MUARA KESEHATAN Jurnal Ilmiah Ilmu Kesehatan · Oct 16, 2026
Toward a New Approach to Brain Health: Stem Cell Therapy for Neurological Disorders in Late-Stage Clinical Trials
By Lestari MSD, Mukti RW
Neurodegenerative neurological disorders such as Parkinson's and Huntington's diseases, as well as ischemic brain injury, still lack therapies that can halt or reverse disease progression. Stem cell therapies, particularly those based on pluripotent stem cells and neural stem cells, are now entering late-stage clinical trials and showing encouraging results. This study reviews recent advances in stem cell therapy for neurological disorders based on ongoing late-stage clinical trials. This review identifies three main approaches: dopaminergic progenitor cell transplantation for Parkinson's disease, which demonstrates safety and feasibility in the STEM-PD clinical trial; embryonic neural stem cell transplantation for Huntington's disease in the first-in-human REGEN4HD clinical trial; and mesenchymal stem cell therapy for various neurological indications that harnesses paracrine effects to modulate neuroinflammation. The findings indicate that the STEM-PD clinical trial in 8 patients with Parkinson's disease reported no serious side effects related to the transplanted cells during one year of follow-up, with 6 of the 7 patients who completed the study able to substantially reduce their doses of dopaminergic medication. The REGEN4HD clinical trial for Huntington's disease has administered hNSC-01 neural stem cells to the first patient and reports no serious adverse events at this early stage. Mesenchymal stem cell therapy also shows potential as a modulator of neuroinflammation through paracrine mechanisms that avoid the risks associated with living cells. Remaining challenges include immunosuppression, product standardization, and scalability toward widely accessible therapies.