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Gim et al. Hepatoma Res 2023;9:51 Hepatoma Research
DOI: 10.20517/2394-5079.2023.90

Review Open Access

Challenges and opportunities for treating

intrahepatic cholangiocarcinoma: targeted therapy

Gahyun Gim , Nabeel Badri
Division of Hematology and Oncology, Department of Medicine, Wilmot Cancer Institute, University of Rochester Medical
Center, Rochester, NY 14642, USA.

Correspondence to: Dr. Gahyun Gim, Division of Hematology and Oncology, Department of Medicine, Wilmot Cancer Institute,
University of Rochester Medical Center, Rochester, NY 14642, USA. E-mail: gahyun_gim@urmc.rochester.edu

How to cite this article: Gim G, Badri N. Challenges and opportunities for treating intrahepatic cholangiocarcinoma: targeted
therapy. Hepatoma Res 2023;9:51. https://dx.doi.org/10.20517/2394-5079.2023.90

Received: 11 Aug 2023 First Decision: 16 Oct 2023 Revised: 13 Nov 2023 Accepted: 20 Dec 2023 Published: 31 Dec 2023

Academic Editors: Antonio Bertoletti, Hongping Xia Copy Editor: Yanbing Bai Production Editor: Yanbing Bai

Abstract
Over the last two decades, substantial progress has been made in the scope of molecular targeted therapy, leading
to transformative advancements in the treatment of various malignancies, including biliary tract cancer (BTC). BTC
represents a heterogeneous group of aggressive tumors with historically poor prognoses. However, recent
discoveries of novel molecular alterations in BTC have provided new avenues for targeted therapeutic
interventions, exemplified by the approval of pemigatinib, specifically designed for FGFR2 gene fusions or
rearrangements in advanced BTC. Furthermore, subsequent regulatory approvals and ongoing clinical trials
focusing on specific gene mutations have considerably expanded the array of treatment options available,
augmenting the potential for personalized treatment strategies. In light of these developments, this review aims to
furnish a comprehensive and up-to-date account of the molecular characteristics and potential targeted therapies
in BTC. By presenting insights into novel therapeutic approaches and outlining prospective directions for
translational and clinical investigations, this review seeks to contribute to the ongoing progress and optimization of
therapeutic approaches in managing BTC.

Keywords: Cholangiocarcinoma, biliary tree cancer, biliary tract cancer, targeted therapy, immunotherapy, next-
generation sequencing, bile duct neoplasms, hepatobiliary malignancy, intrahepatic cholangiocarcinoma

© The Author(s) 2023. Open Access This article is licensed under a Creative Commons Attribution 4.0
International License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, sharing,
adaptation, distribution and reproduction in any medium or format, for any purpose, even commercially, as
long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and
indicate if changes were made.

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