FGFR Inhibitors: Emerging Treatments in Advanced or Metastatic Cholangiocarcinoma

Cholangiocarcinoma (CCA) is an aggressive cancer with poor prognosis, where the median overall survival remains less than 1 year with standard treatment for unresectable or metastatic disease. This highlights the need for new therapeutic approaches. The fibroblast growth factor receptor (FGFR) family of transmembrane receptors, which are implicated in tumorigenesis, may represent one such target. In this article, we summarize the therapeutic rationale for targeting these receptors in CCA and the role of molecular testing to identify potential responders to treatment. We examine the current available safety and efficacy data on the several small molecule tyrosine kinase inhibitors under investigation for this disease. With a move toward identifying and targeting tumor specific mutations, FGFR inhibitors represent an exciting development which may be available for patients in the near future.