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Updated: Sep 1, 2025

Author Spotlight: Investigating Liver Cancer Pathogenesis Using Patient-Derived Organoids
Published on: August 18, 2023
Patient-derived functional organoids as a personalized approach for drug screening against hepatobiliary cancers
1Division of Gastroenterology and Hepatology, School of Medicine, The Johns Hopkins University, Baltimore, MD, United States.
Abstract:
Patient-derived organoids (PDOs) established from hepatobiliary cancers are seen as valuable models of the cancer of origin. More precisely, PDOs have the ability to retain the original cancer genetic, epigenetic and phenotypic features. By extension, hepatobiliary cancer PDOs have the potential to (1) increase our understanding of cancer biology; (2) allow high-throughput drug screening for more efficient identification and testing of small molecule therapeutics, and (3) permit the design of personalized drug choice approaches for patients with liver cancer. Here, we review general principles for PDO establishment from hepatocellular carcinoma and cholangiocarcinoma, their utilization in drug screening strategies, and last, the establishment of complex PDOs to include tumor stroma. We conclude that PDOs represent a promising and important development in investigating interaction between liver cancer cell types and their microenvironment, as well as for positioning PDOs for high throughput drug screening for hepatobiliary cancers, and that further work is now needed to fully realize their potential.
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