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Gene Symbol PTEN
Synonyms 10q23del | BZS | CWS1 | DEC | GLM2 | MHAM | MMAC1 | PTEN1 | PTENbeta | PTENgama | TEP1
Gene Description PTEN, phosphatidylinositol 3,4,5-trisphosphate 3-phosphatase and dual-specificity protein phosphatase PTEN, is a tumor suppressor (PMID: 30562755) with roles in the cell cycle, growth, DNA repair, cell survival and regulation of the Akt-mTOR pathway (PMID: 24656806, PMID: 30145641). PTEN germline mutations are common in Cowden syndrome (PMID: 30562755) and PTEN somatic alterations resulting in loss of function have been found in many types of cancer including, but not limited to endometrial (PMID: 30142194), melanoma (PMID: 30148988), and prostate (PMID: 18767981, PMID: 30153654).
ACMG Incidental List v3.0:
Yes, PTEN hamartoma tumor syndrome (PMID: 34012068)

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
PTEN del lung non-small cell carcinoma predicted - sensitive PF-04691502 Preclinical - Cell line xenograft Actionable In a preclinical study, PF-04691502 inhibited tumor growth in PTEN-deleted non-small cell lung cancer cell line xenograft models (PMID: 21750219). 21750219
PTEN loss lung non-small cell carcinoma sensitive Pictilisib Preclinical - Cell line xenograft Actionable In preclinical studies, the PI3K inhibitor GDC-0941 demonstrated efficacy against NSCLC tumor cell lines in both culture and xenograft models harboring alterations in the PI3K pathway (PMID: 23136191). 23136191
PTEN wild-type glioblastoma sensitive 2-Methoxyestradiol Preclinical Actionable In a preclinical study, 2-Methoxyestradiol (2ME2) inhibited tumor-induced angiogenesis and reduced tumor growth in PTEN reconstituted GBM cell lines and mouse models (PMID: 24162827). 24162827
PTEN loss glioblastoma sensitive CCT128930 Preclinical - Cell line xenograft Actionable In preclinical studies, CCT128930 prevented tumor growth in a PTEN-null human glioblastoma cell line xenograft model (PMID: 21191045). 21191045
PTEN negative Advanced Solid Tumor sensitive Talazoparib Preclinical - Cell culture Actionable In a preclinical study, PTEN-deficient cancer cell lines with DNA repair deficiency demonstrated high sensitivity to the PARP inhibitor Talzenna (talazoparib) (PMID: 23881923). 23881923
BRAF V600E PTEN loss melanoma predicted - resistant Everolimus Case Reports/Case Series Actionable In a retrospective analysis, a melanoma patient harboring PTEN loss and BRAF V600E demonstrated resistance to Afinitor (everolimus) treatment, resulting in progressive disease (PMID: 20664172). 20664172
PTEN loss Advanced Solid Tumor sensitive BAY1125976 Preclinical Actionable In a preclinical study, BAY1125976 demonstrated anti-tumor efficacy in multiple xenograft tumor models of different cancers with PIK3CA mutations or PTEN deletions and displayed synergy with other anti-cancer therapies (Cancer Res 2013;73(8 Suppl):Abstract nr 2050). detail...
PTEN mutant breast cancer sensitive Sapanisertib Preclinical Actionable In a preclinical study, Sapanisertib (MLN0128) induced apoptosis and inhibited MTORC1 signaling in breast cancer cells harboring a PTEN mutation (PMID: 25261369). 25261369
PTEN mut TP53 mut skin cancer sensitive Sapanisertib Preclinical Actionable In a preclinical study, a skin cancer cell line harboring mutations in PTEN and TP53 demonstrated sensitivity to Sapanisertib (MLN0128) in culture (PMID: 25261369). 25261369
PIK3CA R88Q PTEN mut endometrial cancer sensitive Dactolisib Preclinical Actionable In a preclinical study, BEZ235 inhibited proliferation of endometrial cancer cells harboring PIK3CA R88Q and PTEN mutations in culture (PMID: 22662154). 22662154
PIK3CA R88Q PTEN mut endometrial cancer sensitive Everolimus Preclinical Actionable In a preclinical study, Afinitor (everolimus) inhibited proliferation of endometrial cancer cells harboring PIK3CA R88Q and PTEN mutations in culture (PMID: 22662154). 22662154
PIK3CA R108H PTEN mut endometrial cancer sensitive Dactolisib Preclinical Actionable In a preclinical study, BEZ235 inhibited proliferation of endometrial cancer cells harboring PIK3CA R108H and PTEN mutations in culture (PMID: 22662154). 22662154
PIK3CA R38C PTEN mut endometrial cancer sensitive Dactolisib Preclinical Actionable In a preclinical study, BEZ235 decreased growth of endometrial cancer cells harboring PIK3CA R38C and PTEN mutations in culture and in xenograft models (PMID: 22662154). 22662154
PIK3CA E365K PTEN mut endometrial cancer sensitive Dactolisib Preclinical Actionable In a preclinical study, BEZ235 inhibited proliferation of endometrial cancer cells harboring PIK3CA E365K and PTEN mutations in culture (PMID: 22662154). 22662154
PTEN mutant endometrial cancer sensitive Dactolisib Preclinical Actionable In a preclinical study, BEZ235 decreased growth of endometrial cancer cells harboring a PTEN nonsense mutation in culture and in xenograft models (PMID: 22662154). 22662154
PTEN mutant endometrial cancer sensitive Everolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Afinitor (everolimus) inhibited growth of endometrial cancer cells harboring a PTEN nonsense mutation in culture and in xenograft models (PMID: 22662154). 22662154
PIK3CA E365K PTEN mut endocervical carcinoma sensitive Dactolisib Preclinical Actionable In a preclinical study, BEZ235 inhibited proliferation of endometrial cancer cells harboring PIK3CA E365K and PTEN mutations in culture (PMID: 22662154). 22662154
PTEN del prostate cancer sensitive VS-5584 Preclinical - Cell line xenograft Actionable In a preclinical study, VS-5584 inhibited PI3K/mTOR signaling and cell proliferation in a human prostate cancer cell line harboring a PTEN deletion in culture, and inhibited PI3K/MTOR signaling and tumor growth in xenograft models (PMID: 23270925). 23270925
PTEN loss glioblastoma sensitive PF-04691502 Preclinical - Cell line xenograft Actionable In a preclinical study, PF-04691502 inhibited Akt phosphorylation, resulted in growth inhibition of PTEN-null glioblastoma cells in culture and in cell line xenograft models (PMID: 21750219). 21750219
PTEN del prostate cancer sensitive Rucaparib Preclinical - Cell culture Actionable In a preclinical study, Rubraca (rucaparib) induced senescence and increased radiosensitivity in PTEN null prostate cancer cells in culture (PMID: 23565244). 23565244
PIK3CA R38C PTEN loss endometrial cancer sensitive MEN1611 Preclinical Actionable In a preclinical study, MEN1611 (CH5132799) inhibited proliferation of an endometrial cancer cell line harboring PIK3CA R38C and PTEN loss in culture (PMID: 21558396). 21558396
PTEN del stomach cancer sensitive MEN1611 Preclinical - Cell line xenograft Actionable In a preclinical study, MEN1611 (CH5132799) induced tumor regression in xenograft models of a human stomach cancer cell line with deletion of PTEN (PMID: 21558396). 21558396
PTEN del prostate cancer sensitive MEN1611 Preclinical - Cell line xenograft Actionable In a preclinical study, MEN1611 (CH5132799) inhibited tumor growth in xenograft models of a human prostate cancer cell line with deletion of PTEN (PMID: 21558396). 21558396
PTEN del prostate cancer sensitive Pictilisib + Vorinostat Preclinical Actionable In a preclinical study, GDC-0941 and Zolinza (vorinostat) acted synergistically to inhibit growth of a human prostate cancer cell line harboring a PTEN deletion in culture (PMID: 9661880, PMID: 22693356). 22693356 9661880
PTEN A72fs breast cancer sensitive Gedatolisib Preclinical Actionable In a preclinical study, Gedatolisib (PKI-587) inhibited growth of human breast cancer cells harboring PTEN A72fs*5 in culture (PMID: 21325073, PMID: 17314276). 17314276 21325073
PTEN del prostate cancer sensitive Gedatolisib Preclinical Actionable In a preclinical study, Gedatolisib (PKI-587) inhibited growth of human prostate cancer cells harboring PTEN deletion in culture (PMID: 21325073, PMID: 14737113). 14737113 21325073
PTEN loss brain glioma sensitive Gedatolisib Preclinical Actionable In a preclinical study, Gedatolisib (PKI-587) inhibited growth of human glioma cells with PTEN loss in culture (PMID: 21325073). 21325073
PTEN loss renal carcinoma decreased response Gedatolisib Preclinical Actionable In a preclinical study, human renal carcinoma cells with PTEN loss had a decreased response to Gedatolisib (PKI-587) in culture (PMID: 21325073). 21325073
PTEN loss VHL loss renal carcinoma decreased response Gedatolisib Preclinical Actionable In a preclinical study, human renal carcinoma cells with PTEN loss and VHL loss had a decreased response to Gedatolisib (PKI-587) in culture (PMID: 21325073). 21325073
PTEN negative glioblastoma no benefit Temozolomide + Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetinib), in combination with radiation therapy and Temodar (temozolomide), demonstrated no difference in PFS and OS when compared between glioblastoma patients negative for PTEN versus those positive for PTEN (PMID: 25910950). 25910950
PTEN del Advanced Solid Tumor no benefit GSK2636771 Phase I Actionable In a Phase I trial, patients with advanced solid tumors deficient in PTEN lacked benefit from GSK2636771 (PMID: 26117819). 26117819
PTEN loss prostate cancer sensitive AZD8186 Preclinical Actionable In a preclinical study, AZD8186 inhibited tumor growth of PTEN deficient prostate xenografts models (PMID: 25514658). 25514658
PTEN del prostate cancer predicted - sensitive AZD8186 Phase I Actionable In a Phase I trial, AZD8186 demonstrated preliminary efficacy in patients with tumor types with prevalent PTEN-deficiency, including prostate cancer (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr CT329). detail...
PTEN loss triple-receptor negative breast cancer predicted - sensitive AZD8186 Phase I Actionable In a Phase I trial, AZD8186 demonstrated preliminary efficacy in patients with tumor types with prevalent PTEN-deficiency, including triple negative breast cancer (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr CT329). detail...
PTEN loss lung squamous cell carcinoma predicted - sensitive AZD8186 Phase I Actionable In a Phase I trial, AZD8186 demonstrated preliminary efficacy in patients with tumor types with prevalent PTEN-deficiency, including squamous non-small cell lung cancer (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr CT329). detail...
PTEN loss prostate cancer sensitive Panulisib Preclinical - Cell line xenograft Actionable In a preclinical study, Panulisib (P7170) inhibited tumor growth in PTEN null prostate cancer cell xenograft models (PMID: 25700704). 25700704
PTEN loss prostate cancer no benefit MK2206 Phase I Actionable In a Phase I clinical trial, 8 patients with metastatic, castration-resistant prostate cancer harboring a loss of PTEN did not respond to MK-2206 therapy, but prolonged stable disease was observed in 2 patients (PMID: 26187616). 26187616
PTEN mutant head and neck squamous cell carcinoma resistant Taselisib Preclinical - Cell line xenograft Actionable In a preclinical study, head and neck squamous cell carcinoma cell lines and cell line xenograft models with PTEN alterations were resistant to the apoptotic effects of Taselisib (GDC-0032) (PMID: 26589432). 26589432
PTEN wild-type melanoma sensitive E6201 Preclinical Actionable In a preclinical study, E6201 inhibited proliferation of several melanoma cell lines in culture and sensitivity was associated with wild-type PTEN (PMID: 23039341). 23039341
PTEN dec exp melanoma decreased response Pembrolizumab Clinical Study - Cohort Actionable In a clinical study, melanoma patients with PTEN expression in less than 10% of tumor cells demonstrated decreased response to anti-PD-1 antibodies, including Keytruda (pembrolizumab), compared to patients in which PTEN is present in over 10% of tumor cells (PMID: 26645196). 26645196
PTEN dec exp melanoma decreased response Nivolumab Clinical Study - Cohort Actionable In a clinical study, melanoma patients with PTEN expression in less than 10% of tumor cells demonstrated decreased response to anti-PD-1 antibodies, including Opdivo (nivolumab), as compared to patients in which PTEN is present in over 10% of tumor cells (PMID: 26645196). 26645196
PTEN positive melanoma sensitive Pembrolizumab Clinical Study - Cohort Actionable In a clinical study, melanoma patients with PTEN positive tumors demonstrated a decrease in tumor size when treated with Keytruda (pembrolizumab) (PMID: 26645196). 26645196
PTEN positive melanoma sensitive Nivolumab Clinical Study - Cohort Actionable In a clinical study, melanoma patients with PTEN positive tumors demonstrated a decrease in tumor size when treated with Opdivo (nivolumab) (PMID: 26645196). 26645196
PTEN loss melanoma sensitive GSK2636771 Preclinical Actionable In a preclinical study, human melanoma cells with PTEN loss were sensitive to GSK2636771, resulting in decreased activation of Akt and some inhibition of tumor growth (PMID: 26645196). 26645196
BRAF mut PTEN loss melanoma sensitive GSK2636771 + Pembrolizumab Preclinical Actionable In a preclinical study, a melanoma mouse model harboring a BRAF mutation and PTEN loss was sensitive to the combination of GSK2636771 and Keytruda (pembrolizumab), demonstrating greater tumor growth inhibition and improved survival when compared to either therapy alone (PMID: 26645196). 26645196
BRAF mut PTEN loss melanoma no benefit GSK2636771 Preclinical Actionable In a preclinical study, GSK2636771 resulted in no benefit in a melanoma mouse model co-harboring a BRAF mutation and PTEN loss (PMID: 26645196). 26645196
BRAF mut PTEN loss melanoma no benefit Pembrolizumab Preclinical Actionable In a preclinical study, Keytruda (pembrolizumab) resulted in no benefit in a melanoma mouse model co-harboring a BRAF mutation and PTEN loss (PMID: 26645196). 26645196
PTEN del Advanced Solid Tumor sensitive Onatasertib Phase I Actionable In a Phase I trial, CC-223 demonstrated safety and preliminary efficacy in patients with solid tumors, including stable disease for greater than 110 days in 2 patients with PIK3CA mutation or PTEN deletion (PMID: 26177599). 26177599
PTEN mutant uterine cancer sensitive GSK2256098 Preclinical Actionable In a preclinical study, uterine cancer cells harboring a PTEN mutation demonstrated sensitivity to GSK2256098, resulting in inhibition of Ptk2 (Fak) phosphorylation, decreased tumor growth, and apoptosis both in culture and in mouse models (PMID: 25833835). 25833835
PTEN wild-type uterine cancer decreased response GSK2256098 Preclinical Actionable In a preclinical study, GSK2256098 treatment resulted in a decreased response in uterine cancer cells with PTEN wild-type versus uterine cancer cells harboring a PTEN mutation (PMID: 25833835). 25833835
PTEN loss prostate cancer sensitive OP449 Preclinical Actionable In a preclinical study, prostate cancer mouse models deficient for Pten demonstrated inhibition of the PI3K/Akt signaling pathway and a decrease in both tumor size and cell proliferation when treated with OP449 (PMID: 26563471). 26563471
PTEN loss colon cancer sensitive KU-55933 Preclinical Actionable In a preclinical study, KU-55933 induced cell cycle arrest and caspase activation in PTEN deficient human colon cancer cells in culture (PMID: 25870146). 25870146
PTEN loss prostate cancer sensitive KU-60019 Preclinical Actionable In a preclinical study, KU-60019 blocked tumor growth in PTEN-deficient human prostate cancer xenograft models (PMID: 25870146). 25870146
PTEN loss prostate cancer sensitive AT13148 Preclinical - Cell line xenograft Actionable In a preclinical study, AT13148 inhibited tumor growth in a PTEN-deficient human prostate cancer cell line xenograft model (PMID: 22781553). 22781553
PTEN loss uterine corpus sarcoma sensitive AT13148 Preclinical - Cell line xenograft Actionable In a preclinical study, AT13148 inhibited tumor growth in a PTEN-deficient human uterine sarcoma cell line xenograft model (PMID: 22781553). 22781553
PTEN A126G prostate cancer sensitive Alpelisib Preclinical - Cell culture Actionable In a preclinical study, Alpelisib (BYL719) inhibited Akt signaling and cell migration in prostate cancer cells expressing PTEN A126G in culture (PMID: 26504226). 26504226
PTEN A126G prostate cancer sensitive AZD6482 Preclinical Actionable In a preclinical study, AZD6482 inhibited Akt signaling and cell migration in prostate cancer cell lines overexpressing PTEN A126G (PMID: 26504226). 26504226
PTEN dec exp melanoma sensitive SAR260301 Preclinical - Cell line xenograft Actionable In a preclinical study, SAR260301 inhibited tumor growth in xenograft models of melanoma cell lines harboring PTEN deficiency (PMID: 24387221). 24387221
PTEN dec exp Advanced Solid Tumor sensitive GSK2636771 Phase Ib/II Actionable In a Phase I/II trial, GSK2636771 treatment inhibited Akt signaling, and resulted in partial response in 2% (1/53) and stable disease in 25% (13/53) of patients with PTEN-deficient advanced solid tumors (J Clin Oncol 32:5s, 2014 (suppl; abstr 2514)). detail...
