Parkinson’s symptoms improved by stem cell treatments in small study
With around one million people living with Parkinson’s disease in the U.S. and 90,000 new diagnoses each year, the need for a cure is urgent. Researchers at Memorial Sloan Kettering Cancer Center (MSK) have made significant progress in this area by developing a new therapy that uses stem cells to treat advanced Parkinson’s.
In a phase 1 trial, researchers utilized donated stem cells from early-stage embryos to create nerve cells (neurons) that were then transplanted into the brains of 12 Parkinson’s patients. These injected cells were able to produce dopamine, a hormone crucial for movement and coordination in the brain. Low levels of dopamine are a hallmark of Parkinson’s and contribute to symptoms such as tremors, stiffness, balance issues, and difficulty walking.
After 18 months, the results were promising. The transplanted cells had successfully taken hold in the brain with no serious side effects. Participants experienced noticeable improvements in their symptoms, particularly those in the high-dose group. Patients in this group reported an additional 2.7 hours of “on time” each day, indicating periods of normal functioning with minimal symptoms.
Dr. Lorenz Studer, co-author of the study and director of MSK’s Center for Stem Cell Biology, noted that typically, Parkinson’s symptoms worsen over time. However, in this study, the high-dose group saw a drop of more than 20 points in their MDS-UPDRS score, a ratings scale for symptoms developed by the International Parkinson and Movement Disorder Society.
Based on the success of the phase 1 trial, the U.S. Food and Drug Administration (FDA) has approved a phase 3 clinical trial to be conducted with a larger patient group of around 100 individuals. This trial is set to take place in the first half of 2025.
Dr. Viviane Tabar, lead study author and chair of the Department of Neurosurgery at Memorial Sloan Kettering Cancer Center, emphasized the safety and promise of this stem cell therapy for Parkinson’s patients. The findings, published in the journal Nature, represent over a decade of research and development.
Dr. Mary Ann Picone, medical director of the MS Center at Holy Name Medical Center, highlighted the potential of this therapy to slow disability, stop disease progression, and improve motor function in Parkinson’s patients. While there are risks associated with immune suppression and the procedure itself, the benefits of replacing lost dopaminergic neurons could be significant.
Dr. Ann Murray, director of movement disorders at WVU Rockefeller Neuroscience Institute, described the study as “unbelievably exciting” for Parkinson’s patients. She emphasized the groundbreaking clinical improvement seen in the UPDRS score and the potential benefits of stem cell brain therapy.
While there are limitations to the study, including its small size and focus on safety, the results are promising. Dr. Tabar believes that stem cells, when derived and differentiated properly, hold great potential to repair the brain in Parkinson’s and other conditions.
The cell therapy was developed at MSK and licensed to BlueRock Therapeutics, which funded the study. This groundbreaking research offers hope for the millions of individuals living with Parkinson’s disease and paves the way for future advancements in treatment.
