How Your Own Skin Cells Can Help Treat Parkinson's Disease

A new clinical trial is testing the use of stem cells derived from the skin cells of Parkinson's patients to restore dopamine production in the brain. The study primarily assesses the safety of the procedure, which involves the surgical transplantation of these cells into brain areas responsible for movement. Although still in its early stages, the approach represents hope for treating the cause of the disease, and not just its symptoms.

Parkinson's disease is a progressive neurological condition that affects millions of people worldwide and currently has no cure. It develops slowly as specific brain cells responsible for dopamine production cease to function and eventually die.

Dopamine is a neurotransmitter essential for controlling movement, as well as playing important roles in mood, memory, and other cognitive functions. When its levels drop, symptoms such as tremors, muscle rigidity, and slowness of movement appear.

Currently available treatments help alleviate symptoms, but do not stop or reverse the progression of the disease. Therefore, researchers have been seeking strategies that attack the problem at its source: the loss of dopamine-producing cells. In this context, a new clinical trial using stem cells emerges as a promising approach to restore this lost brain function.

The main objective of this study is to evaluate the safety and tolerability of transplanting dopaminergic progenitor cells directly into the brains of people with Parkinson's disease.

This is an open-label, Phase I clinical trial in which the transplanted cells are produced from the participant's own skin cells. These cells are reprogrammed in the laboratory to become capable of producing dopamine, reducing the risk of rejection by the body.

After preparation, the cells are surgically implanted in a specific region of the brain called the putamen, which is part of the basal ganglia and is directly involved in movement control. The procedure is performed under general anesthesia and guided by advanced imaging exams, such as magnetic resonance imaging (MRI).

After surgery, participants are followed for at least two years, with clinical evaluations and neuroimaging exams to monitor both safety and possible signs of benefit.

This pioneering study is being conducted by researchers at Keck Medicine, USC, who seek to go beyond treating symptoms. Instead of simply artificially increasing available dopamine, the strategy attempts to rebuild part of the brain circuitry damaged by the disease.

The expectation is that if the brain returns to producing dopamine more naturally, the progression of Parkinson's disease can be slowed and motor function partially restored.

According to Brian Lee, neurosurgeon and principal investigator of the study, restoring dopamine levels closer to normal could represent a significant advance in the treatment of the disease. The procedure involves the extremely precise implantation of cells into the brain, performed with the support of high-resolution imaging technologies.

Brian Lee, MD, PhD, is a neurosurgeon at Keck Medicine, USC, and principal investigator of the study. Credit: Ricardo Carrasco III

The treatment uses induced pluripotent stem cells, known as iPSCs. Unlike embryonic stem cells, iPSCs are obtained from adult cells, such as skin cells, that are reprogrammed to a versatile state.

According to Xenos Mason, a neurologist and co-investigator of the study, these cells have great potential to reliably mature into dopamine-producing neurons, offering one of the best current chances of restoring this function in the brain.

Xenos Mason, MD, is a neurologist specializing in Parkinson's disease and other movement disorders at Keck Medicine, USC, and co-principal investigator of the study. Credit: Ricardo Carrasco III

After surgery, participants are monitored for up to five years to assess changes in Parkinson's symptoms and possible side effects, such as dyskinesias (excessive involuntary movements) or infections.

The study involves 12 participants with moderate to moderately severe Parkinson's disease and is conducted at three centers in the United States. The ultimate goal is to develop a safe and effective therapy that improves the quality of life and autonomy of patients.

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Treating Parkinson's Disease Through Transplantation of Autologous Stem Cell-Derived Dopaminergic Neurons

ClinicalTrials.gov ID NCT06687837

Sponsor Jeffrey S. Schweitzer, MD, PhD

Information provided by Jeffrey S. Schweitzer, MD, PhD, Massachusetts General Hospital (Responsible Party)

Last Update Posted 2025-05-07

https://clinicaltrials.gov/study/NCT06687837?term=NCT06687837&rank=1

Study Overview:

The goal of this clinical trial is to assess the safety and tolerability of the surgical transplantation of dopaminergic progenitor cells into the brains of participants with Parkinson's disease. The transplanted dopaminergic cells will be derived from the participant's own skin cells.

Detailed Description:

This Phase I, open-label clinical trial aims to assess the feasibility and safety of autologous midbrain dopaminergic progenitor cell (mDAP) transplantation for the treatment of Parkinson's disease. mDAPs will be produced for each participant from a fibroblast sample and then transplanted bilaterally into the putamen under general anesthesia. The study will assess the safety and tolerability of the cell transplant procedure through clinical assessments and neuroimaging (CT, MRI and 18F-DOPA PET) over a 2-year follow-up period.