PTEN mutant endometrial cancer no benefit Temsirolimus Phase II Actionable In a retrospective study of a Phase II trial, mutation status of PTEN was not associated with progression-free survival or response rate in advanced endometrial cancer patients treated with Torisel (temsirolimus) (PMID: 27016228). 27016228
PTEN mutant endometrial cancer resistant GSK2636771 Preclinical Actionable In a preclinical study, endometrioid endometrial cancer cell lines harboring PTEN mutations demonstrated resistance to GSK2636771 induced growth inhibition in culture (PMID: 23674493). 23674493
PTEN mutant endometrial cancer resistant AZD6482 Preclinical Actionable In a preclinical study, endometrioid endometrial cancer cell lines harboring PTEN mutations demonstrated resistance to AZD6482 induced growth inhibition in culture (PMID: 23674493). 23674493
PTEN mutant endometrial cancer resistant TGX-221 Preclinical Actionable In a preclinical study, endometrioid endometrial cancer cell lines harboring PTEN mutations demonstrated resistance to TGX-221 induced growth inhibition in culture (PMID: 23674493). 23674493
PTEN mutant endometrial cancer resistant A66 Preclinical Actionable In a preclinical study, endometrioid endometrial cancer cell lines harboring PTEN mutations demonstrated resistance to A66 induced growth inhibition in culture (PMID: 23674493). 23674493
PTEN loss prostate cancer sensitive AZD6482 Preclinical Actionable In a preclinical study, AZD6482 inhibited viability of prostate cancer cell lines harboring PTEN deficiency in culture (PMID: 23674493). 23674493
PTEN loss breast cancer sensitive AZD6482 Preclinical Actionable In a preclinical study, AZD6482 inhibited viability of breast cancer cell lines harboring PTEN deficiency in culture (PMID: 23674493). 23674493
PTEN loss prostate cancer sensitive GSK2636771 Preclinical Actionable In a preclinical study, GSK2636771 inhibited viability of prostate cancer cell lines harboring PTEN deficiency in culture (PMID: 23674493). 23674493
PTEN loss breast cancer sensitive GSK2636771 Preclinical Actionable In a preclinical study, GSK2636771 inhibited viability of breast cancer cell lines harboring PTEN deficiency in culture (PMID: 23674493). 23674493
PTEN mutant endometrial cancer sensitive A66 + AZD6482 Preclinical Actionable In a preclinical study, A66 and AZD6482 combination treatment inhibited viability of endometrioid endometrial cancer cell lines harboring PTEN mutations in culture (PMID: 23674493). 23674493
PTEN mutant endometrial cancer sensitive A66 + GSK2636771 Preclinical Actionable In a preclinical study, A66 and GSK2636771 combination treatment inhibited viability of endometrioid endometrial cancer cell lines harboring PTEN mutations in culture (PMID: 23674493). 23674493
PIK3CA mut PTEN loss breast cancer resistant Alpelisib Case Reports/Case Series Actionable In a clinical case study, a breast cancer patient harboring a PIK3CA mutation developed resistance to Alpelisib (BYL719) treatment and progressive disease after initial response, accompanied by a loss of PTEN, and the corrresponding patient-derived xenograft model demonstrated resistance to Alpelisib (BYL719)-induced inhibition of tumor growth (PMID: 25409150). detail... 25409150
PIK3CA mut PTEN loss breast cancer sensitive Buparlisib Preclinical - Pdx Actionable In a preclinical study, Buparlisib (BKM120) inhibited Akt signaling and growth in a PDX model of breast cancer cells harboring a PIK3CA mutation and PTEN loss ( Cancer Res October 1, 2014 74; LB-327 ). detail...
PIK3CA mut PTEN loss breast cancer sensitive Alpelisib + AZD6482 Preclinical - Pdx Actionable In a preclinical study, AZD6482 and Alpelisib (BYL719) combination treatment inhibited Akt signaling and growth in a breast cancer patient-derived xenograft (PDX) model harboring a PIK3CA mutation and PTEN loss (Cancer Res October 1, 2014 74; LB-327). detail...
PTEN loss triple-receptor negative breast cancer sensitive AZD6482 + Talazoparib Preclinical Actionable In a preclinical study, the combination of Talazoparib (BMN-673) and AZD6482 inhibited growth of triple-negative breast cancer cell lines with PTEN loss in culture (Mol Cancer Res, Feb 2016 14; B12). detail...
PTEN loss triple-receptor negative breast cancer sensitive AZD6482 + Niraparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Zejula (niraparib) and AZD6482 inhibited growth of triple-negative breast cancer cell lines with PTEN loss in culture (Mol Cancer Res Feb 2016 14; B12). detail...
PTEN loss triple-receptor negative breast cancer sensitive AZD6482 + Olaparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Lynparza (olaparib) and AZD6482 inhibited growth of triple-negative breast cancer cell lines with PTEN loss in culture (Mol Cancer Res Feb 2016 14; B12). detail...
PTEN loss triple-receptor negative breast cancer sensitive AZD6482 + Rucaparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Rubraca (rucaparib) and AZD6482 inhibited growth of triple-negative breast cancer cell lines with PTEN loss in culture (Mol Cancer Res Feb 2016 14; B12). detail...
PTEN loss triple-receptor negative breast cancer sensitive AZD6482 + Veliparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Veliparib (ABT-888) and AZD6482 inhibited growth of triple-negative breast cancer cell lines with PTEN loss in culture (Mol Cancer Res Feb 2016 14; B12). detail...
PTEN loss Advanced Solid Tumor no benefit SAR260301 Phase I Actionable In a Phase I trial, SAR260301 demonstrated acceptable safety but undesirable pharmacokinetics, and resulted in no response in patients with advanced solid tumors harboring PTEN loss (J Clin Oncol 33, 2015 (suppl; abstr 2564)). detail...
PTEN del prostate cancer sensitive Alpelisib + AZD8186 Preclinical Actionable In a preclinical study, AZD8186 and Alpelisib (BYL719) combination treatment resulted in minor inhibition of tumor growth in animal models of prostate cancer harboring PTEN gene deletion (PMID: 25544636). 25544636
PTEN del prostate cancer sensitive Alpelisib + AZD8186 + Enzalutamide Preclinical Actionable In a preclinical study, AZD8186, Alpelisib (BYL719), and Xtandi (enzalutamide) combination treatment resulted in near-complete suppression of tumor growth in animal models of prostate cancer harboring PTEN gene deletion (PMID: 25544636). 25544636
PTEN del prostate cancer sensitive AZD8186 + Enzalutamide Preclinical Actionable In a preclinical study, AZD8186 and Xtandi (enzalutamide) combination treatment resulted in suppression of tumor growth in animal models of prostate cancer harboring PTEN gene deletion (PMID: 25544636). 25544636
PIK3CA wild-type PTEN loss breast cancer resistant AZD8835 Preclinical Actionable In a preclinical study, human breast cancer cells with wild-type PIK3CA and loss of PTEN were resistant to growth inhibition by AZD8835 in culture (PMID: 26839307). 26839307
PTEN del urinary bladder cancer sensitive Metformin Preclinical Actionable In a preclinical study, bladder cancer cells with PTEN deletion were sensitive to Glucophage (metformin) in culture, resulting in inhibition of cell growth (PMID: 26921394). 26921394
PTEN mutant endometrial cancer sensitive Apitolisib Phase II Actionable In a Phase II trial, Apitolisib (GDC-0980) was poorly tolerated, but demonstrated efficacy in endometrial cancer patients harboring mutations in PIK3CA, PTEN, or AKT1 (J Clin Oncol 32:5s, 2014 (suppl; abstr 5513)). detail...
PTEN loss glioblastoma sensitive PKI-402 Preclinical - Cell line xenograft Actionable In a preclinical study, PKI-402 inhibited tumor growth in PTEN-negative glioblastoma multiforme cell line xenograft models (PMID: 20371716). 20371716
PTEN loss sarcoma sensitive YU238259 Preclinical Actionable In a preclinical study, YU238259 demonstrated increased cytotoxicity in PTEN-deficient sarcoma cell lines in culture (PMID: 26116172). 26116172
PTEN mutant uterine cancer sensitive GSK2256098 + Paclitaxel Preclinical Actionable In a preclinical study, uterine cancer cells harboring a PTEN mutation were more sensitive to the combination of GSK2256098 and Taxol (paclitaxel) than uterine cancer cells wild-type for PTEN, resulting in decreased tumor growth in culture and in mouse models (PMID: 25833835). 25833835
PTEN mutant uterine cancer sensitive GSK2256098 + Topotecan Preclinical Actionable In a preclinical study, uterine cancer cells harboring a PTEN mutation were more sensitive to the combination of GSK2256098 and Hycamtin (topotecan) than uterine cancer cells wild-type for PTEN, resulting in decreased tumor growth in culture and in mouse models (PMID: 25833835). 25833835
BRAF mut PTEN mut melanoma decreased response E6201 Preclinical - Cell line xenograft Actionable In a preclinical study, E6201 resulted in a cytostatic response in melanoma cell lines harboring both BRAF and PTEN mutations in culture and only inhibited tumor growth at very high doses in cell line xenograft models (PMID: 23039341). 23039341
BRAF mut PTEN wild-type melanoma sensitive E6201 Preclinical - Cell line xenograft Actionable In a preclinical study, E6201 resulted in a cytocidal response in PTEN wild-type melanoma cell lines harboring BRAF mutations in culture and inhibited tumor growth in cell line xenograft models (PMID: 23039341). 23039341
PTEN dec exp renal cell carcinoma no benefit Apitolisib Case Reports/Case Series Actionable In a retrospective analysis from a Phase II clinical trial, patients with metastatic renal cell carcinoma treated with Apitolisib (GDC-0980) had no difference in progression-free survival when stratified by high expression (n=17) and low expression (n=19) of Pten (PMID: 26951309). 26951309
PIK3CA act mut PTEN dec exp renal cell carcinoma no benefit Apitolisib Case Reports/Case Series Actionable In a retrospective analysis from a Phase II clinical trial, patients with metastatic renal cell carcinoma treated with Apitolisib (GDC-0980) had no difference in progression-free survival when stratified by the presence (n=21) or absence (n=15) of PIK3CA activating mutation and/or decreased Pten expression (PMID: 26951309). 26951309
PTEN dec exp renal cell carcinoma no benefit Everolimus Case Reports/Case Series Actionable In a retrospective analysis from a Phase II clinical trial, patients with metastatic renal cell carcinoma treated with Afinitor (everolimus) had no difference in progression-free survival when stratified by high expression (n=17) and low expression (n=21) of Pten (PMID: 26951309). 26951309
PIK3CA act mut PTEN dec exp renal cell carcinoma no benefit Everolimus Case Reports/Case Series Actionable In a retrospective analysis from a Phase II clinical trial, patients with metastatic renal cell carcinoma treated with Afinitor (everolimus) had no difference in progression-free survival when stratified by the presence (n=21) or absence (n=17) of PIK3CA activating mutations (PMID: 26951309). 26951309
PTEN loss RB1 loss triple-receptor negative breast cancer no benefit Palbociclib + Pictilisib Preclinical Actionable In a preclinical study, the combination of Ibrance (palbociclib) and Pictilisib (GDC-0941) did not improve growth inhibition compared to single drug treatment in triple-receptor negative breast cancer cell lines harboring PTEN and RB1 loss in culture (PMID: 27020857). 27020857
PTEN loss RB1 loss triple-receptor negative breast cancer resistant Pictilisib Preclinical Actionable In a preclinical study, a triple-receptor negative breast cancer line harboring PTEN and RB1 loss was resistant to Pictilisib (GDC-0941) induced growth inhibition in culture (PMID: 27020857). 27020857
PTEN loss RB1 loss triple-receptor negative breast cancer resistant Palbociclib Preclinical Actionable In a preclinical study, a triple-receptor negative breast cancer line harboring PTEN and RB1 loss was resistant to Ibrance (palbociclib) induced growth inhibition in culture (PMID: 27020857). 27020857
PTEN del head and neck squamous cell carcinoma resistant Taselisib Preclinical Actionable In a preclinical study, head and neck squamous cell carcinoma cells homozygous for PTEN deletion were resistant to Taselisib (GDC-0032) in culture (PMID: 26589432). 26589432
PTEN wild-type ovarian mucinous neoplasm sensitive KX2-391 Preclinical Actionable In a preclinical study, KX2-391 inhibited survival of PTEN wild-type mucinous ovarian carcinoma cell lines in culture, and reduced tumor growth in xenograft models (PMID: 24100628). 24100628
PTEN wild-type ovarian mucinous neoplasm sensitive KX2-391 + Oxaliplatin Preclinical Actionable In a preclinical study, KX2-391 and Eloxatin (oxaliplatin) synergistically inhibited survival of PTEN wild-type mucinous ovarian carcinoma cell lines in culture, and reduced tumor growth in xenograft models (PMID: 24100628). 24100628
PTEN loss ovarian mucinous neoplasm decreased response KX2-391 Preclinical Actionable In a preclincal study, mucinous ovarian carcinoma cell lines harboring PTEN loss were less sensitive to KX2-391 induced growth inhibition in culture and tumor suppression in xenograft models (PMID: 24100628). 24100628
PTEN G129R ovarian mucinous neoplasm decreased response KX2-391 Preclinical Actionable In a preclincal study, mucinous ovarian carcinoma cell lines over-expressing PTEN G129R were less sensitive to KX2-391 induced growth inhibition in culture and tumor suppression in xenograft models (PMID: 24100628). 24100628
PTEN loss ovarian mucinous neoplasm no benefit KX2-391 + Oxaliplatin Preclinical Actionable In a preclinical study, KX2-391 and Eloxatin (oxaliplatin) combination treatment did not show improved tumor suppression in xenograft models of ovarian mucinous carcinoma harboring PTEN loss when compared to single agent treatment (PMID: 24100628). 24100628
