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Gene | IDH1 |
Variant | R132C |
Impact List | missense |
Protein Effect | gain of function |
Gene Variant Descriptions | IDH1 R132C lies within the active site of the Idh1 protein (PMID: 19228619). R132C confers a gain of function to Idh1 as indicated by conversion of alpha-ketoglutarate to the onco-metabolite 2HG (R(-)-2-hydroxyglutarate in culture (PMID: 19935646, PMID: 26161668, PMID: 22885298). |
Associated Drug Resistance | |
Category Variants Paths |
IDH1 mutant IDH1 act mut IDH1 R132C IDH1 mutant IDH1 R132X IDH1 R132C |
Transcript | NM_005896.4 |
gDNA | chr2:g.208248389G>A |
cDNA | c.394C>T |
Protein | p.R132C |
Source Database | RefSeq |
Genome Build | GRCh38/hg38 |
Transcript | gDNA | cDNA | Protein | Source Database | Genome Build |
---|---|---|---|---|---|
NM_001282387.1 | chr2:g.208248389G>A | c.394C>T | p.R132C | RefSeq | GRCh38/hg38 |
NM_001282386.1 | chr2:g.208248389G>A | c.394C>T | p.R132C | RefSeq | GRCh38/hg38 |
NM_005896 | chr2:g.208248389G>A | c.394C>T | p.R132C | RefSeq | GRCh38/hg38 |
NM_005896.3 | chr2:g.208248389G>A | c.394C>T | p.R132C | RefSeq | GRCh38/hg38 |
NM_001282386.1 | chr2:g.208248389G>A | c.394C>T | p.R132C | RefSeq | GRCh38/hg38 |
NM_001282387.1 | chr2:g.208248389G>A | c.394C>T | p.R132C | RefSeq | GRCh38/hg38 |
NM_001282386 | chr2:g.208248389G>A | c.394C>T | p.R132C | RefSeq | GRCh38/hg38 |
NM_005896.4 | chr2:g.208248389G>A | c.394C>T | p.R132C | RefSeq | GRCh38/hg38 |
NM_001282387 | chr2:g.208248389G>A | c.394C>T | p.R132C | RefSeq | GRCh38/hg38 |
Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
---|---|---|---|---|---|---|---|
IDH1 R132C | high grade glioma | sensitive | Talazoparib | Preclinical - Patient cell culture | Actionable | In a preclinical study, patient-derived glioma cells harboring IDH1 R132C demonstrated increased sensitivity to Talzenna (talazoparib)-induced growth inhibition in culture (PMID: 28148839). | 28148839 |
IDH1 R132C | intrahepatic cholangiocarcinoma | sensitive | Dasatinib | Preclinical | Actionable | In a preclinical study, Sprycel (dasatinib) inhibited growth of intrahepatic cholangiocarcinoma cells harboring IDH1 R132C in culture, and suppressed tumor growth in PDX models (PMID: 27231123). | 27231123 |
IDH1 R132C | cholangiocarcinoma | no benefit | Niraparib | Preclinical - Patient cell culture | Actionable | In a preclinical study, cholangiocarcinoma patient-derived xenograft (PDX) cell lines harboring IDH1 R132C were insensitive to Zejula (niraparib) in culture (PMID: 36374558). | 36374558 |
IDH1 R132C | sarcoma | sensitive | Olaparib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Lynparza (olaparib) treatment delayed tumor growth in cell line xenograft models of sarcoma harboring IDH1 R132C (PMID: 28148839). | 28148839 |
IDH1 R132C | chondrosarcoma | predicted - sensitive | Olaparib | Case Reports/Case Series | Actionable | In a Phase II trial (OLAPCO), Lynparza (olaparib) treatment resulted in a partial response with 59% tumor reduction lasting 14 months in one patient and stable disease lasting 7.5 months in one patient out of four patients with chondrosarcoma harboring IDH1 R132C (PMID: 34994649, NCT02576444). | 34994649 |
IDH1 R132C | cholangiocarcinoma | no benefit | Olaparib | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, cholangiocarcinoma patient-derived xenograft (PDX) cell lines harboring IDH1 R132C were insensitive to Lynparza (olaparib) in culture and did not inhibit tumor growth in the patient-derived xenograft (PDX) model (PMID: 36374558). | 36374558 |
IDH1 R132C | intrahepatic cholangiocarcinoma | sensitive | Saracatinib | Preclinical | Actionable | In a preclinical study, intrahepatic cholangiocarcinoma cells harboring IDH1 R132C demonstrated increased sensitivity to Saracatinib (AZD0530) induced growth inhibition compared to IDH1 wild-type cells in culture (PMID: 27231123). | 27231123 |
