Personalized cell therapy for Parkinson’s disease

Presented by Kwang-Soo Kim, McLean Hospital/Harvard Medical School Parkinson's disease (PD) is a neurodegenerative disorder associated with loss of midbrain dopamine (mDA) neurons in the substantia nigra, rendering cell transplantation a promising therapeutic strategy. Toward human induced pluripotent stem cell (hiPSC)-based autologous cell therapy for PD, we established a platform of core techniques for the production of mDA progenitors as a safe and effective therapeutic product, including an improved reprogramming method, a novel “spotting”-based in vitro differentiation methodology, a chemical method that safely eliminates undifferentiated hiPSCs, and an improved neurosurgical device. Using this platform, we extensively studied and confirmed the in vivo safety and efficacy in rodent models of PD.