Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson's Disease.

TitlePersonalized iPSC-Derived Dopamine Progenitor Cells for Parkinson's Disease.
Publication TypeJournal Article
Year of Publication2020
AuthorsSchweitzer JS, Song B, Herrington TM, Park T-Y, Lee N, Ko S, Jeon J, Cha Y, Kim K, Li Q, Henchcliffe C, Kaplitt M, Neff C, Rapalino O, Seo H, Lee I-H, Kim J, Kim T, Petsko GA, Ritz J, Cohen BM, Kong S-W, Leblanc P, Carter BS, Kim K-S
JournalN Engl J Med
Date Published2020 05 14
KeywordsAged, Animals, Basal Ganglia, Cell Differentiation, Disease Models, Animal, Dopaminergic Neurons, Follow-Up Studies, Humans, Induced Pluripotent Stem Cells, Male, Mice, Mice, SCID, Parkinson Disease, Pars Compacta, Positron-Emission Tomography, Putamen, Tomography, X-Ray Computed, Transplantation, Autologous, Transplantation, Homologous

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.)

Alternate JournalN. Engl. J. Med.
PubMed ID32402162
Grant ListK23NS099380 / NH / NIH HHS / United States
R01 NS070577 / NS / NINDS NIH HHS / United States
OD024622 / NH / NIH HHS / United States
NS070577 / NH / NIH HHS / United States
NS084869 / NH / NIH HHS / United States
R24 OD024622 / OD / NIH HHS / United States
R01 NS084869 / NS / NINDS NIH HHS / United States
N/A / US / United States / United States