Researchers at Memorial Sloan Kettering Cancer Center (MSK) have announced that 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. 

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.  

Noticeable improvements 

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. 

“Neurologists say things usually get a little bit worse every year with this disease, meaning the score goes up by a few points,” said study co-author Lorenz Studer, MD, Director of MSK’s Center for Stem Cell Biology.  

“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. 

Study showed safety 

“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,” said lead study author Viviane Tabar, MD, Chair of the Department of Neurosurgery at Memorial Sloan Kettering Cancer Center in New York City. 

“The findings were rewarding, as this work is more than a dozen years in the making.”  

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. 

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. 

Sources 

Fox News Digital 

Nature journal