Stanford study reveals potential breakthrough in Parkinson's disease treatment through LRRK2 inhibition
Shreeaa Rathi | Jul 04, 2025, 00:38 IST
LRRK2 Inhibitor Revitalizes Mouse
Image credit : TIL Creatives
Exciting developments from Stanford Medicine highlight a breakthrough in Parkinson's research. A team of scientists zeroed in on a specific enzyme in genetically modified mice, successfully using an inhibitor to counteract its detrimental effects. Remarkably, after just three months, there was a noticeable improvement in neuron communication.
A recent study from Stanford Medicine, published in Science Signaling, explored the role of enzymes in Parkinson's disease and found that targeting a specific enzyme in mice helped restore neuron and cell communication, potentially slowing disease progression. The study, which focused on mice with a genetic mutation causing overactive LRRK2, found that an inhibitor reversed the effects of the enzyme after three months, suggesting early intervention and treatment of at-risk individuals could halt or reverse neuron loss in Parkinson's patients. The research team was "totally surprised that we saw as much improvement as we did," according to lead author Suzanne Pfeffer. 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.
Parkinson's disease affects nearly one million Americans, according to the Parkinson's Foundation. It involves the slow death of dopamine-producing neurons, leading to symptoms like tremors and stiffness.
In about 25% of Parkinson’s cases, a genetic mutation is the cause. 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 Stanford team's goal 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.
Initially, there were 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 had 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. This means this treatment could help multiple types of Parkinson's patients and possibly those with other neurodegenerative diseases, the researchers claimed.
Looking ahead, the team plans to investigate whether other forms of Parkinson’s could benefit.
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.
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.
Parkinson's disease affects nearly one million Americans, according to the Parkinson's Foundation. It involves the slow death of dopamine-producing neurons, leading to symptoms like tremors and stiffness.
In about 25% of Parkinson’s cases, a genetic mutation is the cause. 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 Stanford team's goal 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.
Initially, there were 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 had 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. This means this treatment could help multiple types of Parkinson's patients and possibly those with other neurodegenerative diseases, the researchers claimed.
Looking ahead, the team plans to investigate whether other forms of Parkinson’s could benefit.
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.
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.