Stem Cells in Translation

Induced pluripotent stem cell (iPSC) technology has three major clinical applications in clinics: toxicology, drug screening, and regenerative medicine. Cardiac myocytes derived fromSCs have already been used in pharmaceutical companies to predict cardiac toxicity of drug candidates. Similar approach can be applied to other types of cells derived from iPSCs. Inmyopinion, themost promising application of iPSCs resides in drug screening. iPSCs reprogrammed from patients’ somatic cells can be used to recapitulate disease processes.More than 100 papers reporting disease modeling using patientderived iPSCs have been published in the last few years. These include not only monogenicdiseases suchasamyotrophic lateral sclerosis, but also polygenic diseases such as Alzheimer’s disease. I expect that several effective drugs will be developed for currently intractable diseases within the next 10 years. Regenerative medicine is also promising. Given the cost and time required to generate iPSCsundergoodmanufacturing protocol (GMP), pursuing autologous transplantation may not be practical in the near future. Therefore, banking of iPSCs fromHLAhomozygousdonors is anattractive alternative. A collection of 100 donors would sufficiently cover 50%–90% of patients, depending on ethnic groups. To effectively identify these donors, international collaborations are important, including the standardization of informed consent fromdonors andGMP regulations and the cooperation of existing medical banks such as bone marrow and cord blood banks. Transplantation