Updates on Targeted Therapy in Advanced Biliary Tract Cancers

Abstract

Advances in tumor molecular profiling have revealed key genetic alterations in many malignancies and significantly changed the treatment of patients with metastatic cancer. Biliary tract cancers (BTC) are associated with poor prognosis in the metastatic setting. Recurrent gene mutations have been found in a subset of patients with BTC that play key roles in the oncogenesis of this disease. Differences in molecular profiles based on tumor location (intrahepatic, extrahepatic, gallbladder) reflect the known heterogeneity of BTC. Importantly, the identification of several actionable mutations, including FGFR2 fusions and IDH1/2 mutations, has enabled progress in the development of targeted agents especially in intrahepatic cholangiocarcinoma. Clinical trials of several targeted inhibitors are ongoing and may expand therapy options for patients in the future.