patients with melanoma harboring BRAF V600E and stable disease in 1 patient (PMID: 38691346; NCT05355701). 38691346
BRAF V600E pleomorphic xanthoastrocytoma predicted - sensitive PF-07799933 Case Reports/Case Series Actionable In a Phase I trial, PF-07799933 treatment resulted in a complete response in a patient with pleomorphic xanthoastrocytoma harboring BRAF V600E (PMID: 38691346; NCT05355701). 38691346
BRAF V600E skin melanoma predicted - sensitive Dabrafenib + Nivolumab + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Mekinist (trametinib), Tafinlar (dabrafenib), and Opdivo (nivolumab) resulted in a partial response in the leptomeningeal disease on the spine and the extracranial melanoma, with the intracranial lesions remaining stable after 6 months, in a patient with cutaneous melanoma harboring BRAF V600E, and a follow-up at least 7 years following diagnosis demonstrated complete remission (PMID: 38960393). 38960393
BRAF V600E malignant fibrous histiocytoma sensitive Binimetinib + Encorafenib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Mektovi (binimetinib) and Braftovi (encorafenib) resulted in a clinical response after two months in a patient with angiomatoid fibrous histiocytoma harboring BRAF V600E, along with EWSR1-CREB1 (e7:e7), and in a preclinical study, combination treatment with Mektovi (binimetinib) and Braftovi (encorafenib) inhibited viability of cells in culture (PMID: 38885476). 38885476
BRAF V600E acinar cell carcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in near complete metabolic remission at 5 months and persistent complete metabolic remission at 12 months in a patient with metastatic acinar cell carcinoma of unknown primary harboring BRAF V600E (PMID: 38820503). 38820503
BRAF V600E thyroid cancer sensitive PLX4720 + Sorafenib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of PLX4720 and Nexavar (sorafenib) inhibited proliferation of thyroid cancer cell lines harboring BRAF V600E in culture and resulted in greater tumor growth inhibition than PLX4720 alone in a cell line xenograft model (PMID: 38795180). 38795180
BRAF V600E thyroid cancer sensitive Lenvatinib + PLX4720 Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of PLX4720 and Lenvima (lenvatinib) inhibited proliferation of thyroid cancer cell lines harboring BRAF V600E in culture and resulted in greater tumor growth inhibition than PLX4720 alone in a cell line xenograft model (PMID: 38795180). 38795180
BRAF V600E pancreatic endocrine carcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a partial response lasting at least 15 months in a patient with unresectable, metastatic pancreatic neuroendocrine carcinoma harboring BRAF V600E (PMID: 38814411). 38814411
BRAF V600E melanoma sensitive DSR6434 + unspecified PD-1 antibody + Vemurafenib Preclinical Actionable In a preclinical study, treatment with the combination of Zelboraf (vemurafenib), DSR6434, and an anti-PD-1 antibody induced tumor regression and resulted in prolonged progression-free survival compared to either of the doublet combination therapies in a syngeneic mouse model of melanoma harboring BRAF V600E (PMID: 38894534). 38894534
BRAF V600E colon neuroendocrine neoplasm predicted - sensitive Cetuximab + Encorafenib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Erbitux (cetuximab) and Braftovi (encorafenib) resulted in reduced liver metastasis and a progression-free survival of 14 months in a patient with colorectal neuroendocrine carcinoma harboring BRAF V600E (PMID: 37981300). 37981300
BRAF V600E childhood pilocytic astrocytoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in a partial response in a pediatric patient with pilocytic astrocytoma harboring BRAF V600E (PMID: 38976815). 38976815
BRAF V600E melanoma no benefit RGT-018 Preclinical - Cell culture Actionable In a preclinical study, RGT-018 did not inhibit proliferation of melanoma cells harboring BRAF V600E in 3D culture (PMID: 39087485). 39087485
BRAF V600E histiocytosis predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical study, Zelboraf (vemurafenib) treatment resulted in complete remission in 2 pediatric patients with Langerhans cell histiocytosis harboring BRAF V600E (PMID: 39190425). 39190425
