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Novel precision therapies for cholangiocarcinoma: an overview of clinical trials

Pedro Luiz Serrano Uson Junior1,2, Jeremiah Bearss1, Hani M Babiker3

  • 1Division of Hematology & Oncology, Department of Medicine, Mayo Clinic, Scottsdale, AZ, USA.

Expert Opinion on Investigational Drugs
|January 30, 2023
PubMed
Abstract

Insights

Targeted therapies for advanced cholangiocarcinoma, including FGFR and IDH1 inhibitors, are transforming treatment. Research is ongoing to understand resistance mechanisms and optimize sequencing strategies for better patient outcomes.

Area of Science:

  • Oncology
  • Genetics
  • Pharmacology

Background:

  • The treatment landscape for biliary cancers, particularly advanced cholangiocarcinoma, is evolving with targeted therapies.
  • Fibroblast Growth Factor Receptor (FGFR) and Isocitrate Dehydrogenase 1 (IDH1) inhibitors are now integrated into international treatment guidelines.

Approach:

  • This review synthesizes findings from clinical trials evaluating inhibitors for FGFR, IDH, BRAF, and HER2 in advanced cholangiocarcinoma.
  • Literature search was conducted on PubMed, covering studies published between 2010 and 2022.
  • Mechanisms of resistance to targeted therapies and strategies to overcome them are discussed.

Key Points:

  • Precision medicine, driven by actionable targets like FGFR2 fusions and IDH1 mutations, is established in biliary cancer treatment.
  • HER2 and BRAF are emerging targets with potential for tumor-agnostic or cholangiocarcinoma-specific approvals.
  • Circulating tumor DNA (ctDNA) may enhance sequencing accessibility and clinical trial recruitment, though detection limitations require attention.

Conclusions:

  • Targeted therapies represent a significant advancement in managing advanced cholangiocarcinoma.
  • Further research into resistance mechanisms and the application of advanced sequencing technologies like ctDNA is crucial.
  • Optimizing treatment strategies through molecular profiling holds promise for improving patient outcomes in biliary cancers.
Keywords:
BRAFCholangiocarcinomaFGFR2HER2IDHprecision medicine

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