Researchers at Stanford Medicine have discovered a promising way to protect brain cells in a genetic form of Parkinson’s disease.
In a study using mice, they found that blocking an overactive enzyme helped damaged brain cells communicate again and showed early signs of recovery.
The research was published in the journal Science Signaling.
Parkinson’s disease is a brain disorder that slowly gets worse over time. It mainly affects movement, causing symptoms such as shaking, stiffness, slow movement, and balance problems.
These changes happen because brain cells that produce dopamine gradually stop working and die.
Dopamine is a chemical messenger that allows brain cells to communicate with each other. It plays an important role in controlling movement, motivation, learning, and decision-making. As dopamine-producing cells are lost, these brain functions become harder to perform.
In some people, Parkinson’s disease is linked to changes in a gene called LRRK2. These gene changes make an enzyme with the same name become too active. The overactive enzyme interferes with normal communication between brain cells and increases the damage caused by the disease.
The Stanford team focused on a drug-like compound called MLi-2, which blocks the activity of the LRRK2 enzyme. The researchers hoped that turning off the overactive enzyme would allow damaged brain cells to recover and restore normal signaling.
A healthy brain depends on tiny structures called primary cilia, which act like small antennas on the surface of cells. These structures receive important chemical messages from nearby cells. When the LRRK2 enzyme becomes too active, many brain cells lose their primary cilia, making it much harder for them to receive signals that help keep them alive.
One of these signals comes from a protein called sonic hedgehog. This protein tells nearby cells to produce protective substances that support dopamine-producing neurons. Without primary cilia, this protective message cannot be received, leaving vulnerable brain cells at greater risk of dying.
The researchers first treated mice with MLi-2 for two weeks, but they saw little improvement. They then extended the treatment to three months after learning that primary cilia naturally grow and shrink over time. This longer treatment made a major difference.
After three months, the mice regained primary cilia in important brain cells, and communication between brain regions was largely restored. The protective signaling system became active again, and the researchers found evidence that injured dopamine neurons had started to recover.
Senior author Dr. Suzanne Pfeffer said the findings suggest that blocking the LRRK2 enzyme early enough could slow or even delay the disease. Early warning signs of Parkinson’s, including loss of smell, constipation, and certain sleep problems, can appear many years before movement symptoms begin.
The researchers now plan to study whether this approach could also help people with other forms of Parkinson’s disease. Several clinical trials of LRRK2 inhibitors are already in progress, offering hope that this research may one day lead to better treatments.
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 health information, please see recent studies about how wheat gluten might be influencing our brain health, and Olive oil: a daily dose for better brain health..
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