With about a million people living with Parkinson's disease in the US, and 90,000 obtaining new diagnoses every year, the race is a cure.
Researchers at the MSK Sloan Kettering Memorial Cancer Center have announced progress on that front: they have developed a new therapy that uses stem cells to treat the advanced Parkinson.
In phase 1 test, the researchers used donated stem cells (taken from early embryos) to create nerve cells (neurons) and transplant the brain of 12 Parkinson's patients, according to a MSK press release.
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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 Parkinson's distinctive characteristics are low dopamine levels, which causes typical symptoms of tremors, stiffness, balance problems and difficulty walking).
About one million people live with Parkinson's disease in the United States, with 90,000 receiving new diagnoses every year. (Istock)
After 18 months, the injected cells “had been strengthened in the brain without serious side effects,” the researchers said.
Based on the MDS-UPDR, a scale of grades for the symptoms developed by the International Society of Parkinson Disorders and the Movement, the participants experienced “notable improvements”, especially the group that received a higher dose.
Patients in the high dose group reported 2.7 hours of additional “time” every day.
“Neurologists say that things generally get a little worse with this disease, which means that the score increases by some points,” he said in the press release, co -author of the study, Lorenz Studer, director of the MSK Stem Biology Center.
“In our study, not only the score did not get worse, but fell into more than 20 points in the high dose group.”
The researchers used donated stem cells (taken from early embryos) to create nerve cells (neurons) and transplanted them to the brain of 12 Parkinson's patients. (Istock)
On average, patients in the high dose group reported 2.7 hours of additional “time”, indicating normal operation periods with minimal symptoms, “a result that could be quite significant for their daily lives,” Studer said.
Given the success of the phase 1 trial, the United States Food and Medicines Administration (FDA) has granted approval for researchers to go directly to a phase 3 clinical trial in a much larger group of patients, around 100 people, which will take place in the first half of 2025.
The findings were published in Nature magazine.
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“The study showed that the development of specific nerve cells from human embryonic stem cells in the laboratory, then injecting them into the brain of people with Parkinson's disease, is safe and is important promising as a possible future treatment,” said the author of the main study Viviane, MD, president of the department of Neurosurgery of the Sloan Kettering MEMORIAL CENTER in New York City, Fox News Digital.
“The findings were rewarding, since this work is more than a dozen years in process.”
'Great step forward'
Dr. Mary Ann Picone, medical director of the MS downtown in Holy Name Medical Center in Teaneck, New Jersey, said that cells for the treatment of Parkinson's disease could offer potential not only to decelerate disability, but also to stop progression and achieve an improvement in the motor function.
“Although there is a risk involved in the necessary immune suppression before the implementation of stem cells and the procedure itself, it would be a great step forward in the replacement of the dopaminergic neurons lost in the disease,” said Picone, who did not participate in the study, to Fox News Digital.
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Levodopa, currently the first -line treatment for Parkinson's, is limited since patients need greater amounts of dose as time passes, according to Picone, “and the regulation of periods of rigidity or disclines (muscular movements not controlled) becomes more difficult.”
After 18 months, the injected cells “had been strengthened in the brain without serious side effects,” the researchers said. (Istock)
Dr. Ann Murray, director of movement disorders at the WVU Rockefeller Neuroscience Institute in West Virginia, referred to the study as “incredibly exciting” for Parkinson's patients.
“Although the objective of this particular research project was to guarantee security, obtaining that significant clinical improvement in the UPDR is absolutely innovative,” Murray told Fox News Digital. (She was not involved in the study either).
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“This is only the first step to obtain this type of therapy approved for patients suffering from Parkinson's disease, but this is a surprising first step for the potential benefits of stem cell brain therapy.”
Potential limitations
There were some limitations associated with the study, Tabar said.
“This is a small study designed to show security: it is essential to carry out a larger and well -controlled study to demonstrate that treatment really works, otherwise, known as a study of 'effectiveness' of phase 3,” he said.
“This is a surprising first step for the potential benefits of stem cell brain therapy.”
These first findings, however, are “suggestive of a great promise.”
“I think we can finally say that stem cells, when they derive and differ properly, have a great promise to repair the brain in Parkinson and potentially in other conditions one day,” said Tabar.
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Cell therapy developed in MSK and graduated Bluerock Therapeutics in Massachusetts, which financed the study.
