The use of human induced pluripotent stem cell (hiPSC)-derived midbrain dopaminergic cells (mDACs) in Parkinson’s disease (PD) therapy is gaining attention as a potential autologous treatment approach. A preclinical study examined multiple clinical-grade hiPSC lines generated from sporadic PD patients, assessing their safety and efficacy in preparation for clinical application.
Researchers developed hiPSC lines from freshly biopsied fibroblasts of four PD patients using episomal reprogramming. These cells were then differentiated into mDACs over a refined 21-day protocol and subjected to rigorous evaluation, including whole-genome and exome sequencing, RNA sequencing, and in vivo safety and efficacy studies. A 39-week Good Laboratory Practice (GLP)-compliant mouse safety study confirmed that mDACs from all patients met safety criteria, supporting their potential for clinical use.
These findings highlight the need for standardized regulatory criteria and robust quality control measures in autologous cell therapy. The study’s results will inform an upcoming clinical trial involving eight sporadic PD patients, set to begin in 2025. As research progresses, addressing patient-specific differences will be critical for optimizing stem cell-based treatments for Parkinson’s disease.
