{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["12(11)"],"submitter":["Salati M"],"pubmed_abstract":["Biliary tract cancers are anatomically distinct and genetically diverse tumors, evenly characterized by poor response to standard treatments and a bleak outlook. The advent of comprehensive genomic profiling using next-generation sequencing has unveiled a plethora of potentially actionable aberrations, changing the view of biliary tract cancers from an \"orphan\" to a \"target-rich\" disease. Recently, mutations in isocitrate dehydrogenase genes (IDH1/2) and fusions of the fibroblast growth factor receptor have emerged as the most amenable to molecularly targeted inhibition, with several compounds actively investigated in advanced-phase clinical trials. Specifically, the IDH1 inhibitor ivosidenib has been the first targeted agent to show a survival benefit in a randomized phase III trial of cholangiocarcinoma patients harboring IDH1 mutations. In this review article, we will focus on the IDH1/IDH2 pathway, discussing the preclinical rationale of its targeting as well as the promises and challenges of the clinical development of IDH inhibitors in biliary tract cancers."],"journal":["Cancers"],"pagination":["E3310"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC7696955"],"repository":["biostudies-literature"],"pubmed_title":["IDH Signalling Pathway in Cholangiocarcinoma: From Biological Rationale to Therapeutic Targeting."],"pmcid":["PMC7696955"],"pubmed_authors":["Galassi B","Dominici M","Salati M","Caputo F","Baldessari C","Grossi F","Ghidini M"],"additional_accession":[]},"is_claimable":false,"name":"IDH Signalling Pathway in Cholangiocarcinoma: From Biological Rationale to Therapeutic Targeting.","description":"Biliary tract cancers are anatomically distinct and genetically diverse tumors, evenly characterized by poor response to standard treatments and a bleak outlook. The advent of comprehensive genomic profiling using next-generation sequencing has unveiled a plethora of potentially actionable aberrations, changing the view of biliary tract cancers from an \"orphan\" to a \"target-rich\" disease. Recently, mutations in isocitrate dehydrogenase genes (IDH1/2) and fusions of the fibroblast growth factor receptor have emerged as the most amenable to molecularly targeted inhibition, with several compounds actively investigated in advanced-phase clinical trials. Specifically, the IDH1 inhibitor ivosidenib has been the first targeted agent to show a survival benefit in a randomized phase III trial of cholangiocarcinoma patients harboring IDH1 mutations. In this review article, we will focus on the IDH1/IDH2 pathway, discussing the preclinical rationale of its targeting as well as the promises and challenges of the clinical development of IDH inhibitors in biliary tract cancers.","dates":{"release":"2020-01-01T00:00:00Z","publication":"2020 Nov","modification":"2025-04-26T20:15:06.504Z","creation":"2025-04-06T16:20:49.234Z"},"accession":"S-EPMC7696955","cross_references":{"pubmed":["33182517"],"doi":["10.3390/cancers12113310"]}}