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PRE-RECORDED: CELL REPLACEMENT THERAPY FOR PARKINSON’S DISEASE WITH IPS CELLS
Abstract
Abstract Body
Parkinson’s disease (PD) is one of the most promising target diseases for cell therapy based on the history of fetal nigral transplantation in clinics. Due to the shortage of donor supply of fetal tissue and ethical problems, fetal nigral transplantation has not been a standard treatment. The technology of pluripotent stem cells such as induced pluripotent stem cells (iPSCs) and embryonic stem cells (ESCs) offer a limitless donor supply and more advantageous donor source.
After many experiments of non-clinical studies with PD models of mice, rats, and cynomolgus monkeys, several groups have started or are about to start clinical trials.
Kyoto University has started a clinical trial for Parkinson’s disease that transplants dopaminergic progenitors generated from iPSCs since 2018; Kyoto Trial to Evaluate the Safety and Efficacy of iPSC-derived dopaminergic progenitors in the treatment of Parkinson's Disease (Phase I/II). The study is ongoing without any serious adverse event. In this trial, donor iPSCs from healthy volunteer are used with immune suppression to avoid immune rejection. There are several options to reduce the risk of immune rejection by iPSCs beside the immune suppression for the future; 1) autologous transplantation with patient's own iPSCs, 2) matching human leukocyte antigens (HLAs) between donor and recipient with HLA-homo iPSCs, and 3) HLA-depleted iPSCs by gene-editing so called "universal iPSCs". The advantages and disadvantages of each option will be discussed.