BRAF mut PTEN inact mut melanoma sensitive AZD6482 Preclinical Actionable In a preclincal study, AZD6482 inhibited Akt activation and proliferation in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive TGX-221 Preclinical Actionable In a preclincal study, TGX-221 inhibited Akt activation and proliferation in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Pictilisib Preclinical Actionable In a preclincal study, Pictilisib (GDC-0941) inhibited Akt activation and proliferation in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma decreased response Alpelisib Preclinical Actionable In a preclincal study, melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations were less sensitive to Alpelisib (BYL719)-induced growth inhibition in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Alpelisib + AZD6482 Preclinical Actionable In a preclinical study, AZD6482 and Alpelisib (BYL719) synergistically inhibited proliferation of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Alpelisib + GSK2636771 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of GSK2636771 and Alpelisib (BYL719) synergized to inhibit proliferation of human melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture, and inhibited tumor growth in xenograft models of one cell line harboring these mutations (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive AZD6482 + NVP-AEW541 Preclinical Actionable In a preclinical study, AZD6482 and NVP-AEW541 synergistically inhibited proliferation of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive GSK2636771 + NVP-AEW541 Preclinical Actionable In a preclinical study, GSK2636771 and NVP-AEW541 synergistically inhibited proliferation of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive NVP-AEW541 + Pictilisib Preclinical Actionable In a preclinical study, Pictilisib (GDC-0941) and NVP-AEW541 synergistically inhibited proliferation of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma no benefit Alpelisib + NVP-AEW541 Preclinical Actionable In a preclinical study, Alpelisib (BYL719) and NVP-AEW541 combination treatment did not enhance growth inhibition compared to single agent in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN wild-type melanoma no benefit AZD6482 + NVP-AEW541 Preclinical Actionable In a preclinical study, AZD6482 and NVP-AEW541 combination treatment did not enhance growth inhibition compared to single agent in PTEN wild-type melanoma cell lines harboring BRAF mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN wild-type melanoma no benefit GSK2636771 + NVP-AEW541 Preclinical Actionable In a preclinical study, GSK2636771 and NVP-AEW541 combination treatment did not enhance growth inhibition compared to single agent in PTEN wild-type melanoma cell lines harboring BRAF mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Encorafenib + Pictilisib Preclinical Actionable In a preclinical study, Pictilisib (GDC-0941) and Encorafenib (LGX818) synergistically inhibited proliferation of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive AZD6482 + Encorafenib Preclinical Actionable In a preclinical study, AZD6482 and Encorafenib (LGX818) synergistically inhibited proliferation of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Encorafenib + GSK2636771 Preclinical Actionable In a preclinical study, GSK2636771 and Encorafenib (LGX818) synergistically inhibited proliferation of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma no benefit Alpelisib + Encorafenib Preclinical Actionable In a preclinical study, Alpelisib (BYL719) and Encorafenib (LGX818) combination treatment did not enhance growth inhibition in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Binimetinib + Pictilisib Preclinical Actionable In a preclinical study, Pictilisib (GDC-0941) and Binimetinib (MEK162) synergistically inhibited proliferation of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive AZD6482 + Binimetinib Preclinical Actionable In a preclinical study, AZD6482 and Binimetinib (MEK162) synergistically inhibited proliferation of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Binimetinib + GSK2636771 Preclinical Actionable In a preclinical study, GSK2636771 and Binimetinib (MEK162) synergistically inhibited proliferation of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma no benefit Alpelisib + Binimetinib Preclinical Actionable In a preclinical study, Alpelisib (BYL719) and Binimetinib (MEK162) combination treatment did not enhance growth inhibition compared to single agent in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN wild-type melanoma no benefit AZD6482 + Binimetinib Preclinical Actionable In a preclinical study, AZD6482 and Binimetinib (MEK162) combination treatment did not enhance growth inhibition compared to single agent in PTEN wild-type melanoma cell lines harboring BRAF mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN wild-type melanoma no benefit Binimetinib + GSK2636771 Preclinical Actionable In a preclinical study, GSK2636771 and Binimetinib (MEK162) combination treatment did not enhance growth inhibition compared to single agent in PTEN wild-type melanoma cell lines harboring BRAF mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN wild-type melanoma no benefit AZD6482 + Encorafenib Preclinical Actionable In a preclinical study, AZD6482 and Encorafenib (LGX818) combination treatment did not enhance growth inhibition compared to single agent in PTEN wild-type melanoma cell lines harboring BRAF mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN wild-type melanoma no benefit Encorafenib + GSK2636771 Preclinical Actionable In a preclinical study, GSK2636771 and Encorafenib (LGX818) combination treatment did not enhance growth inhibition compared to single agent in PTEN wild-type melanoma cell lines harboring BRAF mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Alpelisib + AZD6482 + Encorafenib Preclinical Actionable In a preclinical study, combination treatment consisting of AZD6482, Encorafenib (LGX818), and Alpelisib (BYL719) efficiently induced apoptosis in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive AZD6482 + Encorafenib + NVP-AEW541 Preclinical Actionable In a preclinical study, combination treatment consists of AZD6482, Encorafenib (LGX818), and NVP-AEW541 efficiently induced apoptosis in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Alpelisib + AZD6482 + Binimetinib Preclinical - Cell culture Actionable In a preclinical study, combination treatment consisting of AZD6482, Binimetinib (MEK162), and Alpelisib (BYL719) efficiently induced apoptosis in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive AZD6482 + Binimetinib + NVP-AEW541 Preclinical Actionable In a preclinical study, combination treatment consists of AZD6482, Binimetinib (MEK162), and NVP-AEW541 efficiently induced apoptosis in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive AZD6482 + Binimetinib + Encorafenib + NVP-AEW541 Preclinical Actionable In a preclinical study, combination treatment consists of AZD6482, Binimetinib (MEK162), Encorafenib (LGX818), and NVP-AEW541 efficiently induced apoptosis in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Alpelisib + AZD6482 + Binimetinib + Encorafenib Preclinical Actionable In a preclinical study, combination treatment consisting of AZD6482, Binimetinib (MEK162), Encorafenib (LGX818), and Alpelisib (BYL719) efficiently induced apoptosis in melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations in culture (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Alpelisib + Encorafenib + GSK2636771 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of GSK2636771, Encorafenib (LGX818), and Alpelisib (BYL719) efficiently inhibited tumor growth in xenograft models of a human melanoma cell line harboring BRAF mutations and PTEN inactivating mutations (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive GSK2636771 + unspecified IGF-1R antibody Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment consisting of GSK2636771 and a figitumumab-like antibody inhibited tumor growth in xenograft models of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Encorafenib + unspecified IGF-1R antibody Preclinical Actionable In a preclinical study, combination treatment consists of Encorafenib (LGX818) and a figitumumab-like antibody inhibited tumor growth in xenograft models of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations (PMID: 26577700). 26577700
BRAF mut PTEN inact mut melanoma sensitive Encorafenib + GSK2636771 + unspecified IGF-1R antibody Preclinical - Cell line xenograft Actionable In a preclinical study, combination treatment consisting of GSK2636771, Encorafenib (LGX818), and a figitumumab-like antibody inhibited tumor growth in xenograft models of melanoma cell lines harboring BRAF mutations and PTEN inactivating mutations (PMID: 26577700). 26577700
PTEN V54fs glioblastoma sensitive XL147 Preclinical - Cell line xenograft Actionable In a preclinical study, XL147 inhibited tumor growth in xenograft models of a human glioblastoma cell line harboring PTEN V54fs (PMID: 25637314). 25637314
BRAF V600E PTEN loss melanoma sensitive XL147 Preclinical - Cell line xenograft Actionable In a preclinical study, XL147 inhibited tumor growth in xenograft models of a PTEN-deficient human melanoma cell line harboring BRAF V600E (PMID: 25637314). 25637314
BRAF V600D PTEN loss melanoma sensitive XL147 Preclinical - Cell line xenograft Actionable In a preclinical study, XL147 inhibited tumor growth in xenograft models of a PTEN-deficient human melanoma cell line harboring BRAF V600D (PMID: 25637314). 25637314
PTEN inact mut prostate adenocarcinoma sensitive XL147 Preclinical - Cell line xenograft Actionable In a preclinical study, XL147 inhibited PI3K signaling, growth, and migration of a human prostate adenocarcinoma cell line harboring a PTEN inactivating mutation in culture, and inhibited tumor growth and vascularization in xenograft models (PMID: 25637314). 25637314
PTEN inact mut prostate adenocarcinoma sensitive Paclitaxel + XL147 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of XL147 and Taxol (paclitaxel) inhibited tumor growth and angiogenesis in xenograft models of a human prostate adenocarcinoma cell line harboring an inactivating mutation in PTEN, with increased efficacy compared to either agent alone (PMID: 25637314). 25637314
PTEN loss melanoma sensitive DETD-35 Preclinical - Cell line xenograft Actionable In a preclinical study, DETD-35 treatment resulted in reduced tumor size in cell line xenograft models of Zelboraf (vemurafenib)-resistant melanoma harboring PTEN loss (PMID: 27048951). 27048951
PTEN loss melanoma sensitive DETD-35 + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, DETD-35 and Zelboraf (vemurafenib) combination treatment resulted in further reduction of tumor size in cell line xenograft models of Zelboraf (vemurafenib)-resistant melanoma harboring PTEN loss when compared to single agent treatment (PMID: 27048951). 27048951
BRAF V600E PTEN loss melanoma sensitive Pictilisib + PLX4720 Preclinical Actionable In a preclinical study, a melanoma cell line harboring BRAF V600E and PTEN loss demonstrated sensitivity when treated with a combination of Pictilisib (GDC-0941) and PLX4720, resulting in decreased cell proliferation in culture (PMID: 24265153). 24265153
BRAF V600X PTEN H93D melanoma predicted - sensitive Vemurafenib Clinical Study - Cohort Actionable In a clinical study, a melanoma patient harboring a BRAF V600 mutation and PTEN H93D treated with Zelboraf (vemurafenib) for 66 weeks demonstrated a partial response (PMID: 24265153). 24265153
PTEN dec exp Advanced Solid Tumor predicted - sensitive Pemetrexed Disodium + Sorafenib Phase I Actionable In a Phase I clinical trial, low Pten expression or lack of Pten expression was associated with better response to treatment with the combination of Alimta (pemetrexed) and Nexavar (sorafenib) in patients with advanced solid tumors (PMID: 27213589). 27213589
PTEN loss glioblastoma resistant A66 Preclinical - Cell line xenograft Actionable In a preclinical study, A66 did not inhibit tumor growth in a PTEN-null cell line xenograft model of glioblastoma (PMID: 21668414). 21668414
BRAF mut PTEN loss melanoma sensitive SAR260301 Preclinical - Cell line xenograft Actionable In a preclinical study, SAR260301 inhibited proliferation of PTEN deficient melanoma cells harboring BRAF mutations in culture and suppressed tumor growth in cell line xenograft models (PMID: 27196754). 27196754
PTEN loss prostate carcinoma decreased response SAR260301 Preclinical - Cell line xenograft Actionable In a precliinical study, PTEN deficient prostate carcinoma cells demonstrated reduced response to SAR260301 in cell culture and in cell line xenograft models (PMID: 27196754). 27196754
BRAF mut PTEN loss melanoma sensitive SAR260301 + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, SAR260301 and Zelboraf (vemurafenib) synergistically inhibited proliferation of PTEN deficient melanoma cells harboring BRAF mutations in culture and suppressed tumor growth in cell line xenograft models (PMID: 27196754). 27196754
BRAF mut PTEN loss melanoma sensitive SAR260301 + Selumetinib Preclinical - Cell line xenograft Actionable In a preclinical study, SAR260301 and Selumetinib (AZD6244) synergistically inhibited proliferation of PTEN deficient melanoma cells harboring BRAF mutations in culture and suppressed tumor growth in cell line xenograft models (PMID: 27196754). 27196754
BRAF V600E PTEN loss melanoma sensitive GDC0879 + Pictilisib Preclinical - Cell culture Actionable In a preclinical study, GDC0879 and Pictilisib (GDC-0941) synergistically inhibited survival of melanoma cell lines harboring BRAF V600E and PTEN loss in cell culture (PMID: 19276360). 19276360
PIK3CA mut PTEN loss prostate cancer sensitive PKI-179 Preclinical - Cell culture Actionable In a preclinical study, PKI-179 inhibited growth of prostate cancer cells harboring PIK3CA mutations and PTEN loss in culture (PMID: 20797855). 20797855
BRAF V600X PTEN neg melanoma sensitive Uprosertib Preclinical - Cell culture Actionable In a preclinical study, lack of PTEN expression was associated with increased sensitivity to GSK2141795 in BRAF V600-mutant melanoma cell lines in culture (PMID: 24265152). 24265152
PTEN R130* follicular thyroid carcinoma sensitive MK2206 Preclinical - Cell culture Actionable In a preclinical study, MK2206 inhibited AKT activation, proliferation, and growth of thyroid cancer cell lines with PI3K/AKT pathway alterations in culture, including an follicular thyroid cancer cell line harboring PTEN R130* and loss of one PTEN allele (PMID: 21289267). 21289267
PTEN loss prostate cancer sensitive Acalisib Preclinical - Cell line xenograft Actionable In a preclinical study, Acalisib (GS-9820) resulted in tumor growth inhibition in xenograft models of prostate cancer with PTEN deficiency (Mol Cancer Ther 2009;8(12 Suppl):B136). detail...