IDH1 R132C | chondrosarcoma | predicted - sensitive | Ivosidenib | Case Reports/Case Series | Actionable | In a Phase I trial, Tibsovo (ivosidenib) treatment was tolerated, substantially decreased plasma 2-HG levels, and resulted in a median progression-free survival of 5.6 months and stable disease in 52% (11/21) of patients with chondrosarcoma harboring IDH1 mutations, including IDH1 R132C (n=13) (PMID: 32208957; NCT02073994). | 32208957 |
IDH1 R132C | cholangiocarcinoma | sensitive | Ivosidenib | FDA approved - On Companion Diagnostic | Actionable | In a Phase III (ClarIDHy) trial that supported FDA approval, Tibsovo (ivosidenib) treatment significantly improved median progression-free survival (2.7 vs 1.4 mo, HR=0.37, p<0.001) and prolonged median overall survival (10.8 vs 9.7 mo, HR=0.69, p=0.06) compared to placebo in patients with advanced cholangiocarcinoma harboring IDH1 mutations including R132C/H/L/G/S, resulted in favorable objective response rate (2%, 3/124 vs 0%, 0/61) and stable disease rate (51% vs 28%) (PMID: 32416072; NCT02989857). | detail... detail... 32416072 |
IDH1 R132C | acute myeloid leukemia | sensitive | Ivosidenib | FDA approved - On Companion Diagnostic | Actionable | In a Phase I trial that supported FDA approval, Tibsovo (ivosidenib) treatment resulted in complete remission (CR) in 21.6% (27/125), CR with partial hematological recovery (CRh) in 8.8% (11/125), and overall response (OR) in 41.6% (52/125) of patients with relapsed or refractory acute myeloid leukemia harboring a susceptible IDH1 mutation (R132C/G/H/L/S) as detected by an FDA-approved test (PMID: 29860938; NCT02074839). | detail... detail... detail... 29860938 |
IDH1 R132C | acute myeloid leukemia | sensitive | Ivosidenib | FDA approved - On Companion Diagnostic | Actionable | In a Phase I trial that supported FDA approval, Tibsovo (ivosidenib) treatment resulted in complete remission (CR) in 28.6% (8/28), CR with partial hematological recovery (CRh) in 14.3% (4/28) of patients age 75 or older with untreated acute myeloid leukemia harboring a susceptible IDH1 mutation (R132C/G/H/L/S) as detected by an FDA-approved test, with a median treatment duration of 4.3 months (PMID: 29860938; NCT02074839). | detail... 29860938 detail... |
IDH1 R132C | myelodysplastic syndrome | sensitive | Ivosidenib | FDA approved - On Companion Diagnostic | Actionable | In a Phase I trial that supported FDA approval, Tibsovo (ivosidenib) was tolerated and resulted in a complete response (CR) in 44% (7/16), partial response (PR) in 6% (1/16), and marrow CR in 31% (5/16) of patients with relapsed or refractory myelodysplastic syndrome harboring a susceptible IDH1 mutation (R132C/G/H/L/S) as detected by an FDA-approved test, with a hematologic improvement in >/=1 lineages achieved by 69% (11/16) of patients (J Clin Oncol 40, no. 16_suppl (June 01, 2022) 7053; NCT02074839). | detail... detail... detail... |
IDH1 R132C | high grade glioma | sensitive | AGI-5198 | Preclinical | Actionable | In a preclinical study, AGI-5198 inhibited growth and promoted differentiation in glioma cells expressing IDH1 R132C (PMID: 23558169). | 23558169 |
IDH1 R132C | cholangiocarcinoma | no benefit | Pamiparib | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, cholangiocarcinoma patient-derived xenograft (PDX) cell lines harboring IDH1 R132C were insensitive to Pamiparib (BGB-290) in culture and did not inhibit tumor growth in the patient-derived xenograft (PDX) model (PMID: 36374558). | 36374558 |
IDH1 R132C | fibrosarcoma | predicted - sensitive | Vorasidenib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Voranigo (vorasidenib) treatment decreased tumor 2HG levels in a fibrosarcoma cell line xenograft model harboring IDH1 R132C (Mol Cancer Ther Jan 1 2018 (17) (1 Supp) B126). | detail... |