BRAF V600E colorectal cancer sensitive MTX-531 + Trametinib Preclinical - Pdx Actionable In a preclinical study, treatment with the combination of Mekinist (trametinib) and MTX-531 inhibited tumor growth with a 40% partial response rate and increased survival in a patient-derived xenograft (PDX) model of colorectal cancer harboring BRAF V600E (PMID: 38992135). 38992135
BRAF V600E renal Wilms' tumor predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical study, combination treatment with Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in regression in the metastatic disease of the liver, spleen, and peritoneum along with clinical symptom improvement in an adult patient with metastatic Wilms' tumor harboring BRAF V600E (PMID: 39234402). 39234402
BRAF V600E nephroblastoma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) treatment resulted in a durable major response with a decrease in the pulmonary metastases in an adult patient with metastatic nephroblastoma harboring BRAF V600E (PMID: 31109800). 31109800
BRAF V600E glioblastoma sensitive PD-0325901 + Radiotherapy Preclinical - Cell culture Actionable In a preclinical study, the combination of PD-0325901 and radiotherapy synergistically inhibited cell growth of glioblastoma cell lines harboring BRAF V600E in culture (PMID: 38714355). 38714355
BRAF V600E pseudomyxoma peritonei sensitive Encorafenib Preclinical - Pdx & cell culture Actionable In a preclinical study, Braftovi (encorafenib) inhibited viability and induced apoptosis in a patient-derived pseudomyxoma peritonei cell line harboring BRAF V600E in culture and inhibited tumor growth and improved survival of a patient-derived xenograft (PDX) model (PMID: 39018564). 39018564
BRAF V600E pseudomyxoma peritonei sensitive Cetuximab + Encorafenib Preclinical - Pdx & cell culture Actionable In a preclinical study, treatment with the combination of Braftovi (encorafenib) and Erbitux (cetuximab) inhibited viability and induced apoptosis in a patient-derived pseudomyxoma peritonei cell line harboring BRAF V600E in culture and inhibited tumor growth and improved survival of a patient-derived xenograft (PDX) model (PMID: 39018564). 39018564
BRAF V600E lung combined large cell neuroendocrine carcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a partial response after 10 weeks in a patient with combined large cell neuroendocrine carcinoma harboring BRAF V600E, who remained recurrence-free for at least a year (PMID: 34969785). 34969785
BRAF V600E large cell neuroendocrine carcinoma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Mekinist (trametinib) and Tafinlar (dabrafenib) resulted in a partial response in one of the lesions and a complete response in other lesions after 1 month in a patient with large cell neuroendocrine carcinoma of unknown primary harboring BRAF V600E (PMID: 36847048). 36847048
BRAF V600E craniopharyngioma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in a partial response and progression-free survival of 1040 days in a patient with craniopharyngioma harboring BRAF V600E (PMID: 39143272). 39143272
BRAF V600E Advanced Solid Tumor predicted - sensitive Binimetinib + Encorafenib Phase II Actionable In a Phase II trial (BELIEVE), treatment with the combination of Braftovi (encorafenib) and Mektovi (binimetinib) resulted in an objective response rate of 32.7%, median progression-free survival (PFS) of 4.8 months, and 6-month PFS rate of 37.5% in patients with advanced solid tumors harboring BRAF V600E (n=43), other BRAF mutation (n=5), or a BRAF fusion (n=1) (Ann Oncol (2024) 35 (Suppl_2): S497). detail...
BRAF V600E melanoma predicted - sensitive CFT1946 Phase I Actionable In a Phase I trial, CFT1946 treatment demonstrated safety and preliminary activity in patients with advanced solid tumors (n=25) including melanoma (40%), colorectal cancer (40%), non-small cell lung cancer (8%), and other non-CNS tumors harboring BRAF V600E (n=22), V600K (n=2), or V600R (n=1), with 1 unconfirmed partial response (uPR) and 7 stable diseases in efficacy evaluable patients (n=14) at data cutoff, and 1 additional uPR after data cutoff (Ann Oncol (2024) 35 (Suppl_2): S490-S491; NCT05668585). detail...