PTEN inact mut estrogen-receptor positive breast cancer sensitive Pictilisib Preclinical - Cell culture Actionable In a preclinical study, a PTEN deficient estrogen-receptor (ER) positive breast cancer cell line demonstrated increased sensitivity to treatment in culture when Pictilisib (GDC-0941) was given at a higher dose for a shorter duration, resulting in inhibition of cell growth (PMID: 26733612). 26733612
PTEN inact mut estrogen-receptor positive breast cancer sensitive Fulvestrant + Pictilisib Preclinical - Cell culture Actionable In a preclinical study, the combination of Faslodex (fulvestrant) and PIctilisib (GDC-0941) resulted in decreased cell viability and increased apoptotic activity in PTEN deficient estrogen-receptor (ER) positive breast cancer cells in culture (PMID: 26733612). 26733612
BRAF V600E PTEN loss TP53 wild-type colorectal cancer sensitive PD-0325901 + Sapanisertib Preclinical - Cell line xenograft Actionable In a preclinical study, Sapanisertib (MLN0128) and PD-0325901 synergistically inhibited Erk and PI3K signaling and proliferation, induced apoptosis in TP53-wild-type colorectal cancer cells harboring BRAF V600E and PTEN loss in culture and in cell line xenograft models (PMID: 26272063). 26272063
PTEN loss glioblastoma no benefit BLZ945 Preclinical Actionable In a preclinical study, BLZ945 resulted in limited benefit in transgenic mouse models of glioblastoma harboring a loss of PTEN (PMID: 27199435). 27199435
PTEN mutant breast cancer predicted - sensitive MS417 + Pictilisib Preclinical - Cell culture Actionable In a preclinical study, Pictilisib (GDC-0941) and MS417 in combination resulted in improved growth inhibition and increased cell death compared to either agent alone in a PTEN-mutant breast cancer cell line in culture (PMID: 26058079). 26058079
PTEN loss ovarian cancer sensitive OSI-027 Preclinical - Cell line xenograft Actionable In a preclinical study, OSI-027 inhibited tumor growth in PTEN-null ovarian cancer cell line xenograft models (PMID: 21673091). 21673091
PTEN loss breast cancer sensitive OSI-027 Preclinical - Cell line xenograft Actionable In a preclinical study, OSI-027 induced tumor regression in PTEN-null breast cancer cell line xenograft models (PMID: 21673091). 21673091
PTEN L108R breast cancer sensitive BAY1125976 Preclinical - Cell culture Actionable In a preclinical study, BAY1125976 inhibited proliferation of a breast cancer cell line harboring PTEN L108R in culture (PMID: 27699769). 27699769
PTEN T319fs breast cancer sensitive BAY1125976 Preclinical - Cell culture Actionable In a preclinical study, BAY1125976 inhibited proliferation of a breast cancer cell line harboring PTEN T319fs*1 in culture (PMID: 27699769). 27699769
PIK3CA H1047R PTEN E307K breast cancer sensitive BAY1125976 Preclinical - Cell culture Actionable In a preclinical study, BAY1125976 inhibited proliferation of a breast cancer cell line harboring PIK3CA H1047R and PTEN E307K in culture (PMID: 27699769). 27699769
PTEN C136Y breast cancer resistant Alpelisib Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PTEN C136Y demonstrated resistance to Alpelisib (BYL719) in culture (PMID: 27604488). 27604488
PTEN loss breast cancer resistant Alpelisib Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PTEN loss demonstrated resistance to Alpelisib (BYL719) in culture (PMID: 27604488). 27604488
PTEN loss glioblastoma sensitive LY3023414 Preclinical - Cell line xenograft Actionable In a preclinical study, LY3023414 inhibited Pi3k/mTor signaling and proliferation in PTEN deficient glioblastoma cells in culture, and resulted in tumor growth inhibition in cell line xenograft models (PMID: 27439478). 27439478
PTEN inact mut kidney cancer sensitive LY3023414 Preclinical - Cell line xenograft Actionable In a preclinical study, LY3023414 inhibited proliferation of renal cancer cells harboring PTEN inactivating mutation in culture, resulted in tumor growth inhibition in cell line xenograft models (PMID: 27439478). 27439478
PTEN del prostate cancer sensitive Ipatasertib Preclinical - Cell line xenograft Actionable In a preclinical study, prostate cancer cell line xenograft models with PTEN deletion demonstrated sensitivity to treatment with Ipatasertib (GDC-0068), resulting in inhibition of tumor growth (PMID: 24141624). 24141624
APC inact mut PTEN inact mut colorectal cancer sensitive Dactolisib Preclinical Actionable In a preclinical study, BEZ235 inhibited PI3K/mTOR signaling in tumors and increased survival in transgenic animal models of colorectal cancer driven by APC and PTEN inactivation (PMID: 26206338). 26206338
APC inact mut PTEN inact mut colorectal cancer no benefit Binimetinib Preclinical Actionable In a preclinical study, Binimetinib (MEK162) reduced proliferation in tumors acutely but did not improve survival in transgenic animal models of colorectal cancer driven by APC and PTEN inactivation (PMID: 26206338). 26206338
APC inact mut PTEN inact mut colorectal cancer no benefit Binimetinib + Dactolisib Preclinical Actionable In a preclinical study, combination of BEZ235 and Binimetinib (MEK162) did not improve survival compared to single agent in transgenic animal models of colorectal cancer driven by APC and PTEN inactivation (PMID: 26206338). 26206338
PTEN loss prostate cancer predicted - sensitive CX-6258 Preclinical Actionable In a preclinical study, CX-6258 treatment resulted in decreased carcinoma in situ in PTEN deficient transgenic animal model of prostate cancer (PMID: 27486174). 27486174
PTEN loss prostate cancer predicted - sensitive CX-5461 + CX-6258 Preclinical Actionable In a preclinical study, CX-6258 and CX-5461 combination treatment resulted in decreased tumor burden in PTEN deficient transgenic animal model of prostate cancer (PMID: 27486174). 27486174
PTEN loss prostate cancer no benefit CX-5461 Preclinical Actionable In a preclinical study, CX-5461 treatment did not result in histological change in PTEN deficient transgenic animal model of prostate cancer (PMID: 27486174). 27486174
PIK3CA R88L PIK3CA E453del PIK3CA T1052K PTEN loss PTEN mut endometrial carcinoma sensitive Copanlisib Phase I Actionable In a Phase I clinical trial, an endometrial carcinoma patient harboring PIK3CA T1052K, R88L, and E453del, as well as a PTEN mutation and loss of PTEN protein expression demonstrated a complete response to treatment with Aliqopa (copanlisib) (PMID: 27672108). 27672108
PTEN loss prostate cancer sensitive Ipatasertib Case Reports/Case Series Actionable In a Phase I trial, a patient with castration resistant prostate cancer harboring a loss of PTEN demonstrated an improved prostate specific antigen when treated with Ipatasertib (GDC-0068) (PMID: 27872130). 27872130
PTEN loss renal carcinoma sensitive Capivasertib Preclinical - Pdx Actionable In a preclinical study, Truqap (capivasertib) inhibited growth of renal cancer Pdx models with Pten loss (PMID: 22294718). 22294718
PTEN inact mut Advanced Solid Tumor sensitive Capivasertib Preclinical - Cell culture Actionable In a preclinical study, Truqap (capivasertib) inhibited growth of various solid tumor cell culture models with inactivating Pten mutations (PMID: 22294718). 22294718
PTEN loss Advanced Solid Tumor sensitive Capivasertib Preclinical - Cell culture Actionable In a preclinical study, Truqap (capivasertib) inhibited growth of various solid tumor cell culture models with loss of Pten (PMID: 22294718). 22294718
PTEN negative lung squamous cell carcinoma sensitive Buparlisib Phase II Actionable In a Phase II trial, one patient with Pten negative squamous NSCLC had a partial response to Buparlisib (BKM120) however, stage 2 of the study was not initiated due to failure of meeting overall stage 1 response endpoints (PMID: 26098748). 26098748
PTEN mutant breast cancer sensitive CUDC-907 Preclinical - Cell culture Actionable In a preclinical study, CUDC-907 inhibited growth of breast cancer cells lines harboring Pten mutations and/or deletions (PMID: 22693356). 22693356
PTEN del breast cancer sensitive CUDC-907 Preclinical - Cell culture Actionable In a preclinical study, CUDC-907 inhibited growth of breast cancer cells lines harboring Pten mutations and/or deletions (PMID: 22693356). 22693356
PTEN mut TP53 mut glioblastoma sensitive Navitoclax + ONC201 Preclinical - Cell culture Actionable In a preclinical study, the combination of ONC201 (TIC10) and Navitoclax (ABT-263) increased apoptosis and worked synergistically to inhibit proliferation of a glioblastoma cell line harboring mutations in PTEN and TP53 in culture (PMID: 26474387). 26474387
PIK3CA H1047R PTEN loss breast cancer no benefit Dactolisib + Venetoclax Preclinical - Cell culture Actionable In a preclinical study, inhibition of Pi3k signaling by BEZ235 did not sensitize breast cancer cell lines harboring PIK3CA H1047R and PTEN loss to Venclexta (venetoclax) in culture (PMID: 27974663). 27974663
PIK3CA H1047R PTEN loss breast cancer predicted - sensitive Dactolisib + WEHI-539 Preclinical - Cell culture Actionable In a preclinical study, inhibition of Pi3k signaling by BEZ235 sensitized breast cancer cell lines harboring PIK3CA H1047R and PTEN loss to WEHI-539 in culture (PMID: 27974663). 27974663
PIK3CA E542K PTEN loss breast cancer predicted - sensitive Dactolisib + WEHI-539 Preclinical - Cell culture Actionable In a preclinical study, inhibition of Pi3k signaling by BEZ235 sensitized breast cancer cell lines harboring PIK3CA E542K and PTEN loss to WEHI-539 in culture (PMID: 27974663). 27974663
PIK3CA H1047R PTEN loss triple-receptor negative breast cancer predicted - sensitive ABT-737 + Dactolisib Preclinical - Cell line xenograft Actionable In a preclinical study, ABT-737 and BEZ235 combination treatment resulted in tumor regression in cell line xenograft models of triple-receptor negative breast cancer harboring PIK3CA H1047R and PTEN loss (PMID: 27974663). 27974663
PIK3CA H1047R PTEN loss triple-receptor negative breast cancer predicted - sensitive ABT-737 + Sapanisertib Preclinical - Cell line xenograft Actionable In a preclinical study, ABT-737 and Sapanisertib (MLN0128) combination treatment resulted in inhibition of tumor growth in cell line xenograft models of triple-receptor negative breast cancer harboring PIK3CA H1047R and PTEN loss (PMID: 27974663). 27974663
PTEN loss castration-resistant prostate carcinoma sensitive Abiraterone + Ipatasertib Phase II Actionable In a Phase II trial, the combination of Ipatasertib (GDC-0068) and Zytiga (abiraterone) resulted in a better progression-free survival in metastatic castration resistant prostate cancer patients with PTEN loss (by IHC) when compared to placebo combined with Zytiga (PMID: 30037818; NCT01485861). 30037818 detail...
PTEN loss castration-resistant prostate carcinoma sensitive Abiraterone + Ipatasertib Phase III Actionable In a Phase III trial, Ipatasertib (GDC-0068) combined with Zytiga (abiraterone) significantly improved median radiographical progression-free survival (PFS) (18.5 vs 16.5 mo, HR 0.77, p=0.034) in patients with metastatic castration resistant prostate cancer harboring PTEN loss (by IHC) when compared to placebo combined with Zytiga, median PFS was not improved (19.2 vs 16.6 mo, HR 0.84, p=0.043) in the intent-to-treat population (PMID: 34246347; NCT03072238). 34246347
PTEN E235* TSC2 G440S endometrial cancer sensitive Miransertib Preclinical - Pdx Actionable In a preclinical study, a patient-derived xenograft (PDX) model of endometrial cancer harboring PTEN E235* and TSC2 G440S was sensitive to Miransertib (ARQ092), demonstrating greater than 90% inhibition of tumor growth (PMID: 26469692). 26469692
PIK3CA R93W PIK3CA D350G PTEN R130G endometrial cancer sensitive Miransertib Preclinical - Pdx Actionable In a preclinical study, a patient-derived xenograft (PDX) model of endometrial cancer harboring PIK3CA D350G, PIK3CA R93W, and PTEN R130G was sensitive to Miransertib (ARQ092), demonstrating greater than 90% inhibition of tumor growth (PMID: 26469692). 26469692
PTEN loss triple-receptor negative breast cancer sensitive M2698 Preclinical - Cell line xenograft Actionable In a preclinical study, a triple-receptor negative breast cancer xenograft model harboring PTEN loss was sensitive to M2698 (MSC2363318A), demonstrating inhibition of tumor growth and tumor regression (PMID: 27186432). 27186432
PTEN mutant melanoma sensitive BI-69A11 Preclinical - Cell line xenograft Actionable In a preclinical study, BI-69A11 resulted in antitumor activity in a melanoma cell line harboring a PTEN mutation, including induction of cell death in culture, and in xenograft models, tumor growth inhibition and tumor regression (PMID: 19175524). 19175524
PTEN loss transitional cell carcinoma predicted - resistant Everolimus Case Reports/Case Series Actionable In a Phase II trial, 57% (8/14) of transitional cell carcinoma patients that demonstrated uncontrolled disease after treatment with Afinitor (everolimus) harbored PTEN loss, while 0% (0/6) of patients with controlled disease demonstrated evidence of PTEN loss (PMID: 22473592). 22473592
PTEN L108R breast cancer sensitive DHM25 Preclinical - Cell culture Actionable In a preclinical study, DHM25 increased cell death in a breast cancer cell line harboring PTEN L108R in culture (PMID: 26237138). 26237138
PTEN A72fs triple-receptor negative breast cancer sensitive DHM25 Preclinical - Cell culture Actionable In a preclinical study, DHM25 increased cell death in a triple-negative breast cancer cell line harboring PTEN A72fs*5 in culture (PMID: 26237138). 26237138
PTEN V275* triple-receptor negative breast cancer sensitive DHM25 Preclinical - Cell culture Actionable In a preclinical study, DHM25 increased cell death and decreased migration of a triple-negative breast cancer cell line harboring PTEN V275* in culture (PMID: 26237138). 26237138
PIK3CA act mut PTEN wild-type breast cancer sensitive Everolimus Preclinical - Cell culture Actionable In a preclinical study, 8/9 breast cancer cell lines harboring a PIK3CA activating mutation and PTEN wild-type demonstrated sensitivity to treatment with Afinitor (everolimus) in culture, resulting in decreased cell viability (PMID: 21358673). 21358673
PIK3CA act mut PTEN wild-type breast cancer sensitive Torkinib Preclinical - Cell culture Actionable In a preclinical study, 9/9 breast cancer cell lines harboring a PIK3CA activating mutation and PTEN wild-type demonstrated sensitivity to treatment with Torkinib (PP242) in culture, resulting in decreased cell viability (PMID: 21358673). 21358673
PTEN loss breast cancer resistant Everolimus Preclinical - Cell culture Actionable In a preclinical study, 10/12 breast cancer cell lines with PTEN loss demonstrated resistance to Afinitor (everolimus) in culture (PMID: 21358673). 21358673
PTEN loss breast cancer resistant Torkinib Preclinical - Cell culture Actionable In a preclinical study, breast cancer cell lines with PTEN loss demonstrated resistance to Torkinib (PP242) in culture (PMID: 21358673). 21358673
PIK3CA wild-type PTEN loss breast cancer no benefit Everolimus + U0126 Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PIK3CA wild-type and PTEN loss did not demonstrate any benefit when treated with a combination of Afinitor (everolimus) and U0126 (PMID: 21358673). 21358673
PIK3CA wild-type PTEN loss breast cancer no benefit Everolimus + Selumetinib Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PIK3CA wild-type and PTEN loss did not demonstrate any benefit when treated with a combination of Afinitor (everolimus) and Selumetinib (AZD6244) (PMID: 21358673). 21358673
PIK3CA wild-type PTEN loss breast cancer sensitive Torkinib + U0126 Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PIK3CA wild-type and PTEN loss demonstrated sensitivity to the combination treatment of Torkinib (PP242) and U0126 in culture (PMID: 21358673). 21358673
PIK3CA wild-type PTEN loss breast cancer sensitive Selumetinib + Torkinib Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PIK3CA wild-type and PTEN loss demonstrated sensitivity to the combination treatment of Torkinib (PP242) and Selumetinib (AZD6244) in culture (PMID: 21358673). 21358673
PTEN loss Advanced Solid Tumor no benefit Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) treatment in patients with advanced solid tumors harboring PTEN loss did not reach its primary endpoint, resulting in only stable disease or progressive disease (PMID: 28330462). 28330462
PTEN loss acute lymphoblastic leukemia sensitive BGT226 Preclinical - Cell culture Actionable In a preclinical study, BGT226 treatment in PTEN-deficient acute lymphoblastic leukemia cells resulted in inhibition of PI3K/Akt signaling and apoptotic activity in culture (PMID: 23705826). 23705826
PTEN loss glioblastoma sensitive GDC-0084 Preclinical - Cell line xenograft Actionable In a preclinical study, two PTEN-deficient glioblastoma cell line xenograft models were sensitive to GDC-0084 treatment, resulting in decreased tumor volumes by 70% and 40%, and inhibition of PI3K pathway signaling (PMID: 27638506). 27638506
PTEN loss prostate cancer sensitive Voxtalisib Preclinical - Cell line xenograft Actionable In a preclinical study, a prostate cancer cell line with PTEN loss was sensitive to XL765 (SAR245409) treatment, demonstrating inhibition of the PI3K pathway and decreased colony formation in culture, and inhibition of tumor growth and reduced tumor vascularization in cell line xenograft models (PMID: 24634413). 24634413
PTEN inact mut glioblastoma sensitive Voxtalisib Preclinical - Cell line xenograft Actionable In a preclinical study, a glioblastoma cell line harboring a PTEN inactivating mutation was sensitive to XL765 (SAR245409), demonstrating inhibition of cell proliferation in culture, and inhibition of tumor growth in cell line xenograft models (PMID: 24634413). 24634413
PTEN loss breast adenocarcinoma sensitive Voxtalisib Preclinical - Cell culture Actionable In a preclinical study, a breast adenocarcinoma cell line harboring PTEN loss was sensitive to XL765 (SAR245409), demonstrating inhibition of cell proliferation in culture (PMID: 24634413). 24634413
PTEN inact mut glioblastoma sensitive SF1126 Preclinical - Cell line xenograft Actionable In a preclinical study, a glioblastoma cell line harboring a PTEN inactivating mutation (PMID: 24634413) was sensitive to SF1126, demonstrating inhibition of tumor growth in cell line xenograft models (PMID: 18172313). 18172313 24634413
PTEN loss prostate cancer sensitive SF1126 Preclinical - Cell line xenograft Actionable In a preclinical study, a glioblastoma cell line harboring PTEN loss (PMID: 24634413) was sensitive to SF1126, demonstrating a 76% decrease in tumor growth in cell line xenograft models (PMID: 18172313). 18172313 24634413
FGFR2 N550K PIK3CA I20M PIK3CA P539R PTEN R130Q PTEN T321fs endometrial cancer sensitive Infigratinib + Pictilisib Preclinical - Cell culture Actionable In a preclinical study, the combination of Truseltiq (infigratinib) and Pictilisib (GDC-0941) resulted in a synergistic effect, demonstrating inhibition of cell proliferation, decreased colony formation, and cell death in an endometrial cancer cell line harboring FGFR2 N550K, PIK3CA I20M and P539R, and PTEN R130Q and T321fs*23 in culture (PMID: 28119489). 28119489
FGFR2 N550K PIK3CA I20M PIK3CA P539R PTEN R130Q PTEN T321fs endometrial cancer sensitive Buparlisib + Infigratinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Truseltiq (infigratinib) and Buparlisib (BKM120) resulted in a synergistic effect, demonstrating inhibition of cell proliferation, decreased colony formation, and cell death in an endometrial cancer cell line harboring FGFR2 N550K, PIK3CA I20M and P539R, and PTEN R130Q and T321fs*23 in culture (PMID: 28119489). 28119489
FGFR2 N550K PIK3CA I20M PIK3CA P539R PTEN R130Q PTEN T321fs endometrial cancer sensitive Alpelisib + Infigratinib Preclinical - Cell culture Actionable In a preclinical study, the combination of Truseltiq (infigratinib) and Alpelisib (BYL719) resulted in a synergistic effect, demonstrating inhibition of cell proliferation and induced cell death in an endometrial cancer cell line harboring FGFR2 N550K, PIK3CA I20M and P539R, and PTEN R130Q and T321fs*23 in culture (PMID: 28119489). 28119489
PTEN loss prostate cancer sensitive Capivasertib Preclinical Actionable In a preclinical study, treatment with Truqap (capivasertib) decreased AKT phosphorylation and downstream signaling and reduced tumor burden in mouse models of PTEN-deficient prostate cancer, including both castration-naive and castration-resistant models (PMID: 26910118). 26910118
PTEN loss TP53 loss prostate cancer sensitive Capivasertib Preclinical Actionable In a preclinical study, treatment with Truqap (capivasertib) improved overall survival and progression-free survival in mouse models of prostate cancer with inactivated PTEN and TP53 (PMID: 26910118). 26910118
PTEN loss head and neck squamous cell carcinoma predicted - sensitive Rigosertib Sodium Case Reports/Case Series Actionable In a Phase I trial, Rigosertib (ON 01910.Na) treatment resulted in a partial response with 53% decrease of liver metastasis in a patients with head and neck squamous cell carcinoma harboring PTEN loss (Cancer Res April 15 2013 (73) (8 Supplement) LB-198). detail...
