With around one million people living with Parkinson’s disease in the U.S. — and 90,000 getting new diagnoses each year — the race is on for a cure.
Researchers at Memorial Sloan Kettering Cancer Center (MSK) have announced progress on that front — they have developed a new therapy that uses stem cells to treat advanced Parkinson’s.
In the phase 1 trial, the researchers used donated stem cells (taken from early-stage embryos) to create nerve cells (neurons) and transplanted them into the brains of 12 Parkinson’s patients, according to a press release from MSK.
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Once the cells are injected, they produce dopamine, a hormone in the brain that helps with movement and coordination.
(One of the hallmarks of Parkinson’s is low levels of dopamine, which causes the typical symptoms of tremors, stiffness, balance issues and difficulty walking.)
After 18 months, the injected cells had “taken hold in the brain with no serious side effects,” the researchers reported.
Based on the MDS-UPDRS — a ratings scale for symptoms developed by the International Parkinson and Movement Disorder Society — the participants experienced “noticeable improvements,” especially the group that received a higher dose.
Patients in the high-dose group reported 2.7 hours of additional “on time” each day.
“Neurologists say things usually get a little bit worse every year with this disease, meaning the score goes up by a few points,” study co-author Lorenz Studer, MD, director of MSK’s Center for Stem Cell Biology, said in the press release.
“In our study, not only did the score not get worse, it dropped by more than 20 points in the high-dose group.”
On average, patients in the high-dose group reported 2.7 hours of additional “on time” — indicating periods of normal functioning with minimal symptoms — “a result that could be quite meaningful for their everyday life,” Studer noted.
Given the success of the phase 1 trial, the U.S. Food and Drug Administration (FDA) has granted approval for the researchers to go straight to a phase 3 clinical trial in a much larger patient group — around 100 people — which will take place in the first half of 2025.
The findings were published in the journal Nature.
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“The study showed that developing specific nerve cells from human embryonic stem cells in the lab, then injecting them in the brain of people with Parkinson’s disease, is safe and holds significant promise as a possible future treatment,” lead study author Viviane Tabar, MD, chair of the Department of Neurosurgery at Memorial Sloan Kettering Cancer Center in New York City, told Fox News Digital.
“The findings were rewarding, as this work is more than a dozen years in the making.”
‘Major step forward’
Dr. Mary Ann Picone, medical director of the MS Center at Holy Name Medical Center in Teaneck, New Jersey, said that cells for the treatment of Parkinson’s disease could offer potential for not only slowing disability, but also stopping progression and bringing about improvement in motor function.
“Although there is risk involved in the immune suppression necessary before the stem cell implantation and the procedure itself, it would be a major step forward in replacing the dopaminergic neurons lost in the disease,” Picone, who was not involved in the study, told Fox News Digital.
Levodopa, currently the first-line treatment for Parkinson’s, is limited in that patients need greater dosage amounts as time goes on, according to Picone — “and regulating off and on periods of either stiffness or dyskinesias (uncontrolled muscle movements) becomes more difficult.”
Dr. Ann Murray, director of movement disorders at WVU Rockefeller Neuroscience Institute in West Virginia, referred to the study as “unbelievably exciting” for Parkinson’s patients.
“Although the goal of this particular research project was to ensure safety, getting that significant clinical improvement in the UPDRS is absolutely groundbreaking,” Murray told Fox News Digital. (She also was not involved in the study.)
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“This is just the first step in getting this type of therapy approved for patients suffering from Parkinson’s disease, but this is an amazing first step for the potential benefits of stem cell brain therapy.”
Potential limitations
There were some limitations associated with the study, Tabar noted.
“This is a small study designed to show safety — it is critical to conduct a larger, well-controlled study to prove that the treatment indeed works, otherwise referred to as a Phase 3 ‘efficacy’ study,” she said.
“This is an amazing first step for the potential benefits of stem cell brain therapy.”
These early findings, however, are “suggestive of a strong promise.”
“I think we can finally say that stem cells, when derived and differentiated properly, hold great promise to repair the brain in Parkinson’s and potentially in other conditions someday,” Tabar said.
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The cell therapy was developed at MSK and was licensed to BlueRock Therapeutics in Massachusetts, which funded the study.
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