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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
IDH1 R132H high grade glioma sensitive IDH1 Inhibitor AGI-5198 Preclinical - Cell line xenograft Actionable In a preclinical study, AGI-5198 inhibited growth of a glioma cell line harboring IDH1 R132H in culture and in xenograft models (PMID: 23558169). 23558169
IDH1 R132H acute myeloid leukemia sensitive IDH1 Inhibitor 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 R132H (n=4), R132C (n=16), or R132L (n=3) mutations (PMID: 33119479; NCT02677922). 33119479
IDH1 R132H acute myeloid leukemia sensitive IDH1 Inhibitor 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 R132H Advanced Solid Tumor sensitive Niraparib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing IDH1 R132H demonstrated increased sensitivity to Zejula (niraparib)-induced growth inhibition in culture (PMID: 28148839). 28148839
IDH1 R132H Advanced Solid Tumor sensitive Ceralasertib Preclinical - Cell culture Actionable In a preclinical study, treatment with Ceralasertib (AZD6738) resulted in decreased survival in cell lines expressing IDH1 R132H in culture (PMID: 34027408). 34027408
IDH1 R132H colorectal cancer predicted - sensitive Olaparib Preclinical - Cell culture Actionable In a preclinical study, colorectal cancer cells harboring IDH1 R132H were sensitive to treatment with Lynparza (olaparib) in culture, demonstrating decreased colony formation (PMID: 29339439). 29339439
IDH1 R132H glioblastoma resistant Trichostatin A Preclinical - Cell culture Actionable In a preclinical study, glioblastoma cells expressing IDH1 R132H were resistant to treatment with Trichostatin (TSA) in culture, demonstrating decreased apoptotic activity and increased cell viability (PMID: 31151327). 31151327
IDH1 R132H colorectal cancer sensitive Metformin Preclinical Actionable In a preclinical study, colorectal carcinoma cells expressing IDH1 R132H were sensitive to Glucophage (metformin), resulting in decreased cell proliferation in culture (PMID: 26363012). 26363012
IDH1 R132H glioblastoma resistant Vorinostat Preclinical - Cell culture Actionable In a preclinical study, glioblastoma cells expressing IDH1 R132H were resistant to treatment with Zolinza (vorinostat) in culture, demonstrating increased cell viability (PMID: 31151327). 31151327
IDH1 R132H anaplastic astrocytoma sensitive Azacitidine Preclinical Actionable In a preclinical study, long-term treatment with Vidaza (azacitidine) resulted in increased cellular differentiation, decreased proliferation, and tumor regression in a patient-derived xenograft (PDX) model of anaplastic astrocytoma harboring IDH1 R132H (PMID: 24077805). 24077805
IDH1 R132H glioblastoma predicted - sensitive IDH1 Inhibitor AGI-5198 + Trichostatin A Preclinical - Cell culture Actionable In a preclinical study, the addition of AGI-5198 treatment in glioblastoma cells expressing IDH1 R132H led to decreased resistance to Trichostatin (TSA) treatment in culture, resulting in reduced cell viability (PMID: 31151327). 31151327
IDH1 R132H colorectal cancer predicted - sensitive Talazoparib Preclinical - Cell culture Actionable In a preclinical study, colorectal cancer cells harboring IDH1 R132H were sensitive to treatment with Talzenna (talazoparib) in culture, demonstrating decreased colony formation (PMID: 29339439). 29339439
IDH1 R132H Advanced Solid Tumor sensitive Cisplatin + Talazoparib Preclinical - Cell culture Actionable In a preclinical study, Talazoparib (BMN-673) and Cisplatin synergistically inhibited growth of transformed cells over expressing IDH1 R132H in culture (PMID: 28148839). 28148839
IDH1 R132H Advanced Solid Tumor sensitive BAY1895344 + Olaparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Lynparza (olaparib) and BAY1895344 resulted in a greater decrease in cell survival compared to BAY1895344 alone in cells expressing IDH1 R132H in culture (PMID: 34027408). 34027408
IDH1 R132H oligodendroglioma sensitive Decitabine Preclinical - Pdx & cell culture Actionable In a preclinical study, Dacogen (decitabine) decreased colony formation and tumor growth in patient derived xenograft (PDX) models of patient derived oligodendroglioma cells with IDH1 R132H mutations and co-deletion of 1p and 19q (PMID: 24077826). 24077826
IDH1 R132H colon carcinoma sensitive Olaparib Preclinical - Cell culture Actionable In a preclinical study, colon carcinoma cells over expressing IDH1 R132H demonstrated increased sensitivity to Lynparza (olaparib)-induced growth inhibition in culture (PMID: 28148839). 28148839
IDH1 R132H Advanced Solid Tumor sensitive Berzosertib Preclinical - Cell culture Actionable In a preclinical study, transformed cells expressing IDH1 R132H demonstrated increased sensitivity to Berzosertib (VX-970)-induced growth inhibition in culture (PMID: 28148839). 28148839
IDH1 R132H high grade glioma sensitive BPTES Preclinical - Cell culture Actionable In a preclinical study, a glioma cell line expressing IDH1 R132H demonstrated increased sensitivity to growth inhibition by BPTES compared to a cell line expressing wild-type IDH1 in culture (PMID: 21045145). 21045145
IDH1 R132H acute myeloid leukemia sensitive IDH1 Inhibitor 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). 29860938 detail... detail...
