Multi-cancer patient-derived organoid platform for translational cancer research

Purpose Patient-derived organoids (PDOs) are useful cancer models because they reflect important features of each patient's tumor. In Korea, however, researchers have not had enough PDO samples with clear clinical information. To improve this situation, the National Cancer Center created a platform to collect organoids from different cancers and provide reliable models that can be used for research. Methods Tumor specimens were obtained through surgical resection, image-guided biopsy, and malignant body fluids. All samples were processed using a unified workflow covering tissue handling, enzymatic dissociation, organoid culture, and criteria for long-term growth. PDOs that continued to grow for more than five passages were classified as successfully established. Quality checks included STR profiling, mycoplasma testing, and histologic and genomic evaluation to ensure accuracy and safety. Clinical and pathological information was linked to each PDO, and biobanking procedures were used for long-term storage. Selected models were evaluated in drug-response assays using a 384-well screening format. Results The platform currently maintains 122 PDO models across multiple cancer types, including oral (n = 33), pancreatic (n = 20), tongue (n = 16), gastric (n = 16), ovarian (n = 9), gallbladder (n = 8), biliary tract (n = 8), breast (n = 5), liver (n = 4), and colorectal cancers (n = 3). These PDOs preserved key histopathologic, genetic, and phenotypic characteristics of their matched tumors and were successfully cryopreserved for long-term use. Drug-response profiling of 42 PDOs with 46 therapeutic agents revealed substantial inter-tumoral variability, and several investigational compounds demonstrated notable antitumor activity. In representative cases, ex vivo cytotoxic responses corresponded with clinical treatment outcomes, underscoring the translational relevance of the platform. Conclusions This PDO platform provides a centralized and high-quality resource that reflects the biological and clinical diversity of human cancers. By supporting systematic drug screening, mechanistic studies, and biomarker-based precision approaches, the platform offers essential infrastructure to advance translational oncology research and promote the development of personalized therapeutic strategies in Korea. This research was supported by the Bio&Medical Technology Development Program of the National Research Foundation (NRF) funded by the Korean government (MSIT) (No. RS-2025-19542979)