Kayhanian H, Smyth EC, Braconi C. Emerging molecular targets and therapy for cholangiocarcinoma. World J Gastrointest Oncol 2017; 9(7): 268-280 [PMID: 28808500 DOI: 10.4251/wjgo.v9.i7.268]
Corresponding Author of This Article
Dr. Elizabeth C Smyth, Department of Gastrointestinal Oncology, Royal Marsden Hospital, London and Surrey SM2 5PT, United Kingdom. elizabeth.smyth@rmh.nhs.uk
Research Domain of This Article
Gastroenterology & Hepatology
Article-Type of This Article
Review
Open-Access Policy of This Article
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/
World J Gastrointest Oncol. Jul 15, 2017; 9(7): 268-280 Published online Jul 15, 2017. doi: 10.4251/wjgo.v9.i7.268
Emerging molecular targets and therapy for cholangiocarcinoma
Hamzeh Kayhanian, Elizabeth C Smyth, Chiara Braconi
Hamzeh Kayhanian, Elizabeth C Smyth, Chiara Braconi, Department of Gastrointestinal Oncology, Royal Marsden Hospital, London and Surrey SM2 5PT, United Kingdom
Chiara Braconi, Institute of Cancer Research, London and Surrey SM2 5PT, United Kingdom
Author contributions: Smyth EC and Braconi C should be as the co-corresponding authors; all authors wrote and edited the paper.
Conflict-of-interest statement: Smyth EC declares payment for advisory board participation from Five Prime Therapeutics and for an advisory role from Bristol Meyer-Squibb.
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: Dr. Elizabeth C Smyth, Department of Gastrointestinal Oncology, Royal Marsden Hospital, London and Surrey SM2 5PT, United Kingdom. elizabeth.smyth@rmh.nhs.uk
Telephone: +44-20-86426011 Fax: +44-20-86439414
Received: September 28, 2016 Peer-review started: October 7, 2016 First decision: December 1, 2016 Revised: January 5, 2017 Accepted: March 23, 2017 Article in press: March 24, 2017 Published online: July 15, 2017 Processing time: 286 Days and 8.5 Hours
Abstract
Cholangiocarcinoma (CCA) is a rare cancer arising from the biliary tree with a poor prognosis and limited therapeutic options. Recent large scale molecular characterisation studies have identified recurrent genetic alterations in CCA which may be amenable to therapeutic targeting. In this review we explore the genomic landscape of CCA and examine results from trials of molecularly targeted agents and immunotherapy in this disease. Challenges in CCA diagnosis, treatment and trial design are discussed and we reflect on future directions which may lead to improved outcomes for CCA patients.
Core tip: Cholangiocarcinoma (CCA) is a clinically challenging malignancy; it is rare, molecularly heterogenous and associated with a poor prognosis. Here we review recent data on the genomic landscape of CCA and highlight the results of clinical trials using targeted agents and immunotherapy. We find a number of promising therapeutic agents in development and discuss strategies to improve diagnosis and outcomes in this patient group.