Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson’s Disease
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 im-planted 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-dihydroxyphe-nylalanine suggested graft survival. Clinical measures of symptoms of Parkinson’s disease after surgery stabilized or improved at 18 to 24 months after implantation.
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https://www.nejm.org/doi/full/10.1056/NEJMoa1915872