Molecularly Targeted Therapy in Cholangiocarcinoma

Biliary tract cancers (BTCs) are a heterogeneous group of aggressive malignancies of the liver and biliary tract. While traditional approaches for advanced disease patients have comprised cytotoxic therapies such as gemcitabine and cisplatin, next generation sequencing has revolutioned the field and has cast growing appreciation of the molecular underpinnings of the disease. Towards this end, inhibitors of fibroblast growth factor receptor 2 (FGFR2) and isocitrate dehydrogenase 1 (IDH1) have yielded compelling data in pivotal clinical studies and subsequently garnered regulatory approval across a number of geographies. Enhancing this paradigm are studies that have yielded promising early data for targets such as BRAF and HER2 and microsatellite instability. These trends are expected to continue and the role of precision medicine deepens in the treatment of BTCs and these therapies are studies in relevant combinations, earlier disease settings and as next generation therapies towards these targets are developed.