Challenges and opportunities for treating intrahepatic cholangiocarcinoma: targeted therapy

Abstract

Over the last two decades, substantial progress has been made in the scope of molecular targeted therapy, leading to transformative advancements in the treatment of various malignancies, including biliary tract cancer (BTC). BTC represents a heterogeneous group of aggressive tumors with historically poor prognoses. However, recent discoveries of novel molecular alterations in BTC have provided new avenues for targeted therapeutic interventions, exemplified by the approval of pemigatinib, specifically designed for FGFR2 gene fusions or rearrangements in advanced BTC. Furthermore, subsequent regulatory approvals and ongoing clinical trials focusing on specific gene mutations have considerably expanded the array of treatment options available, augmenting the potential for personalized treatment strategies. In light of these developments, this review aims to furnish a comprehensive and up-to-date account of the molecular characteristics and potential targeted therapies in BTC. By presenting insights into novel therapeutic approaches and outlining prospective directions for translational and clinical investigations, this review seeks to contribute to the ongoing progress and optimization of therapeutic approaches in managing BTC.