A new study from Stanford Medicine has found a way to help protect brain cells in a certain type of Parkinson’s disease that is caused by a single genetic mutation. This discovery could also help people with other forms of Parkinson’s or related brain conditions.
The study focuses on an enzyme called LRRK2. In some people, a genetic mutation causes LRRK2 to become overactive. When this happens, it interferes with how brain cells talk to each other—especially between dopamine-producing neurons and a deep brain area called the striatum. The striatum is important for movement, decision-making, and motivation.
The research was led by Dr. Suzanne Pfeffer. Her team found that blocking the LRRK2 enzyme early in the disease may slow down or even stop symptoms from getting worse. In their experiments, the scientists used a molecule called MLi-2 to block LRRK2 activity in mice that had the Parkinson’s-related mutation.
The study, published in the journal Science Signaling, was done with help from researchers in Scotland. They looked closely at what goes wrong in brain cells when LRRK2 is too active.
One of the key problems is that the brain cells lose tiny structures called primary cilia. These are like little antennas that help cells receive important signals from other parts of the body. One of those signals is called “sonic hedgehog.” It tells support cells to release special proteins that protect brain cells, especially when they are under stress.
In the mice with the LRRK2 mutation, many striatal cells lost their cilia, and communication with dopamine neurons was disrupted. This made the neurons more stressed and led to their death over time.
At first, the team gave the mice the LRRK2 blocker for two weeks, but nothing changed. Then, they extended the treatment to three months. That’s when they saw big improvements.
The cilia began to grow back, and the brain cells started responding to the hedgehog signals again. The support cells released the protective proteins, and the stress on dopamine neurons went down. Some of the damaged neurons even showed signs of recovery.
Dr. Pfeffer said this result may not just stop the disease—it might actually help brain cells get better and work again. That’s a big step forward.
Parkinson’s disease often starts with symptoms like loss of smell, constipation, or sleep problems—many years before tremors or movement issues appear. Dr. Pfeffer hopes that, in the future, people with the LRRK2 mutation could start treatment early, long before the serious symptoms show up.
The researchers now want to test this treatment in people who don’t have the LRRK2 mutation, to see if it can help them too. Some clinical trials are already testing LRRK2 inhibitors in humans.
“This research gives us real hope,” said Dr. Pfeffer. “If we can restore brain cell health, people with Parkinson’s might have a much better future.”
The study was funded by The Michael J. Fox Foundation, the Aligning Science Across Parkinson’s initiative, and the UK Medical Research Council.
If you care about Parkinson’s, you might want to read about how Vitamin B may help slow cognitive decline, and how the Mediterranean diet could lower the risk of Parkinson’s. Other recent studies have shown that blueberries and plant-based diets might also help protect brain health.
If you care about Parkinson’s disease, please read studies that Vitamin B may slow down cognitive decline, and Mediterranean diet could help lower risk of Parkinson’s.
For more information about brain health, please see recent studies that blueberry supplements may prevent cognitive decline, and results showing Plant-based diets could protect cognitive health from air pollution.
This research was published in the journal Science Signaling.
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