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

N Engl J Med. 2020 May 14;382(20):1926-1932. doi: 10.1056/NEJMoa1915872.


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.).

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Animals
  • Basal Ganglia / diagnostic imaging
  • Basal Ganglia / metabolism
  • Cell Differentiation
  • Disease Models, Animal
  • Dopaminergic Neurons / cytology*
  • Dopaminergic Neurons / metabolism
  • Dopaminergic Neurons / transplantation
  • Follow-Up Studies
  • Humans
  • Induced Pluripotent Stem Cells / immunology
  • Induced Pluripotent Stem Cells / transplantation*
  • Male
  • Mice
  • Mice, SCID
  • Parkinson Disease / diagnostic imaging
  • Parkinson Disease / therapy*
  • Pars Compacta / cytology*
  • Positron-Emission Tomography
  • Putamen / diagnostic imaging
  • Tomography, X-Ray Computed
  • Transplantation, Autologous
  • Transplantation, Homologous