Stem cell-derived brain repair for Parkinson’s disease: a review of strategies to improve engraftment

Induced pluripotent stem cell-derived dopaminergic progenitor transplantation has received conditional approval in Japan for Parkinson’s disease following the Kyoto Phase I/II trial. Early clinical studies demonstrate favourable safety and preliminary efficacy, but interpretation is limited by open-label designs. Preclinical evidence reveals variable graft survival and low differentiation efficiency, highlighting opportunities for optimisation.

This review examines strategies to improve survival, differentiation, and functional integration of transplanted dopaminergic progenitors. This study was supported by research grants from the Michael J Fox Foundation for Parkinson’s Research (Grant Numbers: 17244 and 023410) and Science Foundation Ireland (Grant Numbers: 19/FFP/6554 and 13/RC/2073_P2). Pharmacology & Therapeutics, Galway Neuroscience Centre, Institute for Health Discovery and Innovation, and School of Pharmacy and Medical Sciences, University of Galway, Galway, Ireland Department of Molecular Medicine, Aarhus University Hospital, Aarhus, Denmark The authors declare no competing interests.

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If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. Comini, G., Denham, M. & Dowd, E.

Stem cell-derived brain repair for Parkinson’s disease: a review of strategies to improve engraftment. npj Parkinsons Dis. (2026). https://doi.org/10.1038/s41531-026-01574-7 DOI: https://doi.org/10.1038/s41531-026-01574-7