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- W3100638202 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." @default.
- W3100638202 created "2020-11-23" @default.
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- W3100638202 date "2020-11-09" @default.
- W3100638202 modified "2023-09-30" @default.
- W3100638202 title "IDH Signalling Pathway in Cholangiocarcinoma: From Biological Rationale to Therapeutic Targeting" @default.
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- W3100638202 doi "https://doi.org/10.3390/cancers12113310" @default.
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