USC Researchers Begin First Human Trial of Stem Cell Therapy for ParkinsonS Disease
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In a groundbreaking step for Parkinson’s disease treatment,researchers at keck Medicine of the University of Southern California (USC) have initiated the first human clinical trial of a novel stem cell therapy. The trial aims to assess the safety and potential efficacy of transplanting lab-grown dopamine-producing brain cells into patients with moderate to moderate-severe Parkinson’s disease. SciTechDaily originally reported on this progress.
A Novel Approach Using iPSCs
The treatment utilizes induced pluripotent stem cells (iPSCs), a versatile type of cell generated in the lab. Unlike embryonic stem cells, iPSCs are created by reprogramming adult cells – such as skin or blood cells – into a “blank slate” state. This allows them to be developed into various cell types, offering a personalized approach to treatment. In this case, the iPSCs are directed to become dopamine-producing neurons, which are progressively lost in Parkinson’s disease. The National Institute of Neurological Disorders and Stroke (NINDS) provides comprehensive information on Parkinson’s disease.
“We believe that these iPSCs can reliably mature into dopamine-producing brain cells, and offer the best chance of jump-starting the brain’s dopamine production,” said Xenos Mason, MD, a neurologist specializing in Parkinson’s disease and other movement disorders at Keck Medicine and co-principal investigator of the study.
Surgical Procedure and Monitoring
The surgical procedure, led by Brian Lee, MD, PhD, a neurosurgeon with Keck Medicine and principal investigator of the study, involves drilling a small hole in the patient’s skull to access the brain. The stem cells are then precisely implanted into the basal ganglia, the brain region responsible for movement control, guided by magnetic resonance imaging (MRI).Mayo Clinic offers detailed information on procedures involving the basal ganglia.
Following the surgery, patients will be monitored for 12-15 months to assess changes in their Parkinson’s disease symptoms and to identify any potential side effects, such as dyskinesia (excess movements) or infection. Long-term monitoring will continue for up to five years.
Trial Details and Future Goals
Keck Medicine is one of three organizations in the United States participating in this clinical trial. The multi-site study will involve a total of 12 participants with moderate to moderate-severe Parkinson’s disease.
“Our ultimate goal is to pioneer a technique that can repair patients’ motor function and offer them a better quality of life,” said Lee.
Key Takeaways
- This is the first human trial of iPSC-derived dopamine neuron transplantation for Parkinson’s disease.
- The therapy aims to replace dopamine-producing cells lost in the disease.
- The trial involves 12 participants with moderate to moderate-severe Parkinson’s disease across three US sites.
- Patients will be monitored for up to five years for safety and efficacy.