Novel Targeted Therapies in Clinical Use and on the Horizon for Cholangiocarcinoma

Combination gemcitabine and cisplatin has been the standard of care for cholangiocarcinoma (CCA) since 2010, with a median overall survival of 11.7 months. There is a dire need for improved treatment options for patients with CCA in both the first-line and second-line settings, given the 5-year survival for CCA remains 5–10%. Advances in next-generation sequencing have helped elucidate the pathogenesis of CCA and have identified mutations holding considerable promise for the development of targeted therapies. Clinical trials with inhibitors targeting FGFR2 fusion and IDH1 and IDH2 mutations have been encouraging, with expectations that these drugs will enter clinical practice. KRAS and B-raf proto-oncogenes, Her2/neu genes, and BRCA1 and 2 tumor-suppressor genes have also been proposed as potential targets for future therapies. Targeted therapies have generated optimism that new treatments will soon be available for patients with CCA, with the hope of improved outcomes for these patients.