IDH1 R132H acute myeloid leukemia sensitive IDH1 Inhibitor 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). 29860938 detail... detail...
IDH1 R132H Advanced Solid Tumor sensitive Rucaparib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing IDH1 R132H demonstrated increased sensitivity to Rubraca (rucaparib)-induced growth inhibition in culture (PMID: 28148839). 28148839
IDH1 R132H glioblastoma predicted - sensitive IDH1 Inhibitor AGI-5198 + Vorinostat Preclinical - Cell culture Actionable In a preclinical study, the addition of AGI-5198 treatment in glioblastoma cells expressing IDH1 R132H led to decreased resistance to Zolinza (vorinostat) treatment in culture, resulting in reduced cell viability (PMID: 31151327). 31151327
IDH1 R132H high grade glioma sensitive Talazoparib Preclinical - Patient cell culture Actionable In a preclinical study, patient-derived glioma cells harboring IDH1 R132H demonstrated increased sensitivity to Talazoparib (BMN-673)-induced growth inhibition in culture (PMID: 28148839). 28148839
IDH1 R132H high grade glioma sensitive Talazoparib Preclinical - Cell culture Actionable In a preclinical study, Talzenna (talazoparib) treatment inhibited viability of immortalized astrocytes expressing IDH1 R132H in culture (PMID: 34118569). 34118569
IDH1 R132H low grade glioma predicted - sensitive IDH1 Inhibitor Ivosidenib Phase I Actionable In a Phase I trial, low grade glioma patients with an IDH1 mutation (n=66; R132H=57, R132C/G/S=1 each, R132X=5) treated with Tibsovo (ivosidenib) demonstrated an overall response rate of 2.9% (1/35, 1 partial response) and stable disease in 85.7% (30/35) of patients with a non-enhancing glioma versus no responses and stable disease in 45.2% (14/31) of patients with an enhancing glioma, and led to a median progression-free survival of 13.6 months and 1.4 months, respectively (PMID: 32530764; NCT02073994). 32530764
IDH1 R132H high grade glioma predicted - sensitive IDH Inhibitor (Pan) AG-881 Preclinical - Cell line xenograft Actionable In a preclinical study, AG-881 treatment decreased brain tumor 2HG levels in an orthotopic glioma cell line xenograft model harboring IDH1 R132H (Mol Cancer Ther Jan 1 2018 (17) (1 Supp) B126). detail...
IDH1 R132H Advanced Solid Tumor decreased response IDH1 Inhibitor AGI-5198 + Talazoparib Preclinical - Cell culture Actionable In a preclinical study, AGI-5198 reverted the sensitivity of transformed cells over expressing IDH1 R132H to Talazoparib (BMN-673)-induced growth inhibition in culture (PMID: 28148839). 28148839
IDH1 R132H high grade glioma not applicable N/A Guideline Diagnostic IDH1 R132H is diagnostic and aids in the diagnosis of gliomas (NCCN.org). detail...
