Stanford scientists 'totally surprised' by potential Parkinson's treatment discovery

Fox News

9 hours ago

A recent study from Stanford Medicine that "totally surprised" researchers highlighted what could be a promising approach to slowing Parkinson's disease progression.
The research, published in the journal Science Signaling, took a closer look at enzymes — proteins in the body that speed up chemical reactions and are essential for digestion, liver function and other key functions, according to Cleveland Clinic — and their role in Parkinson's.
The team found that targeting a certain enzyme helped to restore neuron and cell communication in mice.
Lead author Suzanne Pfeffer, PhD, the Emma Pfeiffer Merner Professor in Medical Sciences and a professor of biochemistry at Stanford, told Fox News Digital that the team was "totally surprised that we saw as much improvement as we did."
In about 25% of Parkinson's cases, the culprit is some form of genetic mutation. One of the most common mutations creates an overactive enzyme called LRRK2, according to a Stanford press release.
When there is too much LRRK2 activity, it changes the structure of the brain cells, disrupting important communication between neurons and cells. This system is crucial to movement, motivation and decision-making, according to the researchers.
The goal of the study was to determine whether a specific molecule — the MLi-2 LRRK2 kinase inhibitor — could reverse the effect of overactive enzymes.
Using mice that had the genetic mutation that causes overactive LRRK2 and also had symptoms consistent with early Parkinson's disease, the scientists tried feeding them the inhibitor for two weeks.
There were initially no changes detected in brain structure, signaling or function of the dopamine neurons.
However, after three months of eating the inhibitor, mice affected by the overactive enzyme appeared to have restored their neurons to the point where they were virtually the same as those without the genetic mutation, the study found.
"Findings from this study suggest that inhibiting the LRRK2 enzyme could stabilize the progression of symptoms if patients can be identified early enough," Pfeffer said in the press release.
The study did have some limitations, the researchers acknowledged.
"This was in mice, not people, but our current results indicate that similar pathways are important in humans," Pfeffer told Fox News Digital.
While the study focused on a specific genetic form of the disease, overactive LRRK2 is also present in other cases, meaning this treatment could help multiple types of Parkinson's patients and possibly those with other neurodegenerative diseases, the reseachers claimed.
Looking ahead, the team plans to investigate whether other forms of Parkinson's could benefit.
Parkinson's — a disease that involves the slow death of dopamine-producing neurons, leading to symptoms like tremors and stiffness — affects nearly one million Americans, according to the Parkinson's Foundation, which has offices in New York and Miami.
Experts agree that early intervention is key, as Parkinson's symptoms often appear years after the disease begins.
"These findings suggest that it might be possible to improve, not just stabilize, the condition of patients with Parkinson's disease."
Identifying and treating at-risk individuals sooner could potentially halt or reverse neuron loss.
"These findings suggest that it might be possible to improve, not just stabilize, the condition of patients with Parkinson's disease," Pfeffer said.
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The researcher told Fox News Digital that it's important to encourage patients to undergo genetic testing to learn more about their suitability for clinical trials and future treatments.
The study was funded by The Michael J. Fox Foundation for Parkinson's Research, the Aligning Science Across Parkinson's initiative and the United Kingdom Medical Research Council.