BRAF V600E PTEN loss melanoma sensitive GSK2636771 + unspecified PD-1 antibody Preclinical Actionable In a preclinical study, treatment with the combination of GSK2636771 and a PD-1 antibody resulted in greater tumor infiltration of T-cells and tumor growth inhibition in mouse melanoma models expressing BRAF V600E with PTEN loss compared to either agent alone (PMID: 26645196). 26645196
BRAF V600E PTEN mut melanoma sensitive ASN003 Preclinical - Cell line xenograft Actionable In a preclinical study, a melanoma cell line xenograft model co-harboring BRAF V600E and a PTEN mutation demonstrated tumor growth inhibition when treated with ASN003 (Mol Cancer Ther 2015;14(12 Suppl 2):Abstract nr B100). detail...
PTEN loss colorectal cancer sensitive AZD8186 + Vistusertib Phase I Actionable In a Phase I trial, the combination of AZD8186 and Vistusertib (AZD2014) resulted in a partial response in a patient with PTEN-deficient colorectal cancer (J Clin Oncol 35, 2017 (suppl; abstr 2570)). detail...
PTEN dec exp triple-receptor negative breast cancer predicted - sensitive Ipatasertib + Paclitaxel Phase II Actionable In a Phase II trial, Ipatasertib (GDC-0068) in combination with Abraxane (paclitaxel) resulted in improved progression free survival (6.2 vs 3.7 months) compared to placebo in triple-receptor negative breast cancer patients with low Pten expression (PMID: 28800861; NCT02162719). 28800861
PTEN loss head and neck squamous cell carcinoma predicted - sensitive AZD8055 Preclinical - Cell culture Actionable In a preclinical study, treatment with AZD8055 resulted in antiproliferative activity in head and neck squamous cell carcinoma cells harboring PTEN loss in culture (PMID: 28446642). 28446642
PTEN loss head and neck squamous cell carcinoma predicted - sensitive Dactolisib Preclinical - Cell culture Actionable In a preclinical study, BEZ235 resulted in antiproliferative activity in head and neck squamous cell carcinoma cells harboring PTEN loss in culture (PMID: 28446642). 28446642
PTEN loss breast adenocarcinoma sensitive Gemcitabine + LY2780301 Phase Ib/II Actionable In a Phase Ib trial, a breast adenocarcinoma patient harboring PTEN loss demonstrated a partial response when treated with a combination of LY2780301 and Gemzar (gemcitabine) (PMID: 28750271). 28750271
PTEN loss diffuse large B-cell lymphoma predicted - sensitive Idelalisib + MK2206 Preclinical - Cell culture Actionable In a preclinical study, MK2206 partially restored sensitivity to Zydelig (idelalisib) in diffuse large B-cell lymphoma cells harboring PTEN loss with acquired resistance to Zydelig (idelalisib), resulting in increased apoptosis in culture (PMID: 28178345). 28178345
PTEN loss diffuse large B-cell lymphoma predicted - sensitive GSK2334470 + Idelalisib Preclinical - Cell culture Actionable In a preclinical study, GSK2334470 partially restored sensitivity to Zydelig (idelalisib) in diffuse large B-cell lymphoma cells harboring PTEN loss that acquired resistance to Zydelig (idelalisib), resulting in increased apoptosis in culture (PMID: 28178345). 28178345
PTEN R130Q head and neck squamous cell carcinoma predicted - sensitive Carboplatin + Paclitaxel + Temsirolimus Case Reports/Case Series Actionable In a Phase II trial, a patient with head and neck squamous cell carcinoma harboring PTEN R130Q demonstrated a partial response when treated with the combination of Torisel (temsirolimus), Paraplatin (carboplatin), and Taxol (paclitaxel) (PMID: 28961834). 28961834
BRAF mut PTEN mut melanoma predicted - sensitive ST-162 Preclinical - Cell line xenograft Actionable In a preclinical study, ST-162 resulted in tumor regression in a melanoma cell line xenograft model co-harboring mutations in BRAF and PTEN (PMID: 28775144). 28775144
PIK3CA E545K PTEN loss stomach cancer unknown Sirolimus Phase 0 Actionable In a pilot clinical trial, Rapamune (sirolimus) demonstrated modest clinical activity in patients with PIK3CA-mutant gastric cancer (n=3) or hilar cholangiocarcinoma (n=1), including 2 gastric cancer patients harboring PIK3CA E545K and PTEN loss, with a 0% response rate, median progression-free survival of 1.9 months, and median overall survival of 3.6 months (PMID: 28685070). 28685070
PIK3CA E542K PTEN loss stomach cancer unknown Sirolimus Phase 0 Actionable In a pilot clinical trial, Rapamune (sirolimus) demonstrated modest clinical activity in patients with PIK3CA-mutant gastric cancer (n=3) or hilar cholangiocarcinoma (n=1), including a gastric cancer patient harboring PIK3CA E542K and PTEN loss, with a 0% response rate, median progression-free survival of 1.9 months, and median overall survival of 3.6 months (PMID: 28685070). 28685070
PTEN N48I urinary bladder cancer decreased response Pictilisib Preclinical - Cell culture Actionable In a preclinical study, Pictilisib (GDC-0941) demonstrated limited inhibition of Akt phosphorylation and growth in bladder cancer cells harboring PTEN N48I in culture (PMID: 28808038). 28808038
PIK3CA P124L PTEN del urinary bladder cancer decreased response Pictilisib Preclinical - Cell culture Actionable In a preclinical study, Pictilisib (GDC-0941) demonstrated limited inhibition of Akt phosphorylation and growth in bladder cancer cells harboring PIK3CA P124L and PTEN deletion in culture (PMID: 28808038). 28808038
BRAF V600E/K PTEN loss Advanced Solid Tumor predicted - sensitive PX-866 + Vemurafenib Phase I Actionable In a Phase I trial, the combination therapy of PX-866 and Zelboraf (vemurafenib) demonstrated safety and resulted in an objective response in 28% (5/18) of advanced solid tumor patients harboring BRAF V600E or K, and of those five patients, 80% (4/5) also demonstrated loss of PTEN (PMID: 29051322). 29051322
PTEN loss prostate cancer predicted - sensitive AR-mTOR-26 Preclinical - Cell line xenograft Actionable In a preclinical study, AR-mTOR-26 treatment resulted in antitumor activity in prostate cancer xenograft models with PTEN loss (Cancer Res 2010;70(8 Suppl):Abstract nr 4484). detail...
KIT Y570_L576del KIT D816E PTEN T321fs gastrointestinal stromal tumor resistant Imatinib Preclinical - Pdx Actionable In a preclinical study, Gleevec (imatinib mesylate) did not inhibit Kit, Erk, or Mtor signaling and resulted in minimal tumor inhibition in patient-derived xenograft (PDX) model of gastrointestinal stromal tumor harboring KIT D816E, KIT Y570_L576del, and PTEN T321fs, which was established from a patient who did not respond to Gleevec (imatinib mesylate), Sutent (sunitinib), and Nexavar (sorafenib) (PMID: 29100343). 29100343
KIT Y570_L576del KIT D816E PTEN T321fs gastrointestinal stromal tumor resistant Sunitinib Preclinical - Pdx Actionable In a preclinical study, Sutent (sunitinib) demonstrated limited inhibition of Kit, Erk, and Mtor signaling and resulted in minimal (22.9%) tumor inhibition in patient-derived xenograft (PDX) model of gastrointestinal stromal tumor harboring KIT D816E, KIT Y570_L576del, and PTEN T321fs, which was established from a patient who did not respond to Gleevec (imatinib mesylate), Sutent (sunitinib), and Nexavar (sorafenib) (PMID: 29100343). 29100343
KIT Y570_L576del KIT D816E PTEN T321fs gastrointestinal stromal tumor sensitive Regorafenib Preclinical - Pdx Actionable In a preclinical study, Stivarga (regorafenib) inhibited Kit, Erk, and Mtor signaling, resulted in tumor growth inhibition in patient-derived xenograft (PDX) model of gastrointestinal stromal tumor harboring KIT D816E, KIT Y570_L576del, and PTEN T321fs, which was established from a patient who did not respond to Gleevec (imatinib mesylate), Sutent (sunitinib), and Nexavar (sorafenib) (PMID: 29100343). 29100343
KIT Y570_L576del KIT D816E PTEN T321fs gastrointestinal stromal tumor sensitive Imatinib + LY294002 Preclinical - Pdx Actionable In a preclinical study, Gleevec (imatinib mesylate) in combination with LY294002 resulted in enhanced tumor growth inhibition compared to monotherapy in patient-derived xenograft (PDX) model of gastrointestinal stromal tumor harboring KIT D816E, KIT Y570_L576del, and PTEN T321fs, which was established from a patient who did not respond to Gleevec (imatinib mesylate), Sutent (sunitinib), and Nexavar (sorafenib) (PMID: 29100343). 29100343
PTEN loss renal cell carcinoma predicted - sensitive Everolimus Clinical Study - Cohort Actionable In a retrospective analysis, loss of PTEN expression was associated with improved progression-free survival compared to PTEN positive (10.5 vs 5.3 months) in renal cell carcinoma patients treated with Afinitor (everolimus) (PMID: 30327302). 30327302
PTEN loss lung squamous cell carcinoma no benefit Buparlisib Preclinical - Pdx & cell culture Actionable In a preclinical study, lung squamous cell carcinoma patient-derived xenograft (PDX) models harboring PTEN loss did not respond to Buparlisib (BKM120) treatment (PMID: 30093452). 30093452
PIK3CA E542K PTEN loss lung squamous cell carcinoma sensitive Alpelisib Preclinical - Pdx Actionable In a preclinical study, Alpelisib (BYL719) treatment resulted in response in a lung squamous cell carcinoma patient-derived xenograft (PDX) model harboring PIK3CA E542K and PTEN loss (PMID: 30093452). 30093452
CDKN2A loss PIK3CA E542K PTEN loss lung squamous cell carcinoma sensitive Buparlisib + Palbociclib Preclinical - Pdx Actionable In a preclinical study, treatment with the combination of Buparlisib (BYL719) and Ibrance (palbociclib) resulted in increased tumor growth inhibition compared to either agent alone in a patient-derived xenograft (PDX) model of lung squamous cell carcinoma harboring PIK3CA E542K and PTEN loss, with p16 (CDKN2A) loss (PMID: 30093452). 30093452
IDH1 wild-type PTEN mut glioblastoma resistant Pembrolizumab Clinical Study - Cohort Actionable In a retrospective analysis, PTEN mutations were significantly enriched in patients with IDH1 wild-type glioblastoma who did not respond to anti-PD-1 therapy, with either Keytruda (pembrolizumab) or Opdivo (nivolumab), compared to those who responded (odds ratio=0.85, p=0.0063), with 23 PTEN mutations identified in 32 non-responders and 3 in 13 responders (PMID: 30742119). 30742119
IDH1 wild-type PTEN mut glioblastoma resistant Nivolumab Clinical Study - Cohort Actionable In a retrospective analysis, PTEN mutations were significantly enriched in patients with IDH1 wild-type glioblastoma who did not respond to anti-PD-1 therapy with either Keytruda (pembrolizumab) or Opdivo (nivolumab), compared to those who responded (odds ratio=0.85, p=0.0063), with 23 PTEN mutations identified in 32 non-responders and 3 in 13 responders (PMID: 30742119). 30742119
PTEN mutant hepatocellular carcinoma decreased response Sorafenib Clinical Study - Cohort Actionable In a clinical case study, Nexavar (sorafenib) treatment of patients with hepatocellular carcinoma harboring Mtor pathway mutations in PIK3CA, PTEN, TSC2, or TSC1 (n=12), resulted in a lower disease control rate (8.3% vs. 40.2%), shorter progression-free survival (1.9 months vs. 5.3 months) and shorter overall survival (10.4 months vs. 17.9 months) compared to patients without mutations in this pathway (n=67) (PMID: 30373752; NCT01775072). 30373752
PIK3CA wild-type PTEN pos head and neck squamous cell carcinoma predicted - sensitive Cetuximab + Cisplatin Clinical Study - Cohort Actionable In a clinical study, combination of Erbitux (cetuximab) with Platinol (cisplatin) tended to improve progression-free survival compared to placebo in PTEN-expressing, PIK3CA wild-type head and neck squamous cell carcinoma patients (HR=0.54, p=0.0502) by multivariable analysis, but not in PTEN null or PIK3CA mutated patients (HR=0.76, p=0.54) (PMID: 30926065). 30926065
PTEN positive head and neck squamous cell carcinoma predicted - sensitive Cetuximab Clinical Study - Cohort Actionable In a clinical study, high PTEN expression was associated with improved progression-free survival (HR=0.35, p=0.008) in head and neck squamous cell carcinoma patients treated with Erbitux (cetuximab) with or without Nexavar (sorafenib) (PMID: 30926065). 30926065
PTEN inact mut triple-receptor negative breast cancer predicted - sensitive Capivasertib + Paclitaxel Phase II Actionable In a Phase II trial (PAKT), addition of Truqap (capivasertib) to Taxol (paclitaxel) as first-line therapy significantly improved median progression-free survival (9.3 vs 3.7 months, HR=0.30, p=0.01) and reduced risk (66%, HR=0.34, p=0.04) compared to Taxol (paclitaxel) alone in patients with metastatic triple-negative breast cancer harboring activating mutations in AKT1 (n=1) or PIK3CA (n=17), or inactivating mutations or gene loss in PTEN (n=13) (PMID: 31841354; NCT02423603). 31841354
PTEN loss breast cancer no benefit MK2206 Phase II Actionable In a Phase II trial, MK2206 treatment resulted in no objective response and 1 stable disease in 7 patients with advanced breast cancer harboring PTEN loss (PMID: 31277699; NCT01277757). 31277699
PTEN loss breast cancer no benefit CYH33 Preclinical - Cell culture Actionable In a preclinical study, PTEN loss was not associated with sensitivity to CYH33 in breast cancer cell lines in culture (PMID: 30003928). 30003928
PIK3CA H1047R PTEN loss breast cancer predicted - sensitive CYH33 Preclinical - Cell culture Actionable In a preclinical study, CYH33 inhibited proliferation of a breast cancer cell line harboring PIK3CA H1047R and PTEN loss in culture (PMID: 30003928). 30003928
PIK3CA E542K PTEN loss breast cancer predicted - sensitive CYH33 Preclinical - Cell culture Actionable In a preclinical study, CYH33 inhibited proliferation of a breast cancer cell line harboring PIK3CA E542K and PTEN loss in culture (PMID: 30003928). 30003928
BRAF V600E PTEN loss melanoma sensitive unspecified PD-1 antibody + VE800 Preclinical Actionable In a preclinical study, PD-1 antibody treatment supplemented with VE800 inhibited tumor growth in a transgenic mouse model of melanoma harboring PTEN loss and BRAF V600E (PMID: 30675064). 30675064
PTEN L108R breast cancer sensitive Borussertib Preclinical - Cell culture Actionable In a preclinical study, borussertib inhibited AKT signaling and proliferation and increased apoptosis in a breast cancer cell line harboring PTEN L108R in culture (PMID: 30858154). 30858154
PTEN L108R breast cancer sensitive Miransertib Preclinical - Cell culture Actionable In a preclinical study, Miransertib (ARQ092) inhibited proliferation of a breast cancer cell line harboring PTEN L108R in culture (PMID: 30858154). 30858154
PTEN L108R breast cancer sensitive Miransertib Preclinical - Cell culture Actionable In a preclinical study, Miransertib (ARQ092) treatment increased cell death and inhibited proliferation of a breast cancer cell line harboring PTEN L108R in culture (PMID: 32439931). 32439931
PTEN L108R breast cancer sensitive Capivasertib Preclinical - Cell culture Actionable In a preclinical study, Truqap (capivasertib) inhibited proliferation of a breast cancer cell line harboring PTEN L108R in culture (PMID: 30858154). 30858154
PTEN L108R breast cancer sensitive MK2206 Preclinical - Cell culture Actionable In a preclinical study, MK-2206 inhibited proliferation of a breast cancer cell line harboring PTEN L108R in culture (PMID: 30858154). 30858154
PTEN L108R breast cancer sensitive MK2206 Preclinical - Cell culture Actionable In a preclinical study, MK2206 treatment increased cell death and inhibited proliferation of a breast cancer cell line harboring PTEN L108R in culture (PMID: 32439931). 32439931
PTEN L108R breast cancer sensitive Ipatasertib Preclinical - Cell culture Actionable In a preclinical study, Ipatasertib (GDC-0068) inhibited proliferation of a breast cancer cell line harboring PTEN L108R in culture (PMID: 30858154). 30858154
