A phase 1 clinical trial examining a new stem cell-based therapy for Parkinson’s disease has shown encouraging early results, according to researchers at Memorial Sloan Kettering Cancer Center (MSK). The study, published in Nature, evaluated the safety and feasibility of transplanting dopamine-producing neurons—created from embryonic stem cells—into the brains of 12 patients with advanced Parkinson’s.
The cells, developed in the MSK labs of Dr. Lorenz Studer and Dr. Viviane Tabar, were designed to restore dopamine levels in the brain, which are typically low in individuals with Parkinson’s disease. The therapy, known as bemdaneprocel, was produced from embryonic stem cells and refined to ensure consistency, purity, and clinical-grade standards. It was then frozen and administered to patients during surgery guided by intraoperative MRI imaging.
Participants in the study received either a low or high dose of cells and were monitored over 18 months. No serious side effects were observed, and the study did not find evidence of graft-induced dyskinesia—an issue noted in past efforts involving fetal tissue. Some participants reported improvement in motor function, and those receiving the higher dose experienced an average gain of 2.7 additional “ON” hours per day, indicating periods with better mobility and reduced symptoms.
The preliminary outcomes have led the FDA to approve a move directly to a phase 3 trial. This larger study, sponsored by BlueRock Therapeutics, is expected to begin soon this year and include around 100 participants, incorporating a placebo control to provide more definitive efficacy data.
Researchers emphasize that this development is the result of over two decades of collaborative investigation into stem cell therapy for neurodegenerative disease. Further trials will help determine the long-term effectiveness and broader applicability of this approach.