IDH1 R132H acute myeloid leukemia sensitive IDH1 Inhibitor BAY1436032 Preclinical - Patient cell culture Actionable In a preclinical study, BAY1436032 decreased (R)-2-hydroxyglutarate (R-2HG) levels, and inhibited growth and increased differentiation of patient-derived acute myeloid leukemia cells harboring IDH1 R132H in culture (PMID: 28232670). 28232670
IDH1 R132H Advanced Solid Tumor sensitive Ceralasertib + Olaparib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Lynparza (olaparib) and Ceralasertib (AZD6738) resulted in a greater decrease in cell survival compared to Ceralasterib (AZD6738) alone in cells expressing IDH1 R132H in culture, and a longer delay in tumor growth in cell line xenograft models (PMID: 34027408). 34027408
IDH1 R132H colorectal cancer resistant IDH1 Inhibitor AGI-5198 + Metformin Preclinical Actionable In a preclinical study, colorectal cancer cells harboring IDH1 R132H demonstrated increased cell proliferation when treated with a combination of Glucophage (metformin) and AGI-5198 (PMID: 26363012). 26363012
IDH1 R132H glioblastoma sensitive IDH1 Inhibitor DS-1001b Preclinical - Pdx Actionable In a preclinical study, treatment with DS-1001b inhibited subcutaneous and intracranial tumor growth in a patient-derived xenograft (PDX) model of glioblastoma harboring IDH1 R132H, and also demonstrated reduced levels of the oncometabolite 2-hydroxyglutarate (2-HG) within tumors and plasma and increased expression of the astrocyte differentiation marker glial fibrillary acidic protein in tumor cells (PMID: 31727689). 31727689
IDH1 R132H Advanced Solid Tumor sensitive Olaparib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing IDH1 R132H demonstrated increased sensitivity to Lynparza (olaparib)-induced growth inhibition in culture (PMID: 28148839). 28148839
IDH1 R132H Advanced Solid Tumor sensitive Ceralasertib + Talazoparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Talzenna (talazoparib) and Ceralasertib (AZD6738) resulted in a greater decrease in cell viability compared to Ceralasertib (AZD6738) alone in cells expressing IDH1 R132H in culture (PMID: 34027408). 34027408
IDH1 R132H Advanced Solid Tumor sensitive Ceralasertib + Niraparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Zejula (niraparib) and Ceralasertib (AZD6738) resulted in a greater decrease in cell viability compared to Ceralasertib (AZD6738) alone in cells expressing IDH1 R132H in culture (PMID: 34027408). 34027408
IDH1 R132H high grade glioma predicted - sensitive Olaparib Phase II Actionable In a Phase II trial (OLAGLI), Lynparza (olaparib) therapy was well tolerated in high grade glioma patients harboring IDH1 R132 (32/35) or other IDH mutations (3/35), and led to a partial response in 5% (2/35) and stable disease in 37% (14/35) of 35 evaluable patients, median progression-free survival (PFS) of 2.3 mo, median overall survival of 15.9 mo, a median duration of response of 9 mo, and a 6-mo PFS of 31% (11/35) (J Clin Oncol 39, no. 15_suppl (May 20, 2021) 2007-2007; NCT03561870). detail...
