Overview of Representative Clinical Trials Using Human Pluripotent Stem Cell-Differentiated Cells

Human pluripotent stem cells (hPSCs) can be differentiated into any type of tissue cells in the human body. Several clinical trials have been started for the medical care of patients with organ failure, such as those with retinal degeneration diseases (including age-related macular degeneration), Parkinson’s disease, type 1 diabetes, spinal cord injury, epilepsy, myocardial infarction, graft-versus-host disease (GvHD), and cancer, using specific hPSC-derived tissue cells such as retinal pigment epithelium cells, insulin-secreting progenitor cells, dopamine-secreting progenitor cells, oligodendrocytes, GABAergic neurons, cardiomyocytes, mesenchymal stem cells, and engineered natural killer cells. We discuss which cell sources or cell types are promising for clinical applications, such as (i) universal hPSCs or conventional hPSCs and (ii) mature differentiated cells or progenitor cells. Especially, we discuss the progress of clinical trials using hPSC-differentiated cells in this review.