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Role of the fibroblast growth factor receptor axis in cholangiocarcinoma

Celina Ang1

  • 1Division of Medicine, Hematology, and Medical Oncology, Mount Sinai School of Medicine, New York, New York, USA.

Journal of Gastroenterology and Hepatology
|February 14, 2015
PubMed

Insights

Advanced cholangiocarcinoma (CCA) treatment is limited. Fibroblast growth factor receptor (FGFR) alterations, particularly FGFR2 fusions, show promise for targeted therapy in CCA patients.

Area of Science:

  • Oncology
  • Molecular Biology
  • Genetics

Background:

  • Advanced cholangiocarcinoma (CCA) presents limited therapeutic options, primarily relying on cytotoxic chemotherapy.
  • Molecular profiling reveals the fibroblast growth factor receptor (FGFR) pathway as a key player in CCA pathogenesis and progression.
  • FGFR aberrations are implicated in various malignancies, driving interest in FGFR-targeted therapies.

Purpose of the Study:

  • To review the role of the FGFR pathway in cholangiocarcinogenesis.
  • To summarize the development of FGFR-directed therapies for CCA treatment.

Main Methods:

  • Literature review of studies on FGFR pathway in CCA.
  • Analysis of clinical trial data for FGFR inhibitors in CCA.
  • Summary of molecular profiling findings in CCA.

Main Results:

  • FGFR2 fusions occur in up to 17% of intrahepatic CCAs.
  • FGFR alterations predict sensitivity to FGFR inhibitors, even post-chemotherapy.
  • Ongoing clinical trials are evaluating FGFR inhibition in CCA patients with specific alterations.

Conclusions:

  • The FGFR pathway is a critical target for CCA therapy.
  • FGFR inhibitors represent a promising antineoplastic strategy for CCA.
  • Targeted therapy based on FGFR alterations offers new hope for CCA patients.
Keywords:
cholangiocarcinomafibroblast growth factor receptortyrosine kinase inhibitor

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