Increasing Number of Biomarkers Being Studied in Cholangiocarcinoma Clinical Trials

December 2020, Vol 1, No 3

At diagnosis, the majority of patients with intrahepatic cholangiocarcinoma (CCA) present with advanced disease and a poor prognosis. Comprehensive genomic profiling (CGP) of intrahepatic CCA has revealed multiple potential therapeutic targets, including FGFR2, ERBB2 (HER2), and IDH1. Therefore, performing CGP early in the disease course is critical to increasing first-line clinical trial enrollment and access to treatment with FGFR inhibitors.

Kimberly McGregor, MD, Director, Clinical Development, Foundation Medicine, Cambridge, MA, and co-investigators analyzed data from the Foundation Medicine database to determine if data related to the use of first-line FGFR inhibitors correlate with changing CGP patterns for identifying genomic alterations earlier in the disease course.

“We were really interested in looking at our data set to see if we are making an impact with CGP data that is useful for identifying patients who do not actually have an FDA-approved molecular therapy yet,” said Dr McGregor.

“There has been a dramatic shift in the number of biomarker treatment–related studies presented in cholangiocarcinoma since 2018, and targeted therapy trials have also now moved into the first-line setting. Given that this time frame is prior to any FDA-approved targeted therapies, we propose that the increasing availability of molecular-based clinical trials for IHCC [intrahepatic CCA] could be contributing to changing CGP testing patterns,” she added.

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