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A Three-Dimensional Spheroid Model to Investigate the Tumor-Stromal Interaction in Hepatocellular Carcinoma
Published on: September 30, 2021
Molecular genetics and targeted therapeutics in biliary tract carcinoma
1Eric I Marks, Department of Medicine, Penn State Milton S. Hershey Medical Center, Hershey, PA 17033, United States.
Abstract:
The primary malignancies of the biliary tract, cholangiocarcinoma and gallbladder cancer, often present at an advanced stage and are marginally sensitive to radiation and chemotherapy. Accumulating evidence indicates that molecularly targeted agents may provide new hope for improving treatment response in biliary tract carcinoma (BTC). In this article, we provide a critical review of the pathogenesis and genetic abnormalities of biliary tract neoplasms, in addition to discussing the current and emerging targeted therapeutics in BTC. Genetic studies of biliary tumors have identified the growth factors and receptors as well as their downstream signaling pathways that control the growth and survival of biliary epithelia. Target-specific monoclonal antibodies and small molecules inhibitors directed against the signaling pathways that drive BTC growth and invasion have been developed. Numerous clinical trials designed to test these agents as either monotherapy or in combination with conventional chemotherapy have been completed or are currently underway. Research focusing on understanding the molecular basis of biliary tumorigenesis will continue to identify for targeted therapy the key mutations that drive growth and invasion of biliary neoplasms. Additional strategies that have emerged for treating this malignant disease include targeting the epigenetic alterations of BTC and immunotherapy. By integrating targeted therapy with molecular profiles of biliary tumor, we hope to provide precision treatment for patients with malignant diseases of the biliary tract.
Insights
Targeted therapies show promise for advanced biliary tract cancers (BTC), including cholangiocarcinoma and gallbladder cancer, offering new treatment avenues beyond traditional chemotherapy and radiation. Research focuses on molecular targets and genetic mutations for precision medicine approaches.
Area of Science:
- Oncology
- Molecular Biology
- Genetics
Background:
- Biliary tract carcinoma (BTC), encompassing cholangiocarcinoma and gallbladder cancer, frequently presents at advanced stages.
- Conventional treatments like radiation and chemotherapy demonstrate limited efficacy in treating BTC.
- Molecularly targeted agents represent a promising therapeutic strategy for improving treatment outcomes in BTC.
Purpose of the Study:
- To review the pathogenesis and genetic abnormalities of biliary tract neoplasms.
- To discuss current and emerging targeted therapeutics for BTC.
- To highlight the potential of precision medicine in treating biliary tract malignancies.
Main Methods:
- Review of genetic studies identifying growth factors, receptors, and signaling pathways in biliary tumors.
- Analysis of targeted monoclonal antibodies and small molecule inhibitors.
- Examination of ongoing and completed clinical trials for targeted agents in BTC.
- Exploration of emerging strategies such as targeting epigenetic alterations and immunotherapy.
Main Results:
- Genetic studies have identified key signaling pathways driving BTC growth and survival.
- Target-specific agents, including monoclonal antibodies and small molecule inhibitors, have been developed.
- Clinical trials are evaluating these targeted agents as monotherapy or in combination with chemotherapy.
Conclusions:
- Understanding the molecular basis of biliary tumorigenesis is crucial for identifying therapeutic targets.
- Targeted therapy, integrated with molecular profiling, offers potential for precision treatment in BTC.
- Emerging strategies like epigenetic modification and immunotherapy may further enhance treatment efficacy for biliary tract cancers.
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