(F1256) iPSC-based therapy for Parkinson’s disease

Abstract: We pursue a cell replacement therapy for Parkinson's disease (PD) using induced pluripotent stem cells (iPSCs). To this goal, we have developed a method for 1) scalable dopaminergic (DA) neuron induction on human laminin fragments and 2) sorting DA progenitor cells using a floor plate marker, CORIN. The grafted CORIN+ cells survived well, functioned as midbrain DA neurons in the PD model rats and monkeys, and showed a minimal risk of tumor formation. Based on these results, we started a clinical trial to treat PD patients at Kyoto University Hospital in 2018. The trial evaluated the safety and efficacy of transplanting human iPSC-derived DA progenitors into PD patients' putamen. Using a stereotaxic surgical technique, we implanted approximately 5 or 10 million cells into the bilateral putamen of the patients. The target was seven patients, and we finished the two-year observation of all patients in 2023. In this presentation, I will summarize this project.

Funding Source: This study was supported by a grant from the Research Project for Practical Application of Regenerative Medicine of the Japan Agency for Medical Research and Development (AMED) [23bk0104126h0003] to J.T.

Clinical Trial ID number: jRCT2090220384 and UMIN000033564