PTEN L108R breast cancer sensitive Ipatasertib Preclinical - Cell culture Actionable In a preclinical study, Ipatasertib (GDC-0068) treatment increased cell death and inhibited proliferation of a breast cancer cell line harboring PTEN L108R in culture (PMID: 32439931). 32439931
PTEN negative renal cell carcinoma predicted - sensitive Temsirolimus Clinical Study - Cohort Actionable In a clinical study, negative PTEN IHC staining were detected more frequently in patients with renal cell carcinoma who responded to treatment with Afinitor (everolimus) or Torisel (temsirolimus) (odds ratio = 0.24, p=0.029) than those who did not respond (PMID: 31335987). 31335987
PTEN negative renal cell carcinoma predicted - sensitive Everolimus Clinical Study - Cohort Actionable In a clinical study, negative PTEN IHC staining were detected more frequently in patients with renal cell carcinoma who responded to treatment with Afinitor (everolimus) or Torisel (temsirolimus) (odds ratio = 0.24, p=0.029) than those who did not respond (PMID: 31335987). 31335987
PTEN inact mut lung non-small cell carcinoma predicted - resistant Osimertinib Case Reports/Case Series Actionable In a retrospective analysis, inactivating PTEN mutations were identified in 3 of 100 patients with non-small cell lung cancer at treatment discontinuation of Tagrisso (osimertinib) (PMID: 31839416). 31839416
PTEN negative prostate cancer not predictive Niraparib Phase I Actionable In a Phase I trial, negative PTEN IHC staining was not associated with response to Zejula (niraparib) treatment in patients with castration-resistant prostate cancer (PMID: 23810788; NCT00749502). 23810788
PTEN loss osteosarcoma sensitive MK2206 Preclinical - Pdx Actionable In a preclinical study, a patient derived xenograft (PDX) model of osteosarcoma with biallelic loss of PTEN was sensitive to treatment with MK2206, demonstrating reduced tumor growth and increased apoptotic activity (PMID: 30266815). 30266815
PTEN loss osteosarcoma sensitive Sirolimus Preclinical - Pdx Actionable In a preclinical study, a patient derived xenograft (PDX) model of osteosarcoma with biallelic PTEN loss was sensitive to treatment with Rapamune (sirolimus), demonstrating reduced tumor growth and increased apoptotic activity (PMID: 30266815). 30266815
BRAF V600E PTEN Y27C melanoma predicted - resistant Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment in a metastatic melanoma patient harboring BRAF V600E and PTEN Y27C resulted in disease progression after 2.3 months following an initial partial response (PMID: 31980996). 31980996
BRAF V600E PTEN del melanoma predicted - resistant Dabrafenib Case Reports/Case Series Actionable In a clinical case study, Tafinlar (dabrafenib) treatment in a metastatic melanoma patient harboring BRAF V600E and PTEN deletion resulted in disease progression after 2.8 months following an initial partial response (PMID: 31980996). 31980996
PTEN del glioblastoma no benefit Buparlisib + Capmatinib Phase Ib/II Actionable In a Phase Ib/II trial, combination of Buparlisib (BKM120) and Tabrecta (capmatinib) did not reach target exposure due to potential drug-drug interaction, and demonstrated minimal activity in patients with glioblastoma harboring PTEN alterations including deletion, mutation, or negative protein expression, therefore, Phase II of the trial was not initiated (PMID: 31776899; NCT01870726). 31776899
PTEN mutant glioblastoma no benefit Buparlisib + Capmatinib Phase Ib/II Actionable In a Phase Ib/II trial, combination of Buparlisib (BKM120) and Tabrecta (capmatinib) did not reach target exposure due to potential drug-drug interaction, and demonstrated minimal activity in patients with glioblastoma harboring PTEN alterations including deletion, mutation, or negative protein expression, therefore, Phase II of the trial was not initiated (PMID: 31776899; NCT01870726). 31776899
PTEN negative glioblastoma no benefit Buparlisib + Capmatinib Phase Ib/II Actionable In a Phase Ib/II trial, combination of Buparlisib (BKM120) and Tabrecta (capmatinib) did not reach target exposure due to potential drug-drug interaction, and demonstrated minimal activity in patients with glioblastoma harboring PTEN alterations including deletion, mutation, or negative protein expression, therefore, Phase II of the trial was not initiated (PMID: 31776899; NCT01870726). 31776899
PTEN loss breast cancer decreased response Palbociclib Preclinical - Cell culture Actionable In a preclinical study, two PTEN-deficient breast cancer cell lines demonstrated a decreased response to treatment with Ibrance (palbociclib) compared to control cells in culture (PMID: 31594766). 31594766
PTEN loss breast cancer predicted - resistant Ribociclib Preclinical - Cell culture Actionable In a preclinical study, treatment with Kisqali (ribociclib) resulted in a decreased response in two PTEN-deficient breast cancer cell lines in culture compared to control cells and resistance in a cell-line xenograft model (PMID: 31594766). 31594766
PTEN loss breast cancer resistant Fulvestrant + Ribociclib Preclinical - Cell culture Actionable In a preclinical study, PTEN-deficient breast cancer cells demonstrated resistance to the combination of Kisqali (ribociclib) and Faslodex (fulvestrant) in culture (PMID: 31594766). 31594766
PTEN loss breast cancer sensitive MK2206 + Ribociclib Preclinical - Cell culture Actionable In a preclinical study, the combination treatment of MK2206 and Kisqali (ribociclib) induced cell cycle arrest and inhibited cell proliferation of PTEN-deficient breast cancer cells in culture and led to tumor regression in cell-line xenograft models (PMID: 31594766). 31594766
PTEN loss breast cancer sensitive Ipatasertib + Ribociclib Preclinical - Cell culture Actionable In a preclinical study, the combination of Ipatasertib (GDC-0068) and Kisqali (ribociclib) induced cell cycle arrest and inhibited growth of PTEN-deficient breast cancer cells in culture (PMID: 31594766). 31594766
PTEN loss breast cancer sensitive MK2206 + Palbociclib Preclinical - Cell culture Actionable In a preclinical study, the combination of Ibrance (palbociclib) and MK2206 inhibited growth of PTEN-deficient breast cancer cells in culture (PMID: 31594766). 31594766
PTEN loss breast cancer sensitive Ipatasertib + Palbociclib Preclinical - Cell culture Actionable In a preclinical study, the combination of Ibrance (palbociclib) and Ipatasertib (GDC-0068) inhibited growth of PTEN-deficient breast cancer cells in culture (PMID: 31594766). 31594766
PTEN loss breast cancer sensitive Fulvestrant + MK2206 + Ribociclib Preclinical - Cell culture Actionable In a preclinical study, the combination of Kisqali (ribociclib), Faslodex (fulvestrant), and MK2206 inhibited growth of PTEN-deficient breast cancer cells in culture (PMID: 31594766). 31594766
BRAF V600E PIK3CA H1047R PTEN Y68fs melanoma predicted - resistant Vemurafenib Case Reports/Case Series Actionable In a clinical study, a melanoma patient harboring BRAF V600E and PTEN Y68fs treated with Zelboraf (vemurafenib) was found to have acquired PIK3CA H1047R in the post-progression tumor biopsy (PMID: 24265153). 24265153
PTEN loss lung squamous cell carcinoma no benefit Capivasertib Case Reports/Case Series Actionable In a Phase II trial (NLMT), Truqap (capivasertib) treatment did not result in a confirmed response (0/4) or durable clinical benefit (0/2) in patients with squamous cell lung cancer harboring PTEN loss, thus the cohort was closed due to futility (PMID: 32669708, NCT02664935). 32669708
PTEN mutant lung adenocarcinoma no benefit Capivasertib Case Reports/Case Series Actionable In a Phase II trial (NLMT), Truqap (capivasertib) treatment did not result in a confirmed response (0/8) or durable clinical benefit (0/8) in patients with lung adenocarcinoma harboring mutations in PIK3CA, PTEN, or AKT, thus the cohort was closed due to futility (PMID: 32669708, NCT02664935). 32669708
PTEN dec exp triple-receptor negative breast cancer sensitive Docetaxel + Ipatasertib Preclinical - Pdx Actionable In a preclinical study, the combination of Ipatasertib (GDC-0068) and Taxotere (docetaxel) inhibited tumor growth in a patient-derived xenograft (PDX) model of triple-negative breast cancer with low PTEN expression, and demonstrated increased activity over either agent alone (PMID: 32205017). 32205017
PTEN dec exp triple-receptor negative breast cancer sensitive Ipatasertib Preclinical - Pdx Actionable In a preclinical study, Ipatasertib (GDC-0068) reduced tumor growth in a patient-derived xenograft (PDX) model of triple-negative breast cancer with low PTEN expression (PMID: 32205017). 32205017
PTEN loss stomach cancer sensitive Fluorouracil + Ipatasertib + Leucovorin + Oxaliplatin Preclinical - Pdx Actionable In a preclinical study, the combination of Ipatasertib (GDC-0068) and FOLFOX inhibited tumor growth in a patient-derived xenograft (PDX) model of ERBB2 (HER2)-negative gastric cancer harboring PTEN loss, and demonstrated increased activity over either agent alone (PMID: 32205017). 32205017
PTEN loss stomach cancer sensitive Ipatasertib Preclinical - Pdx Actionable In a preclinical study, Ipatasertib (GDC-0068) demonstrated activity against tumor growth in patient-derived xenograft (PDX) models of ERBB2 (HER2)-negative stomach cancer harboring PTEN loss (PMID: 32205017). 32205017
PTEN loss triple-receptor negative breast cancer predicted - sensitive Capivasertib + Paclitaxel Phase II Actionable In a Phase II trial (PAKT), addition of Truqap (capivasertib) to Taxol (paclitaxel) as first-line therapy significantly improved median progression-free survival (9.3 vs 3.7 months, HR=0.30, p=0.01) and reduced risk (66%, HR=0.34, p=0.04) compared to Taxol (paclitaxel) alone in patients with metastatic triple-negative breast cancer harboring activating mutations in AKT1 (n=1) or PIK3CA (n=17), or inactivating mutations or gene loss in PTEN (n=13) (PMID: 31841354; NCT02423603). 31841354
PTEN del prostate cancer not predictive PX-866 Phase II Actionable In a Phase II trial, Sonolisib (PX-866) treatment resulted in stable disease as best response in 66.7% (2/3) of patients with recurrent or metastatic castration-resistant prostate cancer harboring homozygous PTEN deletion, while in PTEN wild-type patients resulted in a partial response in 14.3% (2/14) and stable disease in 28.6% (4/14) of patients (PMID: 31056399). 31056399
PTEN LOH transitional cell carcinoma predicted - sensitive Buparlisib Case Reports/Case Series Actionable In a Phase II trial, Buparlisib (BKM120) treatment resulted in stable disease in a patient with metastatic urothelial carcinoma harboring PTEN loss of heterozygosity (PMID: 32767682; NCT01551030). 32767682
PTEN R130* Her2-receptor negative breast cancer predicted - resistant Alpelisib + Letrozole Case Reports/Case Series Actionable In a Phase I/II trial, combination of Piqray (Alpelisib) and Femara (letrozole) resulted in no clinical benefit in a patient with hormone receptor positive, ERBB2 (HER2)-negative breast cancer harboring PTEN R130* (PMID: 32864625; NCT01870505). 32864625
PTEN L152P PTEN neg salivary gland carcinoma resistant AZD8186 Preclinical - Cell culture Actionable In a preclinical study, salivary ductal carcinoma cells harboring PTEN L152P and loss of Pten protein expression were resistant to AZD8186-induced inhibition of Akt phosphorylation and proliferation in culture (PMID: 30289966). 30289966
PTEN L152P PTEN neg salivary gland carcinoma resistant GSK2636771 Preclinical - Cell culture Actionable In a preclinical study, salivary ductal carcinoma cells harboring PTEN L152P and loss of Pten protein expression were resistant to GSK2636771-induced inhibition of proliferation in culture (PMID: 30289966). 30289966
PTEN L152P PTEN neg salivary gland carcinoma resistant Alpelisib Preclinical - Cell culture Actionable In a preclinical study, salivary ductal carcinoma cells harboring PTEN L152P and loss of Pten protein expression were resistant to Piqray (Alpelisib)-induced inhibition of Akt phosphorylation and proliferation in culture (PMID: 30289966). 30289966
PTEN L152P PTEN neg salivary gland carcinoma sensitive Alpelisib + AZD8186 Preclinical - Cell culture Actionable In a preclinical study, combination of Piqray (Alpelisib) and AZD8186 inhibited Akt phosphorylation and proliferation of salivary ductal carcinoma cells harboring PTEN L152P and loss of Pten protein expression in culture (PMID: 30289966). 30289966
PTEN L152P PTEN neg salivary gland carcinoma sensitive Pictilisib Preclinical - Cell culture Actionable In a preclinical study, Pictilisib (GDC-0941) inhibited Akt phosphorylation and proliferation of salivary ductal carcinoma cells harboring PTEN L152P and loss of Pten protein expression in culture (PMID: 30289966). 30289966
PTEN dec exp gastroesophageal cancer no benefit Fluorouracil + Ipatasertib + Leucovorin + Oxaliplatin Phase II Actionable In a Phase II trial, the combination of Ipatasertib (GDC-0068) and mFOLFOX6 did not improve median progression-free survival (7.1 vs 7.4 months; HR=1.07, p=0.86) compared to placebo plus mFOLFOX6 in patients with advanced gastroesophageal junction or gastric cancer harboring decreased PTEN expression (IHC=0 in >10% of tumor cells) (PMID: 30592991; NCT01896531). 30592991
PTEN dec exp stomach cancer no benefit Fluorouracil + Ipatasertib + Leucovorin + Oxaliplatin Phase II Actionable In a Phase II trial, the combination of Ipatasertib (GDC-0068) and mFOLFOX6 did not improve median progression-free survival (7.1 vs 7.4 months; HR=1.07, p=0.86) compared to placebo plus mFOLFOX6 in patients with advanced gastric or gastroesophageal junction cancer harboring decreased PTEN expression (IHC=0 in >10% of tumor cells) (PMID: 30592991; NCT01896531). 30592991
PTEN loss glioblastoma predicted - sensitive GDC-0152 Preclinical - Cell culture Actionable In a preclinical study, GDC-0152 treatment inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss glioblastoma predicted - sensitive Birinapant Preclinical - Cell culture Actionable In a preclinical study, TL32711 (birinapant) treatment inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss glioblastoma sensitive UMI-77 Preclinical - Cell culture Actionable In a preclinical study, UMI-77 treatment led to enhanced apoptosis, reduced Akt phosphorylation, and inhibited proliferation, colony formation, spheroid formation, and neurosphere formation in PTEN-deficient glioblastoma cell lines in culture (PMID: 32737157). 32737157
PTEN loss glioblastoma sensitive A-1210477 Preclinical - Cell culture Actionable In a preclinical study, A-1210477 treatment reduced Akt phosphorylation and inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss glioblastoma sensitive S63845 Preclinical - Cell culture Actionable In a preclinical study, S63845 treatment reduced Akt phosphorylation and inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss glioblastoma sensitive AZD5991 Preclinical - Cell culture Actionable In a preclinical study, AZD5991 treatment inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss glioblastoma sensitive Temozolomide + UMI-77 Preclinical - Cell culture Actionable In a preclinical study, UMI-77 in combination with Temodar (temozolomide) enhanced cytotoxicity and apoptosis, and synergistically inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss glioblastoma sensitive AZD5991 + Temozolomide Preclinical - Cell culture Actionable In a preclinical study, AZD5991 in combination with Temodar (temozolomide) synergistically inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN mutant skin melanoma not applicable N/A Guideline Risk Factor Germline PTEN mutations or polymorphisms are associated with increased risk of developing single or multiple primary cutaneous melanomas (NCCN.org). detail...
