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Updated: May 25, 2026

Treatment of Liver Metastases Using an Internal Target Volume Method for Stereotactic Body Radiotherapy
Published on: May 8, 2018
Targeted therapy for biliary tract cancers
1Harvard Medical School, Tucker Gosnell Center for Gastrointestinal Cancers, Massachusetts General Hospital Cancer Center, 55 Fruit Street, Yawkey 7E, Boston, MA 02114, USA. jfaris@partners.org
Abstract:
Biliary tract cancers (BTCs) are a heterogeneous group of malignancies, with a historically poor prognosis as a whole. Until recently, the development of effective therapeutics was hampered by the relatively low incidence, heterogeneity in patients and tumors, and correspondingly poor clinical trial enrollments. With the publication of the landmark phase III ABC-02 trial demonstrating the superiority of gemcitabine and cisplatin combination chemotherapy, the landscape changed for the development of new agents. Despite this progress, there are currently no approved targeted agents for BTC. This review will focus on recent developments in targeted therapeutics, directed against several key signaling pathways in BTC, including epidermal growth factor receptor, angiogenesis, and the mitogen-activated protein kinase pathway. Data from recent phase I and II trials will be discussed, along with a preview of upcoming trials involving targeted therapies.
Insights
Targeted therapies show promise for biliary tract cancers (BTCs), a group of challenging malignancies. This review highlights recent advancements in targeted agents and upcoming clinical trials for improved BTC treatment.
Area of Science:
- Oncology
- Gastroenterology
- Pharmacology
Background:
- Biliary tract cancers (BTCs) are a diverse group of cancers with historically poor outcomes.
- Therapeutic development for BTCs was limited by low incidence, tumor heterogeneity, and insufficient clinical trial participation.
- The gemcitabine and cisplatin combination chemotherapy, proven effective in the ABC-02 trial, has shifted the treatment landscape.
Purpose of the Study:
- To review recent advancements in targeted therapeutics for biliary tract cancers.
- To explore targeted agents focusing on key signaling pathways like EGFR, angiogenesis, and MAPK.
- To discuss current phase I/II trial data and preview future targeted therapy trials in BTC.
Main Methods:
- Review of recent clinical trial data (Phase I and II) for targeted therapies in BTC.
- Analysis of targeted agents acting on specific signaling pathways relevant to BTC.
- Discussion of emerging therapeutic strategies and future clinical trial designs.
Main Results:
- While chemotherapy has improved outcomes, no targeted agents are currently approved for BTC.
- Several targeted agents are under investigation, targeting critical pathways like epidermal growth factor receptor (EGFR), angiogenesis, and the mitogen-activated protein kinase (MAPK) pathway.
- Early-phase trials are providing valuable data on the safety and efficacy of these novel agents.
Conclusions:
- Targeted therapies represent a promising frontier for improving outcomes in biliary tract cancers.
- Further research and clinical trials are essential to identify and validate effective targeted agents for BTC treatment.
- The development of targeted therapies holds potential to overcome current treatment limitations and improve patient prognosis in BTC.
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