Research Overview

Our aim is to develop iPS cell-based therapies for intractable neurological diseases, including use of transplanted cells to improve physical function by repairing neuronal connections. Our main focus is Parkinson's disease, where transplants of fetal midbrain substantia nigra cells has allowed us to gain clinical experience, test efficacy, and identify problems. Our research so far has covered themes such as ES/iPS cell-based induction of dopaminergic neurons and use of cell transplant to improve neurological symptoms in animal models.

To realize cell-based therapy requires attention to a large number of issues, which we have addressed one by one. Among them are: 1) induction of neurons without use of animal factors; 2) cell sorting to inhibit tumor formation; 3) inhibition of cell death during transplant and post-transplant immunosuppression; 4) establishment of a system to confirm long-term efficacy and safety. We have used not only rodents but also non-human primates as Parkinson's disease models for behavioral and imaging analyses to confirm the safety and efficacy of transplanted cells. We have also carried out detailed safety and efficacy testing with the clinical-grade dopaminergic neurons. Based on the findings, we received approval from the Japanese regulatory authorities and have been conducting a physician-initiated trial in Parkinson's disease at Kyoto University Hospital since 2018 (see Figure).

Following on from Parkinson's disease, we are targeting cell-based therapy for stroke and are working on related projects including generating cerebral organoids from human iPS cells and axon extension from the cerebral cortex to the spinal cord.

Physician-initiated trial of iPS cell-based therapy in Parkinson's disease