More than one million Americans live with Parkinson’s disease, and roughly 90,000 new cases are diagnosed annually, yet a cure remains elusive. Current treatments manage symptoms, but don’t halt the disease’s progression—until now, perhaps.
Stem Cell Therapy Offers Hope for Parkinson’s Patients
Researchers are testing a novel stem cell approach to potentially slow the disease and restore motor function.
- A clinical trial is underway to assess the safety and efficacy of implanting specially prepared stem cells into the brain.
- The treatment utilizes induced pluripotent stem cells (iPSCs), created from adult cells and reprogrammed to become dopamine-producing brain cells.
- Researchers aim to restore dopamine levels, which are depleted in Parkinson’s disease, to improve movement and quality of life.
- The U.S. Food & Drug Administration has granted the trial fast-track designation.
Parkinson’s disease progressively erodes the brain’s ability to control movement, leading to tremors, stiffness, and slowed motion. This deterioration stems from a decline in dopamine, a crucial chemical messenger involved in movement, memory, and mood. As the brain cells that produce dopamine are lost, these essential functions are compromised.
A New Approach: Stem Cell Implantation
Researchers at Keck Medicine of USC are pioneering a new strategy through an early-phase clinical trial. The study investigates whether implanting specifically engineered stem cells can safely replace damaged cells and revitalize dopamine production in the brain.
“If the brain can once again produce normal levels of dopamine, Parkinson’s disease may be slowed down and motor function restored,” explained Brian Lee, MD, PhD, a neurosurgeon with Keck Medicine and the study’s principal investigator.
What Sets These Stem Cells Apart?
The treatment employs induced pluripotent stem cells (iPSCs), a cutting-edge type of laboratory-created stem cell. Unlike embryonic stem cells, iPSCs are derived from adult cells—like skin or blood cells—and then reprogrammed into a versatile state, enabling them to develop into various cell types.
“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 at Keck Medicine specializing in Parkinson’s disease and co-principal investigator of the study.
The Surgical Procedure
During the procedure, Lee creates a small opening in the skull to access the brain. Guided by magnetic resonance imaging (MRI), the stem cells are meticulously implanted into the basal ganglia, a brain region vital for movement control.
Following surgery, patients are closely monitored for 12 to 15 months. Doctors will track changes in Parkinson’s symptoms and monitor for potential side effects, such as dyskinesia—excessive movements—or infection. Long-term follow-up will extend up to five years to evaluate safety and long-term outcomes.
“Our ultimate goal is to pioneer a technique that can repair patients’ motor function and offer them a better quality of life,” Lee stated.
Trial Details and Regulatory Oversight
Keck Medicine is one of three sites in the United States participating in the clinical trial, which involves 12 participants with moderate to moderate-severe Parkinson’s disease.
The stem cell therapy, designated RNDP-001, is developed by Kenai Therapeutics, a biotechnology company dedicated to creating treatments for neurological disorders. The U.S. Food & Drug Administration has granted the Phase 1 REPLACE™ clinical trial fast-track designation, expediting the development and review process.
Disclosure: Mason has received an honorarium payment from Kenai Therapeutics in the past.
