期刊
HEPATOLOGY
卷 73, 期 -, 页码 62-74出版社
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1002/hep.31278
关键词
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资金
- Novo Nordisk Foundation
- Danish Cancer Society
- Danish Medical Research Council
- European Association for the Study of the Liver Sheila Sherlock fellowship in 2016
- Marie Sklodowska-Curie individual postdoctoral fellowship (MiRCHOL)
- Marie Sklodowska-Curie individual postdoctoral fellowship (EpiTarget)
Cholangiocarcinoma (CCA) is a rare disease without diagnostic biomarkers, making population screening infeasible. Most CCA patients require systemic clinical management, and this review discusses the therapeutic potential of molecular targets at different stages of development, as well as the successes and failures of CCA-targeted therapies and the lessons learned.
Cholangiocarcinoma (CCA) encompasses a heterogeneous collection of malignancies for which diagnostic biomarkers are lacking and population screening is infeasible because of its status as a rare disease. Coupled with high postsurgical recurrence rates among the minority of patients diagnosed at resectable stages, systemic clinical management will inevitably be required for the majority of patients with CCA with recurrent and advanced disease. In this review, we discuss the therapeutic potential of different classes of molecular targets at various stages of development in CCA, including those targeted to the tumor epithelia (oncogenic, developmental, metabolic, epigenomic) and tumor microenvironment (angiogenesis, checkpoint regulation). Furthermore, we discuss the successes and failures of CCA-targeted therapies, emphasizing key lessons learned that should pave the way for future molecular target evaluation in this uncommon yet bona fide target-rich disease.
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