LAUSR

Simple Summary The biopharmaceutical industry increasingly focuses on the development of new anticancer drugs for effective cancer therapy. Despite these efforts, the success rate is low, and thereby, greater efficacy and better safety properties are required. The high failure rate in drug discovery may be due to limitations of preclinical cancer models that inappropriately recapitulate human cancer biology. To date, patient-derived cancer organoids (PDCOs) have emerged as a model system to recapitulate human cancer biology. In this review, we discuss the advantages and applications of PDCO as a model to investigate anticancer drug efficacy and precision medicine. We also describe the challenges that must be overcome so that PDCOs can substantially represent human cancer biology. Abstract The cancer burden is rapidly increasing in most countries, and thus, new anticancer drugs for effective cancer therapy must be developed. Cancer model systems that recapitulate the biological processes of human cancers are one of the cores of the drug development process. PDCO has emerged as a unique model that preserves the genetic, physiological, and histologic characteristics of original cancer, including inter- and intratumoral heterogeneities. Due to these advantages, the PCDO model is increasingly investigated for anticancer drug screening and efficacy testing, preclinical patient stratification, and precision medicine for selecting the most effective anticancer therapy for patients. Here, we review the prospects and limitations of PDCO compared to the conventional cancer models. With advances in culture success rates, co-culture systems with the tumor microenvironment, organoid-on-a-chip technology, and automation technology, PDCO will become the most promising model to develop anticancer drugs and precision medicine.