[AKN Admin]

Long time AKN member and leader Prof. Kwang-Soo Kim recently published a breakthrough study on the use of personalized iPSC for the cure of Parkinson’s disease. The study was published in New England Journal of Medicine in May 2020.

https://www.nejm.org/doi/full/10.1056/NEJMoa1915872

Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson’s Disease

Jeffrey S. Schweitzer, M.D., Ph.D., Bin Song, M.D., Ph.D., Todd M. Herrington, M.D., Ph.D., Tae-Yoon Park, Ph.D., Nayeon Lee, Ph.D., Sanghyeok Ko, Ph.D., Jeha Jeon, Ph.D., Young Cha, Ph.D., Kyungsang Kim, Ph.D., Quanzheng Li, Ph.D., Claire Henchcliffe, M.D., D.Phil., Michael Kaplitt, M.D., Ph.D., Carolyn Neff, M.D., Otto Rapalino, M.D., Hyemyung Seo, Ph.D., In-Hee Lee, Ph.D., Jisun Kim, Ph.D., Taewoo Kim, Ph.D., Gregory A. Petsko, D.Phil., Jerome Ritz, M.D., Bruce M. Cohen, M.D., Ph.D., Sek-Won Kong, M.D., Pierre Leblanc, Ph.D., Bob S. Carter, M.D., Ph.D., and Kwang-Soo Kim, Ph.D.

We report the implantation of patient-derived midbrain dopaminergic progenitor cells, differentiated in vitro from autologous induced pluripotent stem cells (iPSCs), in a patient with idiopathic Parkinson’s disease. The patient-specific progenitor cells were produced under Good Manufacturing Practice conditions and characterized as having the phenotypic properties of substantia nigra pars compacta neurons; testing in a humanized mouse model (involving peripheral-blood mononuclear cells) indicated an absence of immunogenicity to these cells. The cells were implanted into the putamen (left hemisphere followed by right hemisphere, 6 months apart) of a patient with Parkinson’s disease, without the need for immunosuppression. Positron-emission tomography with the use of fluorine-18-L-dihydroxyphenylalanine suggested graft survival. Clinical measures of symptoms of Parkinson’s disease after surgery stabilized or improved at 18 to 24 months after implantation. (Funded by the National Institutes of Health and others.)