PIK3CA E542K PTEN loss breast cancer sensitive Taselisib Preclinical - Cell culture Actionable In a preclinical study, a breast cancer cell line harboring PIK3CA E542K was sensitive to treatment with Taselisib (GDC-0032) in culture, demonstrating decreased cell viability (PMID: 31534012). 31534012
PIK3CA H1047R PTEN loss breast cancer sensitive Taselisib Preclinical - Cell culture Actionable In a preclinical study, a breast cancer cell line harboring a PIK3CA H1047R and PTEN loss was sensitive to treatment with Taselisib (GDC-0032) in culture, demonstrating decreased cell viability (PMID: 31534012). 31534012
PTEN negative endometrial carcinoma no benefit Olaparib Preclinical - Cell culture Actionable In a preclinical study, Lynparza (olaparib) treatment did not significantly increase DNA damage or inhibit growth of PTEN-deficient endometrial carcinoma cell lines in culture (PMID: 28945226). 28945226
PTEN negative endometrial carcinoma sensitive Buparlisib Preclinical - Cell culture Actionable In a preclinical study, Buparlisib (BKM120) inhibited Akt signaling, induced DNA damage, and inhibited the growth of PTEN-deficient endometrial carcinoma cell lines in culture (PMID: 28945226). 28945226
PTEN negative endometrial carcinoma sensitive Buparlisib + Olaparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Buparlisib (BKM120) and Lynparza (olaparib) resulted in enhanced DNA damage and growth inhibition in PTEN-deficient endometrial carcinoma cell lines in culture (PMID: 28945226). 28945226
PTEN del endometrial carcinoma no benefit Olaparib Preclinical - Cell culture Actionable In a preclinical study, Lynparza (olaparib) treatment did not significantly increase DNA damage or inhibit growth of an endometrial carcinoma cell line with PTEN knocked out via CRISPR/Cas9 system in culture (PMID: 28945226). 28945226
PTEN del endometrial carcinoma sensitive Buparlisib Preclinical - Cell culture Actionable In a preclinical study, Buparlisib (BKM120) inhibited Akt signaling, induced DNA damage, and inhibited the growth of an endometrial carcinoma cell line with PTEN knocked out via CRISPR/Cas9 system in culture (PMID: 28945226). 28945226
PTEN del endometrial carcinoma sensitive Buparlisib + Olaparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Buparlisib (BKM120) and Lynparza (olaparib) resulted in enhanced DNA damage and growth inhibition in an endometrial carcinoma cell line with PTEN knocked out via CRISPR/Cas9 system in culture (PMID: 28945226). 28945226
PTEN inact mut triple-receptor negative breast cancer no benefit Ipatasertib + Paclitaxel Phase II Actionable In a Phase II trial (LOTUS), Ipatasertib (GDC-0068) in combination with Abraxane (paclitaxel) resulted a median progression free survival of 6.2 vs 4.9 months with placebo in triple-receptor negative breast cancer patients harboring activating mutations in PIK3CA or AKT1, or inactivating mutations in PTEN (PMID: 28800861; NCT02162719). 28800861
PTEN inact mut triple-receptor negative breast cancer no benefit Ipatasertib + Paclitaxel Phase III Actionable In a Phase III trial (IPATunity 130), the addition of Ipatasertib (GDC-0068) to treatment with Abraxane (paclitaxel) did not result in improved progression-free survival in patients with triple-negative breast cancer harboring PIK3CA and/or AKT activating mutations or PTEN alterations, with a median PFS of 7.4 months with Ipatasertib (GDC-0068) plus Abraxane (paclitaxel) vs. 6.1 months with placebo plus Abraxane (paclitaxel) (SABCS 2020, Abstract GS3-04; NCT03337724). detail...
PTEN inact mut endometrial cancer no benefit LY3023414 Phase II Actionable In a Phase II trial, patients with advanced endometrial cancer harboring a PI3K pathway mutation, including PTEN inactivating mutations, demonstrated only a modest clinical benefit with an overall response rate of 16% (4/25), a clinical benefit rate of 28% (7/25) at 12 weeks, a progression-free survival of 2.5 months, and overall survival of 9.2 months when treated with LY3023414 (PMID: 31880826; NCT01775072). 31880826
PTEN del prostate cancer sensitive RER Preclinical Actionable In a preclinical study, RER treatment inhibited Tgf-beta and Akt pathways signaling, led to inhibition of tumor cell proliferation and tumorigenesis in a PTEN knock-out mouse model of prostate cancer (PMID: 27863384). 27863384
PTEN loss prostate cancer predicted - sensitive Carboplatin + Paclitaxel + Sapanisertib Case Reports/Case Series Actionable In a Phase I trial, combination treatment with Paraplatin (carboplatin), Taxol (paclitaxel), and Sapanisertib (MLN0128) led to a partial response in a castrate resistant prostate cancer harboring PTEN loss (Cancer Res 2021;81(13_Suppl):Abstract nr CT109; NCT03430882). detail...
PTEN L108R breast cancer sensitive ARQ 751 Preclinical - Cell culture Actionable In a preclinical study, ARQ 751 treatment increased cell death and inhibited proliferation of a breast cancer cell line harboring PTEN L108R in culture (PMID: 32439931). 32439931
PTEN L108R breast cancer predicted - sensitive GSK690693 Preclinical - Cell culture Actionable In a preclinical study, GSK690693 treatment did not increase cell death but inhibited proliferation of a breast cancer cell line harboring PTEN L108R in culture (PMID: 32439931). 32439931
PTEN loss castration-resistant prostate carcinoma no benefit Enzalutamide + GSK2636771 Phase I Actionable In a Phase I trial, combination GSK2636771 and Xtandi (enzalutamide) was safe and tolerable in patients with castration-resistant prostate cancer with PTEN deficiency, but resulted in limited antitumor activity in patients who had previously progressed on Xtandi (enzalutamide), with a 12-week non-progressive disease (non-PD) rate of 50% and a best response of 1 partial response, stable disease in 33% (12/36), non-complete response/non-PD in 22% (8/36), and PD in 28% (10/36) (PMID: 34281912; NCT02215096). 34281912
PTEN inact mut castration-resistant prostate carcinoma predicted - sensitive CC-115 + Enzalutamide Phase I Actionable In a Phase Ib trial, Xtandi (enzalutamide) plus CC-115 was safe and led to a PSA reduction >= 50% (PSA50) in 80% (32/40) and >=90% (PSA90) in 58% (23/40) of patients with metastatic castration-resistant prostate cancer at 12 weeks, and patients harboring PTEN mutation or deletion (n=11) achieved PSA50 of 91%, PSA90 of 55%, and median radiographic progression-free survival was not reached, compared to 75%, 55%, and 19.6 mo, respectively, in PTEN wild-type patients (n=20) (PMID: 37980367; NCT02833883). 37980367
PTEN loss glioblastoma predicted - sensitive Atezolizumab + Ipatasertib Phase I Actionable In a Phase I trial (Ice-CAP), the combination of Ipatasertib (GDC-0068) and Tecentriq (atezolizumab) resulted in a clinical benefit rate (CBR) of 32% (6/19) in patients with glioblastoma, and CBR was 28.6% (4/14) in patients with PTEN loss as determined by IHC (H<30) (Cancer Res (2023) 83 (8_Supplement): CT093; NCT03673787). detail...
PTEN inact mut glioblastoma predicted - sensitive Atezolizumab + Ipatasertib Phase I Actionable In a Phase I trial (Ice-CAP), combination treatment with Ipatasertib (GDC-0068) and Tecentriq (atezolizumab) demonstrated safety and tolerability in glioblastoma patients harboring PTEN loss or PTEN mutations, and led to a clinical benefit rate of 29% (2/7), with 1 pathological complete response, and stable disease in 1 patient of 7 patients (Cancer Res 2021;81(13_Suppl):Abstract nr CT120; NCT03673787). detail...
PTEN N323fs castration-resistant prostate carcinoma predicted - sensitive Everolimus Case Reports/Case Series Actionable In a clinical case study, Afinitor (everolimus) treatment resulted in stable disease for eight months in a patient with castration-resistant prostate cancer harboring PTEN N323fs*21 who had previously progressed on several lines of therapy (PMID: 32399016). 32399016
PTEN mutant gastrointestinal system cancer decreased response Pembrolizumab Clinical Study - Cohort Actionable In a retrospective analysis, patients with MSI high, dMMR gastrointestinal tumors including gastric (n=18), colorectal (n=17), cholangiocarcinoma (n=5), small intestine (n=2), pancreatic (n=2), and duodenal cancer (n=1) harboring PTEN mutations demonstrated a decreased objective response rate (21.4 vs 54.8%), overall survival (15.2 vs 25.7 mo), and progression-free survival (4.3 vs 15.6 mo) compared to PTEN-wild-type patients when treated with Keytruda (pembrolizumab) or Opdivo (nivolumab) (PMID: 33926917). 33926917
PTEN mutant gastrointestinal system cancer decreased response Nivolumab Clinical Study - Cohort Actionable In a retrospective analysis, patients with MSI high, dMMR gastrointestinal tumors including gastric (n=18), colorectal (n=17), cholangiocarcinoma (n=5), small intestine (n=2), pancreatic (n=2), and duodenal cancer (n=1) harboring PTEN mutations demonstrated a decreased objective response rate (21.4 vs 54.8%), overall survival (15.2 vs 25.7 mo), and progression-free survival (4.3 vs 15.6 mo) compared to PTEN-wild-type patients when treated with Keytruda (pembrolizumab) or Opdivo (nivolumab) (PMID: 33926917). 33926917
PTEN loss stomach cancer no benefit GSK2636771 + Paclitaxel Phase II Actionable In a Phase II trial (K-Umbrella), GSK2636771 and Taxol (paclitaxel) combination therapy did not significantly improve median progression-free survival (2.8 vs 4.0 months) or median overall survival (7.0 vs 8.7 months) in patients with advanced ERBB2 (HER2)-negative gastric cancer harboring PTEN loss compared to standard of care in the control group (PMID: 37883723; NCT02951091). 37883723
PTEN inact mut head and neck cancer predicted - sensitive RMC-5552 Case Reports/Case Series Actionable In a Phase I/Ib trial, RMC-5552 treatment resulted in an objective response rate of 20% (1/5) and 3 stable disease in patients with advanced solid tumors, with 1 partial response observed in a patient with head and neck cancer harboring a pathogenic PTEN mutation (J of Clin Oncol40, no. 16_suppl (June 01, 2022) 3098; NCT04774952). detail...
PTEN loss clear cell renal cell carcinoma sensitive Everolimus Preclinical - Cell culture Actionable In a preclinical study, Afinitor (everolimus) inhibited cell proliferation in PTEN-deficient clear cell renal carcinoma cell lines in culture (PMID: 35165399). 35165399
PTEN loss clear cell renal cell carcinoma sensitive Temsirolimus Preclinical - Cell line xenograft Actionable In a preclinical study, Torisel (temsirolimus) inhibited cell proliferation and induced cell cycle arrest in PTEN-deficient clear cell renal carcinoma cell lines in culture and inhibited tumor growth in a cell line xenograft model (PMID: 35165399). 35165399
PTEN Y336* breast cancer sensitive Talazoparib Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells expressing PTEN Y336* demonstrated increased sensitivity to Talzenna (talazoparib) compared to cells expressing wild-type Pten in culture (PMID: 25946202}. 25946202
PTEN inact mut breast cancer predicted - sensitive TAS-117 Case Reports/Case Series Actionable In a Phase II trial, TAS-117 treatment demonstrated tolerability in patients with advanced solid tumors harboring germline inactivating PTEN mutations, and resulted in an unconfirmed partial response in one patient with breast cancer and target tumor lesion shrinkage of approximately 15% and stable disease lasting more than 8 months in another breast cancer patient (Ann Oncol (2022) 33 (suppl_7): S754-S755; NCT04770246). detail...
