Doctors at Keck Medicine of USC are taking part in an early-stage clinical trial testing whether implanted stem cell-derived brain cells can help people with Parkinson’s disease regain motor function. The approach aims to replace lost dopamine-producing cells and potentially reduce symptoms driven by dopamine decline.
Parkinson’s is a progressive neurological condition that affects movement and can also influence mood and cognition. In the United States, more than 1 000 000 people live with the disease, and roughly 90 000 new cases are diagnosed each year, according to recent estimates.
Aiming to restore dopamine production
The trial focuses on replenishing dopamine, a key chemical messenger needed for smooth, coordinated movement. As dopamine-producing neurons deteriorate, people may develop tremor, muscle rigidity, slowed movement, and walking and balance difficulties.
Standard therapies such as levodopa and deep brain stimulation can improve symptoms for many patients, but they do not replace the underlying lost cells. Researchers hope cell replacement could complement existing care by rebuilding the brain’s dopamine-making capacity.
How the stem cell procedure works
The study uses induced pluripotent stem cells, or iPSCs, which are adult cells reprogrammed into a flexible state and then guided to become dopamine-producing neurons. Because iPSCs are not embryonic stem cells, the technology is often presented as a less ethically contentious route for creating specialized cells.
Neurosurgeons implant the cells into the basal ganglia using imaging guidance, aiming for precise placement in a region central to movement control. Participants are monitored closely for 12 to 15 months for safety signals and changes in Parkinson’s symptoms.
Safety focus and limited enrollment
Investigators are watching for risks that can follow brain surgery or cell therapies, including infection and abnormal involuntary movements known as dyskinesia. Longer follow-up is expected to continue for up to five years to better understand durability and longer-term safety.
Keck Medicine is one of three U.S. sites participating in the Phase 1 REPLACE clinical trial, which plans to enroll 12 people with moderate to moderate-severe Parkinson’s disease. The experimental therapy, called RNDP-001, is being developed by Kenai Therapeutics and has received FDA fast-track designation to support an accelerated review pathway if results warrant it.
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