Implantation of Patient-Derived Dopamine Progenitor Cells for Parkinson Disease

Improvements in motor symptoms were mild in a single-patient study.

Loss of midbrain dopamine neurons in the substantia nigra pars compacta has long been considered a promising target for tissue transplantation in patients with Parkinson disease (PD). Investigators now report on a single PD patient who underwent bilateral implantation of pluripotent stem cell–derived midbrain dopaminergic progenitor cells into the putamen (left then right hemisphere, with 6 months between operations).

The patient's own skin cells were differentiated in vitro from autologous induced pluripotent stem cells. The transplanted cells had the phenotypic properties of substantia nigra pars compacta neurons. The authors tested the cells in a humanized mouse model, which revealed an absence of immunogenicity.

Positron-emission tomograp…

Reviewing Author

Michael S. Okun, MD

Disclosures

Consultant/Advisory BoardParkinson’s Foundation; Tourette Association of America

Speaker’s BureauMedscape/WebMD CME, Movement Disorders Society CME, American Academy of Neurology CME

RoyaltiesBooks4Patients; Demos; Cambridge; Taylor and Francis; Robert Rose; Public Affairs

Grant/Research SupportNational Institutes of Health; Parkinson’s Foundation; Tourette Association of America; University of Florida Foundation

Editorial BoardsTremor and Other Hyperkinetic Movements; JAMA Neurology

Leadership Positions in Professional SocietiesParkinson’s Foundation (Medical Advisor)