PIK3CA E542K PTEN loss lung squamous cell carcinoma sensitive Buparlisib Preclinical - Pdx Actionable In a preclinical study, Buparlisib (BKM120) treatment resulted in inhibition of AKT and S6 phosphorylation and durable response in a lung squamous cell carcinoma patient-derived xenograft (PDX) model harboring PIK3CA E542K and PTEN loss (PMID: 30093452). 30093452
PTEN Y174H PTEN K263* colorectal cancer predicted - sensitive Olaparib Preclinical - Patient cell culture Actionable In a preclinical study, Lynparza (olaparib) inhibited cell proliferation in a patient-derived colorectal cancer organoid harboring PTEN Y174H and K263*, along with PIK3CA N1068fs, in culture (PMID: 32376656). 32376656
PTEN Y174H PTEN K263* colorectal cancer predicted - sensitive Rucaparib Preclinical - Patient cell culture Actionable In a preclinical study, Rubraca (rucaparib) inhibited cell proliferation in a patient-derived colorectal cancer organoids harboring PTEN Y174H and K263*, along with PIK3CA N1068fs, in culture (PMID: 32376656). 32376656
PIK3CA N1068Kfs*5 PTEN Y174H PTEN K263* colorectal cancer predicted - sensitive Alpelisib Preclinical - Patient cell culture Actionable In a preclinical study, Piqray (alpelisib) inhibited cell proliferation in a patient-derived colorectal cancer organoid harboring PIK3CA N1068Kfs*5, PTEN Y174H, and PTEN K263* in culture (PMID: 32376656). 32376656
PIK3CA N1068Kfs*5 PTEN Y174H PTEN K263* colorectal cancer predicted - sensitive Taselisib Preclinical - Patient cell culture Actionable In a preclinical study, Taselisib (GDC-0032) inhibited cell proliferation in a patient-derived colorectal cancer organoid harboring PIK3CA N1068Kfs*5, PTEN Y174H, and PTEN K263* in culture (PMID: 32376656). 32376656
HRAS Q61K PTEN dec exp rhabdomyosarcoma resistant Trametinib Preclinical - Cell culture Actionable In a preclinical study, decreasing Pten expression through shRNA knockdown in a rhabdomyosarcoma cell line harboring HRAS Q61K conferred resistance to Mekinist (trametinib) treatment in culture (PMID: 36322002). 36322002
HRAS Q61K PTEN dec exp rhabdomyosarcoma sensitive Ganitumab + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the addition of Ganitumab (AMG-479) sensitized a rhabdomyosarcoma cell line harboring HRAS Q61K with low PTEN expression to treatment with Mekinist (trametinib), resulting in reduced cell growth in culture (PMID: 36322002). 36322002
NRAS Q61H PTEN over exp rhabdomyosarcoma sensitive Ganitumab + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the addition of Ganitumab (AMG-479) sensitized a rhabdomyosarcoma cell line harboring NRAS Q61H and overexpressing PTEN to treatment with Mekinist (trametinib), resulting in reduced cell growth in culture (PMID: 36322002). 36322002
BRAF V600E PTEN loss melanoma predicted - sensitive Dabrafenib + Enzalutamide + Trametinib Preclinical Actionable In a preclinical study, addition of Xtandi (enzalutamide) to Tafinlar (dabrafenib) and Mekinist (trametinib) combination therapy improved tumor control and led to a greater reduction in tumor volume in an allograft mouse model of melanoma harboring BRAF V600E and PTEN loss (PMID: 35705814). 35705814
PTEN dec exp triple-receptor negative breast cancer predicted - sensitive Alpelisib + Everolimus Preclinical - Pdx & cell culture Actionable In a preclinical study, combination treatment with Piqray (albelisib) and Afinitor (everolimus) synergistically inhibited cell viability of PTEN-deficient triple-negative breast cancer patient-derived cells in culture, and led to inhibition of tumor growth in patient-derived xenograft (PDX) models (PMID: 36900375). 36900375
PIK3CA amp PTEN H123P triple-receptor negative breast cancer predicted - sensitive Alpelisib + Everolimus Preclinical - Pdx & cell culture Actionable In a preclinical study, combination treatment with Piqray (albelisib) and Afinitor (everolimus) synergistically inhibited cell viability of patient-derived triple-negative breast cancer cells harboring PIK3CA amplification and PTEN H123P in culture, and led to inhibition of tumor growth in a patient-derived xenograft (PDX) model (PMID: 36900375). 36900375
PIK3CA amp PTEN H123P triple-receptor negative breast cancer predicted - sensitive Afatinib + Alpelisib Preclinical - Pdx & cell culture Actionable In a preclinical study, combination treatment with Piqray (albelisib) and Gilotrif (afatinib) synergistically inhibited cell viability of patient-derived triple-negative breast cancer cells harboring PIK3CA amplification and PTEN H123P in culture, and led to inhibition of tumor growth in a patient-derived xenograft (PDX) model (PMID: 36900375). 36900375
BRAF V600E PTEN N329fs lung adenocarcinoma predicted - resistant Dabrafenib Case Reports/Case Series Actionable In a clinical case study, PTEN N329fs was identified on the post-progression biopsy in a patient with metastatic lung adenocarcinoma harboring BRAF V600E, who previously responded to Tafinlar (dabrafenib) (PMID: 32388065). 32388065
PTEN inact mut Advanced Solid Tumor predicted - sensitive Temsirolimus Phase II Actionable In a Phase II trial (TAPUR), Torisel (temsirolimus) treatment resulted in a disease control rate of 26% (7/27, 2 partial responses and 5 with stable disease >=16 weeks) and an objective response rate of 7% (2/27) in patients with advanced solid tumors harboring inactivating PTEN mutations, with a median progression-free survival of 10 weeks and a median overall survival of 32 weeks (Cancer Res (2023) 83 (8_Supplement): CT231; NCT02693535). detail...
PTEN loss lung squamous cell carcinoma predicted - resistant unspecified PD-1 antibody Preclinical Actionable In a preclinical study, a syngeneic mouse model of squamous cell lung cancer with PTEN loss demonstrated resistance to treatment with a PD-1 antibody (PMID: 37311042). 37311042
FGFR3 S249C FGFR3 amp PTEN C136fs transitional cell carcinoma predicted - resistant Erdafitinib Case Reports/Case Series Actionable In a clinical case study, PTEN C136fs and FGFR3 amplification (6 copies) were identified in the tissue biopsy of a patient with bladder urothelial cancer harboring FGFR3 S249C who progressed on Balversa (erdafitinib) after 1.4 months of treatment (PMID: 37377403). 37377403
PTEN inact mut breast cancer predicted - sensitive AZD5991 + AZD8186 Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of AZD5991 and AZD8186 synergistically inhibited proliferation and induced apoptosis in a triple-negative breast cancer cell line and hormone receptor-positive breast cancer cell line each harboring a PTEN inactivating mutation in culture and inhibited tumor growth in cell line xenograft models (PMID: 36241868). 36241868
PTEN inact mut breast cancer predicted - sensitive AZD5991 + Capivasertib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of AZD5991 and Truqap (capivasertib) synergistically inhibited proliferation and induced apoptosis in a triple-negative breast cancer cell line and hormone-receptor positive breast cancer cell line each harboring a PTEN inactivating mutation in culture and inhibited tumor growth in cell line xenograft models (PMID: 36241868). 36241868
PTEN E43fs choroid plexus carcinoma predicted - sensitive Everolimus Case Reports/Case Series Actionable In a clinical case study, adjuvant Afinitor (everolimus) treatment after surgery and radiotherapy resulted in stable disease in a patient with choroid plexus carcinoma harboring PTEN E43fs, who completed 12 months of therapy and remained in remission at 60 months after initial diagnosis (PMID: 37535883). 37535883
PTEN del Advanced Solid Tumor no benefit Atezolizumab + Ipatasertib Phase II Actionable In a Phase II trial (CRAFT), treatment with Ipatasertib (GDC-0068) plus Tecentriq (atezolizumab) demonstrated safety but limited clinical benefit in advanced solid tumor patients harboring PI3K-AKT pathway mutations (n=13), including PTEN deletion/inactivating mutations or activating mutations in PIK3CA or AKT1, with a partial response in a breast cancer patient with AKT1 E17K and stable disease in a prostate cancer patient with PTEN loss (Ann Oncol (2023) 34 (suppl_2): S256-S257;NCT04551521). detail...
PTEN inact mut Advanced Solid Tumor no benefit Atezolizumab + Ipatasertib Phase II Actionable In a Phase II trial (CRAFT), treatment with Ipatasertib (GDC-0068) plus Tecentriq (atezolizumab) demonstrated safety but limited clinical benefit in advanced solid tumor patients harboring PI3K-AKT pathway mutations (n=13), including PTEN deletion/inactivating mutations or activating mutations in PIK3CA or AKT1, with a partial response in a breast cancer patient with AKT1 E17K and stable disease in a prostate cancer patient with PTEN loss (Ann Oncol (2023) 34 (suppl_2): S256-S257;NCT04551521). detail...
PTEN inact mut Advanced Solid Tumor predicted - sensitive Copanlisib + Nivolumab Phase II Actionable In a Phase II trial (BaCoN), treatment with the combination of Aliqopa (copanlisib) and Opdivo (nivolumab) was well tolerated in patients with advanced solid tumors harboring an inactivating mutation in PTEN, and resulted in a complete response/partial response (CR/PR) in 20% (3/15, 3 cPR) and a clinical benefit rate (CR or PR or stable disease > 4 months) of 33% (Ann Oncol 34 (2023): S487-S488; NCT04317105). detail...
PTEN K6fs endometrial cancer sensitive TAS0612 Preclinical - Cell line xenograft Actionable In a preclinical study, TAS0612 inhibited proliferation in an endometrial cancer cell line harboring PTEN K6fs along with PIK3CA mutations in culture and induced tumor regression in a cell line xenograft model (PMID: 37906695). 37906695
PTEN L320* endometrial cancer sensitive TAS0612 Preclinical - Cell line xenograft Actionable In a preclinical study, TAS0612 inhibited proliferation in an endometrial cancer cell line harboring PTEN L320* along with ERBB2 (HER2) R896H and FGFR2 S252W in culture and induced tumor regression in a cell line xenograft model (PMID: 37906695). 37906695
PTEN P89fs breast cancer sensitive TAS0612 Preclinical - Cell line xenograft Actionable In a preclinical study, TAS0612 inhibited proliferation in a breast cancer cell line harboring PTEN P89fs in culture and induced tumor regression in a cell line xenograft model (PMID: 37906695). 37906695
PTEN R142fs PTEN L265fs ovarian cancer sensitive TAS0612 Preclinical - Cell line xenograft Actionable In a preclinical study, TAS0612 inhibited proliferation in a clear cell ovarian cancer cell line harboring PTEN R142fs and L265fs, along with KRAS G13C and PIK3CA H1047Y, in culture and induced tumor regression in a cell line xenograft model (PMID: 37906695). 37906695
PTEN del breast cancer sensitive MEN1611 Preclinical - Cell culture Actionable In a preclinical study, MEN1611 (CH5132799) inhibited viability in breast cancer cell lines harboring a PTEN deletion in culture (PMID: 36913051). 36913051
PTEN F90Lfs*90 breast cancer sensitive MEN1611 Preclinical - Cell culture Actionable In a preclinical study, MEN1611 (CH5132799) inhibited viability in a breast cancer cell line harboring PTEN F90Lfs*90 in culture (PMID: 36913051). 36913051
PTEN L108R breast cancer sensitive MEN1611 Preclinical - Cell culture Actionable In a preclinical study, MEN1611 (CH5132799) inhibited viability in a breast cancer cell line harboring PTEN L108R in culture (PMID: 36913051). 36913051
PTEN D92H PTEN F278Lfs*12 breast cancer sensitive MEN1611 Preclinical - Cell culture Actionable In a preclinical study, MEN1611 (CH5132799) inhibited viability in a breast cancer cell line harboring PTEN D92H and F278Lfs*12 in culture (PMID: 36913051). 36913051
PIK3CA P539R PIK3CA H1047R PTEN del breast cancer sensitive MEN1611 Preclinical - Cell culture Actionable In a preclinical study, MEN1611 (CH5132799) inhibited viability in a breast cancer cell line with deletion of PTEN and harboring PIK3CA H1047R and P539R in culture (PMID: 36913051). 36913051
PTEN L194fs thyroid cancer predicted - sensitive Ipilimumab + Nivolumab + Temsirolimus Case Reports/Case Series Actionable In a clinical case study, the addition of Torisel (temsirolimus) to the combination treatment of Yervoy (ipilimumab) and Opdivo (nivolumab) resulted in a partial response with a decrease in target lesion size in a patient with poorly differentiated thyroid cancer harboring PTEN L194fs, along with TP53 F270S, who remained on treatment for 6 months (PMID: 38356955). 38356955
PTEN mutant breast cancer not applicable N/A Guideline Risk Factor Germline PTEN mutations result in Cowden syndrome, which is associated with increased risk of developing breast cancer (NCCN.org). detail...
BRAF V600E PTEN del melanoma decreased response Dabrafenib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, a melanoma cell line harboring BRAF V600E and engineered via CRISPR-Cas9 to harbor a PTEN knockout demonstrated decreased sensitivity to treatment with Tafinlar (dabrafenib) and Mekinist (trametinib) compared to cells with wild-type PTEN in culture (PMID: 38854737). 38854737
BRAF V600E PTEN del melanoma decreased response Binimetinib + Encorafenib Preclinical - Cell culture Actionable In a preclinical study, a melanoma cell line harboring BRAF V600E and engineered via CRISPR-Cas9 to harbor a PTEN knockout demonstrated decreased sensitivity to treatment with Mektovi (binimetinib) and Braftovi (encorafenib) compared to cells with wild-type PTEN in culture (PMID: 38854737). 38854737
BRAF V600E PTEN del melanoma decreased response Cobimetinib + Vemurafenib Preclinical - Cell culture Actionable In a preclinical study, a melanoma cell line harboring BRAF V600E and engineered via CRISPR-Cas9 to harbor a PTEN knockout demonstrated decreased sensitivity to treatment with Cotellic (cobimetinib) and Zelboraf (vemurafenib) compared to cells with wild-type PTEN in culture (PMID: 38854737). 38854737
BRAF V600E PTEN del melanoma sensitive Dabrafenib + Neratinib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the addition of Nerlynx (neratinib) restored sensitivity to Tafinlar (dabrafenib) and Mekinist (trametinib) and led to synergistic inhibition of a melanoma cell line harboring BRAF V600E and engineered via CRISPR-Cas9 to harbor a PTEN knockout in culture (PMID: 38854737). 38854737
BRAF V600E PTEN del melanoma sensitive Afatinib + Dabrafenib + Trametinib Preclinical - Cell culture Actionable In a preclinical study, the addition of Gilotrif (afatinib) restored sensitivity to Tafinlar (dabrafenib) and Mekinist (trametinib) and led to synergistic inhibition of a melanoma cell line harboring BRAF V600E and engineered via CRISPR-Cas9 to harbor a PTEN knockout in culture (PMID: 38854737). 38854737
PTEN inact mut Advanced Solid Tumor no benefit Talazoparib Phase II Actionable In a Phase II trial, Talzenna (talazoparib) treatment resulted in limited clinical benefit in patients with advanced solid tumors harboring PTEN deleterious mutations or loss (by IHC), with a clinical benefit rate of 9.4% (1/14, 1 with stable disease >/=24 weeks) and a median overall survival of 8.5 months and a median progression-free survival of 7.7 weeks (PMID: 39085400; NCT02286687). 39085400
PTEN loss Advanced Solid Tumor no benefit Talazoparib Phase II Actionable In a Phase II trial, Talzenna (talazoparib) treatment resulted in limited clinical benefit in patients with advanced solid tumors harboring PTEN deleterious mutations or loss (by IHC), with a clinical benefit rate of 9.4% (1/14, 1 with stable disease >/=24 weeks) and a median overall survival of 8.5 months and a median progression-free survival of 7.7 weeks (PMID: 39085400; NCT02286687). 39085400
HRAS G13R PTEN loss Advanced Solid Tumor resistant AZD8186 Preclinical - Cell culture Actionable In a preclinical study, HRAS-null, NRAS-null, and KRAS knockout cells with PTEN loss and expressing HRAS G13R were not sensitive to treatment with AZD8186 in culture (PMID: 39152269). 39152269
HRAS G13R PTEN loss Advanced Solid Tumor resistant Pictilisib Preclinical - Cell culture Actionable In a preclinical study, HRAS-null, NRAS-null, and KRAS knockout cells with PTEN loss and expressing HRAS G13R were not sensitive to treatment with Pictilisib (GDC-0941) in culture (PMID: 39152269). 39152269
PTEN loss meningioma predicted - sensitive Bevacizumab + Everolimus Case Reports/Case Series Actionable In a clinical case study, treatment with the combination of Afinitor (everolimus) and Avastin (bevacizumab) resulted in a partial response with a progression-free survival of 382 days in a meningioma patient with PTEN loss (by IHC) (PMID: 39143272). 39143272
PTEN loss prostate cancer sensitive Ipatasertib + Onvansertib Preclinical - Cell line xenograft Actionable In a preclinical study, treatment with the combination of Ipatasertib (GDC-0068) and Onvansertib (PCM-075) resulted in greater tumor growth inhibition compared to either therapy alone in PTEN-deficient prostate cancer cell line xenograft models (PMID: 38894678). 38894678
BRAF V600E PTEN R173C colorectal cancer sensitive Cetuximab + Encorafenib + Ribociclib Preclinical - Patient cell culture Actionable In a preclinical study, the combination of Kisqali (ribociclib), Erbitux (cetuximab), and Braftovi (encorafenib) inhibited proliferation and synergistically decreased viability in a patient-derived colorectal cancer cell line harboring BRAF V600E and PTEN R173C (PMID: 39145064). 39145064
PTEN inact mut Advanced Solid Tumor no benefit LY3023414 Case Reports/Case Series Actionable In a Phase II trial (NCI-COG Pediatric MATCH), Samotolisib (LY3023414) treatment resulted in no objective responses in pediatric patients with advanced solid tumors harboring PI3K/mTOR pathway mutations, including PTEN (n=6), PIK3CA (n=5), TSC1 (n=2), TSC2 (n=3), and PIK3R1 (n=1), and resulted in a 3-month progression-free survival of 12%, a 6-month overall survival of 44%, and a 12-month overall survival of 15% (PMID: 39298693; NCT03155620). 39298693