IDH1 R132C | acute myeloid leukemia | sensitive | Azacitidine + Ivosidenib | Phase Ib/II | Actionable | In a Phase Ib trial, Tibsovo (ivosidenib) and Vidaza (azacitidine) combination treatment demonstrated a favorable safety profile and resulted in an objective response rate (ORR) of 78.3% (18/23, 14 complete remission) and a 12-month overall survival probability of 82% in patients with newly diagnosed acute myeloid leukemia harboring IDH1 R132C (n=14), R132H (n=4), or R132L (n=3) mutations (PMID: 33119479; NCT02677922). | 33119479 |
IDH1 R132C | acute myeloid leukemia | sensitive | Azacitidine + Ivosidenib | FDA approved - On Companion Diagnostic | Actionable | In a Phase III trial (AGILE) that supported FDA approval, Tibsovo (ivosidenib) and Vidaza (azacitidine) combination therapy significantly improved event-free survival (HR 0.33, p=0.002) and median overall survival (24.0 vs 7.9 mo, HR 0.44, p=0.001) compared to Vidaza (azacitidine) plus placebo in patients with newly diagnosed acute myeloid leukemia harboring IDH1 mutations including R132C/H/G/L/S (PMID: 35443108; NCT03173248). | 35443108 detail... detail... |
IDH1 R132C | acute myeloid leukemia | sensitive | Olutasidenib | FDA approved - On Companion Diagnostic | Actionable | In a Phase II trial (Study 2102-HEM-101) that supported FDA approval, Rezlidhia (olutasidenib) treatment resulted in an objective response rate of 46% (57/123, 37 complete remission (CR), 4 CR with partial hematologic recovery, 14 CR with incomplete recovery, 1 morphologic leukemia-free state, 1 partial response) in patients with relapsed/refractory acute myeloid leukemia harboring a susceptible IDH1 mutation (R132C/G/H/L/S) (J Clin Oncol 39, no. 15_suppl (May 20, 2021) 7006; NCT02719574). | detail... detail... detail... |
IDH1 R132C | fibrosarcoma | sensitive | Ceralasertib + Olaparib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of Lynparza (olaparib) and Ceralasertib (AZD6738) resulted in a greater delay in tumor growth compared to Ceralasertib (AZD6738) alone in fibrosarcoma cell line xenograft models harboring IDH1 R132C (PMID: 34027408). | 34027408 |
IDH1 R132C | sarcoma | decreased response | AGI-5198 + Talazoparib | Preclinical - Cell culture | Actionable | In a preclinical study, AGI-5198 reverted the sensitivity of sarcoma cells harboring IDH1 R132C to Talzenna (talazoparib)-induced growth inhibition in culture (PMID: 28148839). | 28148839 |
IDH1 R132C | sarcoma | decreased response | AGI-5198 + Olaparib | Preclinical - Cell culture | Actionable | In a preclinical study, AGI-5198 reverted the sensitivity of sarcoma cells harboring IDH1 R132C to Lynparza (olaparib)-induced growth inhibition in culture (PMID: 28148839). | 28148839 |
IDH1 R132C | acute myeloid leukemia | sensitive | BAY1436032 | Preclinical - Pdx & cell culture | Actionable | In a preclinical study, BAY1436032 decreased R-2HG levels and inhibited growth of primary acute myeloid leukemia (AML) cells harboring IDH1 R132C in culture, and decreased blast number and increased survival of two AML patient-derived xenograft (PDX) models, one which harbored additional alterations in FLT3, NPM1, and NRAS and one which harbored a KMT2A (MLL) alteration (PMID: 28232670). | 28232670 |
IDH1 R132C | Advanced Solid Tumor | predicted - sensitive | DS-1001b | Preclinical - Biochemical | Actionable | In a preclinical study, DS-1001b inhibited IDH1 R132C enzymatic activity in an in vitro assay, and inhibited production of the oncometabolite 2-hydroxyglutarate (2-HG) in transformed human cells expressing IDH1 R132C in culture (PMID: 31727689). | 31727689 |
IDH1 R132C | intrahepatic cholangiocarcinoma | sensitive | Olaparib + Radiotherapy | Preclinical - Cell culture | Actionable | In a preclinical study, treatment with Lynparza (olaparib) enhanced the sensitivity of an intrahepatic cholangiocarcinoma cell line harboring IDH1 R132C to radiation therapy in culture, resulting in decreased survival compared to radiation therapy alone (PMID: 32494639). | 32494639 |
IDH1 R132C | leukemia | sensitive | TETi76 | Preclinical - Cell culture | Actionable | In a preclinical study, TETi76 treatment decreased viability of a leukemia cell line expressing IDH1 R132C in culture (PMID: 33681816). | 33681816 |