2018 Volume 27 Issue 12 Pages 874-881
Regenerative medicine based on the use of induced pluripotent stem cells is fast becoming a reality. However, there remain several issues in bridging the gap between basic research and clinical application. The keys to bridging this gap are scientific rationale, pre-clinical study with clinical cell lines, and cooperation. We developed a method for the efficient induction of dopaminergic neurons from human induced pluripotent stem cells and for sorting dopaminergic progenitor cells using CORIN, a floor plate marker. The grafted CORIN+ cells survived functioned well as midbrain dopaminergic neurons in rat and monkey models of Parkinson’s disease, even showing minimal risk of tumor formation. After discussion with a Japanese regulatory agency, we performed several pre-clinical studies using a clinical induced pluripotent stem cell line. We also collaborated with a pharmaceutical company from the early stage of development. These results and efforts led us to start a clinical trial in August 2018 to assess the effectiveness of our method for treating patients with Parkinson’s disease.
