Perspectives regarding the potential use of human induced pluripotent stem cells for the development of and research on medicinal products

: Human induced pluripotent stem cells (hiPSCs) are expected to be used in various life science areas, ranging from basic research to medical applications. This article describes perspectives regarding the potential use of hiPSCs, especially in Japan, for manufacturing products related to regenerative medicine as well as for establishing cell-based assay/screening systems that can be used for effective and efficient assessment of candidates for new drugs. The applications of hiPSCs include the following: hiPSC-derived retinal pigment epithelial cells for treating age-related macular degeneration; potential corneal reconstruction by using a combination of various relevant hiPSC-derived differentiated cells; potential treatment of Parkinson's disease by using dopaminergic neurons generated from hiPSCs; potential treatment of spinal cord injury by using neural stem/progenitor cells generated from hiPSCs; potential treatment of chronic heart failure by using hiPSC-derived functional cardiomyocytes; and development of cell-based drug toxicity screening and drug effect assay systems involving cells such as cardiomyocytes, hepatocytes, and neural cells that are differentiated from hiPSCs and can be used in the early phase of new drug development. The current situation regarding the development of guidelines for ensuring the quality and safety of hiPSC-derived medicinal products has also been described.