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Copyright ©The Author(s) 2016. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastroenterol. Jan 28, 2016; 22(4): 1335-1347
Published online Jan 28, 2016. doi: 10.3748/wjg.v22.i4.1335
Molecular genetics and targeted therapeutics in biliary tract carcinoma
Eric I Marks, Nelson S Yee
Eric I Marks, Department of Medicine, Penn State Milton S. Hershey Medical Center, Hershey, PA 17033, United States
Nelson S Yee, Division of Hematology-Oncology, Department of Medicine, Penn State Hershey Medical Center, Hershey, PA 17033, United States
Nelson S Yee, Program of Experimental Therapeutics, Penn State Hershey Cancer Institute, Hershey, PA 17033, United States
Author contributions: Marks EI and Yee NS conceived and designed the study, reviewed the literature, collected and analyzed the data, and wrote the paper.
Conflict-of-interest statement: The authors Marks EI and Yee NS declare no conflicting interests that are related to this article.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Correspondence to: Nelson S Yee, MD, PhD, Program of Experimental Therapeutics, Penn State Hershey Cancer Institute, 500 University Drive, Hershey, PA 17033-0850, United State. nyee@hmc.psu.edu
Telephone: +1-717-5310003 Fax: +1-717-5315076
Received: May 22, 2015
Peer-review started: May 23, 2015
First decision: September 29, 2015
Revised: October 29, 2015
Accepted: November 19, 2015
Article in press: November 19, 2015
Published online: January 28, 2016
Core Tip

Core tip: For patients with cholangiocarcinoma and gallbladder carcinoma, targeted therapeutics provide new opportunity of treatment that is potentially less toxic and more effective. These target-specific monoclonal antibodies and small molecule inhibitors are directed against the signaling pathways that drive the progression of biliary tract cancers. This article provides an updated review of the molecular pathogenesis of these malignant neoplasms as the framework for describing the mechanisms by which targeted agents work. The preclinical and clinical data from investigation of targeted therapeutics in biliary tract cancer are discussed.