Saito, Yoshimasa

Biliary tract cancer and pancreatic cancer, which are representative refractory malignancies, are difficult to diagnose at an early stage, and the efficacy of current chemotherapy remains limited; consequently, the 5-year survival rate is below 20%, resulting in an extremely poor prognosis. To date, we have successfully established and stably maintained organoids derived from a variety of refractory and rare cancers, including intrahepatic cholangiocarcinoma, gallbladder cancer, pancreatic cancer, and even neuroendocrine carcinoma arising from the ampulla of Vater. These patient-derived organoids exhibit a high degree of histological and functional similarity to tumors in vivo and represent powerful research tools for in vitro biomarker discovery and evaluation of drug sensitivity to therapeutic agents. On the other hand, analyses of genomic alterations, prognosis, and therapeutic responses in cancer patients using real-world data (RWD) constitute an essential approach for optimizing treatment strategies and advancing personalized medicine in clinical practice. In this study, we aim to integrate these two research domains and establish a novel field of integrative oncology that enables bidirectional translation between real-world clinical data and patient-derived tumor models. Specifically, we will identify clinically relevant genetic alterations and candidate biomarkers from large-scale genomic data and treatment response patterns obtained from RWD, and functionally validate these findings using patient-derived organoids. Through this approach, we seek to simultaneously advance the understanding of molecular pathogenesis and the development of novel therapeutic strategies. Ultimately, this integrative approach will enable the establishment of precision oncology aimed at elucidating the molecular mechanisms of refractory cancers, identifying biomarkers, and developing personalized therapeutic strategies.

Tumor diagnostics and therapeutics