Pre-clinical Safety and Efficacy of Human Induced Pluripotent Stem Cell-Derived Midbrain Dopaminergic Progenitor Cells for Parkinson’s Disease
Pre-clinical Safety and Efficacy of Human Induced Pluripotent Stem Cell-Derived Midbrain Dopaminergic Progenitor Cells for Parkinson’s Disease
Author or authors of report : Jeha Jeon, Young Cha, Yean Ju Hong, In-Hee Lee, Heejin Jang, Sanghyeok Ko, Serhiy Naumenko, Minseon Kim, Hannah L. Ryu, Zenith Shrestha, Nayeon Lee, Tae-Yoon Park, HoeWon Park, Seo-Hyun Kim
Date of report : 2025-03-06
Parkinsons
Cell Stem Cell
Summary of the Main Points/Findings:
Background and Objective:
This preclinical study aimed to assess the safety, quality, and functional potential of midbrain dopaminergic progenitor cells derived from patient-specific human induced pluripotent stem cells (hiPSCs) for the treatment of Parkinson’s disease. The objective was to evaluate whether these hiPSC-derived cells could serve as a viable autologous cell replacement therapy for restoring dopaminergic neuron function lost in Parkinson’s.
Methods and Findings:
Researchers generated clinical-grade hiPSC lines from fibroblasts of patients with Parkinson’s disease using non-integrating episomal vectors. These cells were differentiated into midbrain dopaminergic progenitors via a chemically defined, scalable protocol. The resulting cells were then evaluated through in vitro assays and in vivo transplantation in animal models. The cells showed strong expression of midbrain-specific markers, stable genomic profiles, and no tumorigenic potential. In rodent models of Parkinson’s, transplanted cells survived, integrated into host tissue, and significantly improved motor function.
Conclusion:
The findings demonstrate that patient-derived hiPSC midbrain dopaminergic progenitor cells can be safely produced under clinical-grade conditions and show promising preclinical efficacy for Parkinson’s disease. These results support moving toward first-in-human trials, with a focus on ensuring long-term safety, consistency in differentiation, and functional integration in the human brain.
