A clinical trial is investigating whether specialised stem cells implanted in the brain can treat Parkinson’s disease.
Although available treatments can help manage symptoms, there is currently no cure or therapy proven to slow the progression of the disease.
The condition is associated with reduced dopamine release in the brain. Dopamine is a neurotransmitter, a chemical messenger, essential for movement, memory, mood and other functions.
Research has shown that the tremors, stiffness, slow movement and other symptoms are caused by the progressive loss of dopamine-producing brain cells, disrupting the brain’s ability to regulate movement.
Keck Medicine of USC is conducting an early phase clinical trial investigating the safety and effectiveness of implanting specialised stem cells into the brain that have been programmed to replace damaged brain cells and produce dopamine.
“If the brain can once again produce normal levels of dopamine, Parkinson’s disease may be slowed down and motor function restored,” said Brian Lee, a neurosurgeon with Keck Medicine and principal investigator of the study.
The therapy is a relatively new type of lab-generated stem cell called induced pluripotent stem cells.
Unlike embryonic stem cells, these are adult cells, such as skin or blood cells, reprogrammed to a blank slate state capable of evolving into any type of cell.
“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, a neurologist who specialises in the disease and other movement disorders with Keck Medicine and co-principal investigator of the study.
During the procedure, Lee drills a small hole in the patient’s skull to access the brain, then precisely implants the stem cells into the basal ganglia, a part of the brain that controls movement, under the guidance of magnetic resonance imaging.
After surgery, patients are monitored for 12 to 15 months for any changes in their symptoms and for possible side effects including dyskinesia, excess movements, or infection.
The doctors will continue to monitor the patients and their symptoms for up to five years.
“Our ultimate goal is to pioneer a technique that can repair patients’ motor function and offer them a better quality of life,” said Lee.
Keck Medicine is one of three organisations in the US participating in the clinical trial.
The multisite study includes a total of 12 participants with moderate to moderate-severe disease.
The stem cell product, RNDP-001, is manufactured by Kenai Therapeutics, a clinical-stage biotechnology company.
The US Food and Drug Administration has granted the clinical trial, Phase 1 REPLACE, fast-track designation, meaning that the development and review of the drug will be accelerated.