BRAF V600E glomus tumor predicted - sensitive Binimetinib + Encorafenib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Mektovi (binimetinib) and Braftovi (encorafenib) resulted in a complete response lasting more than 2 years in a patient with metastatic malignant glomus tumor harboring BRAF V600E (PMID: 39392364). 39392364
BRAF V600E glomus tumor predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in decreased tumor size lasting at least 9 months in an adolescent (18 years old) patient with malignant glomus tumor harboring BRAF V600E (PMID: 30556047). 30556047
BRAF V600E Cancer of Unknown Primary predicted - sensitive Binimetinib + Cetuximab Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Mektovi (binimetinib) and Erbitux (cetuximab) resulted in a significant but brief response in all lesions in a patient with metastatic cancer of unknown primary harboring BRAF V600E (PMID: 39391046). 39391046
BRAF V600E desmoplastic infantile ganglioglioma / desmoplastic infantile astrocytoma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) treatment resulted in a partial response with resolution of ascites in a pediatric patient with suprasellar metastatic desmoplastic infantile astrocytoma harboring BRAF V600E (PMID: 39399174). 39399174
BRAF V600E diffuse leptomeningeal glioneuronal tumor predicted - sensitive Trametinib Case Reports/Case Series Actionable In a clinical case study, Mekinist (trametinib) treatment resulted in a partial response in the brain and stable disease in the spine in a pediatric patient with diffuse leptomeningeal glioneuronal tumor harboring BRAF V600E (PMID: 39399174). 39399174
BRAF V600E pleomorphic xanthoastrocytoma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) treatment resulted in 1 partial response and 1 stable disease in 2 patients with tempero-parietal pleomorphic xanthoastrocytoma harboring BRAF V600E and CDKN2A deletion (PMID: 39399174). 39399174
BRAF V600E anaplastic pleomorphic xanthoastrocytoma predicted - sensitive Dabrafenib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) treatment resulted in a partial response in a patient with tempero-parietal anaplastic pleomorphic xanthoastrocytoma harboring BRAF V600E (PMID: 39399174). 39399174
BRAF V600E pancreatic ductal adenocarcinoma predicted - sensitive Binimetinib + Cetuximab + Encorafenib Case Reports/Case Series Actionable In a clinical case study, third-line treatment with the combination of Mektovi (binimetinib), Erbitux (cetuximab), and Braftovi (encorafenib) resulted in a partial response with a 74% decrease in primary tumor size in a patient with metastatic pancreatic ductal adenocarcinoma harboring BRAF V600E (PMID: 39461261). 39461261
BRAF V600E papillary thyroid carcinoma sensitive AZD1208 + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, treatment with the combination of Zelboraf (vemurafenib) and AZD1208 synergistically inhibited viability and colony formation of papillary thyroid carcinoma cell lines harboring BRAF V600E in culture (PMID: 38593698). 38593698
BRAF V600E colorectal cancer sensitive Tazemetostat + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the addition of Tazverik (tazemetostat) sensitized a colorectal cancer cell line harboring BRAF V600E to Mekinist (trametinib) treatment in culture, resulting in decreased cell proliferation (PMID: 39121480). 39121480
BRAF V600E colorectal cancer sensitive Encorafenib + Tazemetostat Preclinical - Cell culture Actionable In a preclinical study, the addition of Tazverik (tazemetostat) sensitized a colorectal cancer cell line harboring BRAF V600E to Braftovi (encorafenib) treatment in culture, resulting in decreased cell proliferation (PMID: 39121480). 39121480
BRAF V600E colorectal cancer sensitive Cetuximab + Encorafenib + Tazemetostat Preclinical - Cell culture Actionable In a preclinical study, the addition of Tazverik (tazemetostat) sensitized a colorectal cancer cell line harboring BRAF V600E to treatment with the combination of Braftovi (encorafenib) and Erbitux (cetuximab) in culture, resulting in decreased cell proliferation (PMID: 39121480). 39121480