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Updated: Dec 26, 2025

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Published on: September 30, 2021
Targets for therapy in biliary tract cancers: the new horizon of personalized medicine
Pritish Iyer1, Ming-Huang Chen2, Lipika Goyal3
1Fox Chase Cancer Center, Temple University Health System, Philadelphia, PA, USA.
Abstract:
Biliary tract cancers (BTCs) are a set of molecularly distinct and heterogeneous diseases. While cytotoxic chemotherapy remains the current standard of care for treatment-naïve and treatment-refractory unresectable disease, recently identified mutations driving oncologic development offer opportunities for targeted therapy. Currently, alterations in the fibroblast growth factor receptor (FGFR), isocitrate dehydrogenase (IDH), v-Raf murine sarcoma viral oncogene homolog B (BRAF), DNA damage repair, and HER2 pathways have demonstrated promising new therapeutic avenues, among others, and various studies have demonstrated clinical activity with targeted tyrosine kinase inhibitors and/or antibodies. In this review, we will discuss the currently identified targets for therapy in BTCs and review currently available data regarding clinical development of treatment options in these molecularly distinct subsets.
Insights
Targeted therapies show promise for biliary tract cancers (BTCs) by targeting specific mutations like FGFR, IDH, BRAF, and HER2. This review explores these new treatment avenues beyond traditional chemotherapy.
Area of Science:
- Oncology and Molecular Medicine
- Gastrointestinal Cancers Research
Background:
- Biliary tract cancers (BTCs) are complex and heterogeneous diseases.
- Cytotoxic chemotherapy is the current standard for unresectable BTCs.
- Emerging molecular targets offer new therapeutic strategies.
Purpose of the Study:
- To review identified molecular targets for BTC therapy.
- To discuss the clinical development of targeted treatments for BTCs.
Main Methods:
- Literature review of identified molecular targets in BTCs.
- Analysis of clinical data for targeted therapies.
- Focus on fibroblast growth factor receptor (FGFR), isocitrate dehydrogenase (IDH), v-Raf murine sarcoma viral oncogene homolog B (BRAF), DNA damage repair, and HER2 pathways.
Main Results:
- Specific mutations in FGFR, IDH, BRAF, DNA damage repair, and HER2 pathways are implicated in BTC development.
- Targeted tyrosine kinase inhibitors and antibodies show clinical activity.
- Promising new therapeutic avenues are emerging for distinct BTC molecular subsets.
Conclusions:
- Targeted therapies represent a significant advancement in BTC treatment.
- Understanding molecular alterations is crucial for personalized BTC therapy.
- Further clinical development is warranted for these targeted agents.
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