Organoid Technology as a Next Generation Platform for Evaluating Drug Discovery System

Abstract

Organoids are three-dimensional (3D) stem cell-derived cell masses that recapitulate the architecture, cellular heterogeneity, and functional characteristics of their tissue of origin. Advances in stem cell technology and 3D culture methods have enabled the generation of organoids from pluripotent stem cells and adult stem cells, facilitating the study of organ development, disease modeling, and personalized medicine. Unlike traditional two-dimensional (2D) cultures and patient-derived cancer cell lines (PDCs), organoids maintain in vivo-like cell-cell and cell-extracellular matrix interactions, preserving tissue-specific spatial organization and genetic stability. Patient-derived organoids (PDOs) have emerged as powerful models for high-throughput drug screening and precision oncology, demonstrating differential drug sensitivities reflective of tumor heterogeneity. Organoid models have been successfully established for multiple organs including intestine, brain, lung, liver, pancreas, and stomach, and have shown promise in predicting patient-specific therapeutic responses. Despite limitations in current models such as patient-derived xenografts (PDXs), organoids offer a scalable, cost-effective platform with higher physiological relevance. This review outlines the evolution of organoid technology, key culture techniques, and their applications in cancer research and drug discovery, highlighting future prospects and challenges in clinical translation.