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Alexander Drilon, MD

Gregory J. Riely, MD, PhD


Genetic Abnormalities as Therapeutic Targets in Cholangiocarcinomas

By: Vanessa A. Carter, BS
Posted: Thursday, February 23, 2023

A recent article by Ugo Testa, PhD, of Istituto Superiore di Sanità, Rome, and colleagues focused on the clinical value of identifying genetic aberrations that may be used as therapeutic targets in the treatment of cholangiocarcinomas. This heterogenous group of epithelial malignancies originate at any level of the biliary tree, the investigators noted, and are classified based on their location, whether it be intrahepatic or extrahepatic. These findings were published in Expert Review of Anticancer Therapy.

This updated analysis defines the molecular subgroups and identifies molecular biomarkers demonstrating potential as therapeutic targets. Additional discussion focused on gene expression, copy number alterations, and genetic characterization of cholangiocarcinomas at the level of gene mutation profiling. 

Using genetic sequencing, the study authors discovered several driver mutations they identified as novel therapeutic targets. Genes of interest include NTRK, HER2, BRAF, IDH1, RET, ROS, and FGFR2.

Of note, the identification of the molecular and cellular structure of the cholangiocarcinoma tumor microenvironment was a crucial factor in the development of tumor immunotherapy and other novel therapies. Moreover, combination treatments that include both chemotherapy and targeted molecules or immunotherapy are currently under evaluation for these liver cancers. These treatment regimens demonstrate the potential to improve clinical outcomes of patients with advanced disease, according to the authors.

Disclosure: The study authors reported no conflicts of interest.

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