IDH1 R132H high grade glioma predicted - sensitive Olaparib Preclinical - Cell culture Actionable In a preclinical study, Lynparza (olaparib) treatment inhibited viability of a glioma cell line expressing IDH1 R132H in culture (PMID: 34118569). 34118569
IDH1 R132H colorectal cancer resistant IDH1 Inhibitor AGI-5198 + Radiotherapy Preclinical Actionable In a preclinical study, colorectal cancer cells expressing IDH1 R132H were resistant to radiotherapy when treated with AGI-5198 (PMID: 26363012). 26363012
IDH1 R132H Advanced Solid Tumor sensitive BAY1895344 Preclinical - Cell culture Actionable In a preclinical study, treatment with BAY1895344 resulted in decreased survival in cell lines expressing IDH1 R132H in culture (PMID: 34027408). 34027408
IDH1 R132H glioblastoma sensitive Temozolomide + Vandetanib Phase II Actionable In a Phase II trial, Caprelsa (vandetanib), in combination with Temodar (temozolomide) and radiation therapy, demonstrated a significant increase in PFS and OS in glioblastoma patients harboring IDH1 R132H compared to glioblastoma patients without IDH1 R132H (PMID: 25910950). 25910950
IDH1 R132H Advanced Solid Tumor sensitive Talazoparib Preclinical - Cell culture Actionable In a preclinical study, transformed cells over expressing IDH1 R132H demonstrated increased sensitivity to Talazoparib (BMN-673)-induced growth inhibition in culture (PMID: 28148839). 28148839
IDH1 R132H glioblastoma resistant Valproic acid Preclinical - Cell culture Actionable In a preclinical study, glioblastoma cells expressing IDH1 R132H were resistant to treatment with Valproic Acid in culture, demonstrating increased cell viability (PMID: 31151327). 31151327
IDH1 R132H cholangiocarcinoma sensitive IDH1 Inhibitor 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... 32416072 detail...
IDH1 R132H Advanced Solid Tumor predicted - sensitive IDH1 Inhibitor DS-1001b Preclinical - Cell culture Actionable In a preclinical study, DS-1001b inhibited IDH1 R132H enzymatic activity in an in vitro assay, and inhibited production of the oncometabolite 2-hydroxyglutarate (2-HG) in transformed human cells expressing IDH1 R132H in culture (PMID: 31727689). 31727689
IDH1 R132H colon carcinoma sensitive Talazoparib Preclinical - Cell culture Actionable In a preclinical study, colon carcinoma cells over expressing IDH1 R132H demonstrated increased sensitivity to Talazoparib (BMN-673)-induced growth inhibition in culture (PMID: 28148839). 28148839
Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT02525692 Phase II ONC201 Oral ONC201 in Recurrent GBM, H3 K27M Glioma, and Midline Glioma Active, not recruiting USA 0
NCT03343197 Phase I Ivosidenib AG-881 Study of AG-120 and AG-881 in Subjects With Low Grade Glioma Active, not recruiting USA 0
NCT04056910 Phase II Ivosidenib + Nivolumab Ivosidenib (AG-120) With Nivolumab in IDH1 Mutant Tumors Recruiting USA 0
NCT02193347 Phase I Montanide ISA 51 + PEPIDH1M vaccine + Sargramostim + Temozolomide IDH1 Peptide Vaccine for Recurrent Grade II Glioma (RESIST) Active, not recruiting USA 0
NCT02977780 Phase II CC-115 Abemaciclib + Temozolomide Temozolomide Neratinib + Temozolomide INdividualized Screening Trial of Innovative Glioblastoma Therapy (INSIGhT) Active, not recruiting USA 0
NCT03224104 Phase I Radiotherapy + Zotiraciclib Temozolomide + Zotiraciclib Zotiraciclib Study of TG02 in Elderly Newly Diagnosed or Adult Relapsed Patients With Anaplastic Astrocytoma or Glioblastoma (STEAM) Completed FRA | DEU | AUT 2
NCT03970447 Phase II Lomustine Regorafenib Temozolomide A Trial to Evaluate Multiple Regimens in Newly Diagnosed and Recurrent Glioblastoma (GBM AGILE) Recruiting USA | FRA | CAN 1
NCT03893903 Phase I Avelumab IDH1 R132H peptide vaccine Avelumab + IDH1 R132H peptide vaccine AMPLIFYing NEOepitope-specific VACcine Responses in Progressive Diffuse Glioma (AMPLIFY-NEOVAC) Recruiting DEU 0
NCT04088188 Phase I Cisplatin + Gemcitabine + Pemigatinib Cisplatin + Gemcitabine + Ivosidenib Gemcitabine and Cisplatin With Ivosidenib or Pemigatinib for the Treatment of Unresectable or Metastatic Cholangiocarcinoma Recruiting USA 0
NCT04164901 Phase III AG-881 Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO) Active, not recruiting USA | ITA | FRA | ESP | DEU | CAN 4
NCT03953898 Phase II Olaparib Using the Anticancer Drug Olaparib to Treat Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome With an Isocitrate Dehydrogenase (IDH) Mutation Recruiting USA 0