Scientists at Stanford Medicine have discovered a promising new approach that could help protect and possibly restore brain cells damaged by a form of Parkinson’s disease.
Their research, conducted in mice, suggests that blocking an overactive enzyme may allow brain cells to communicate properly again and even reverse early signs of the disease. The findings were published in the journal Science Signaling and are raising hopes for future treatments.
Parkinson’s disease is a progressive brain disorder that mainly affects movement. It happens when nerve cells that produce dopamine slowly die.
Dopamine is a chemical that allows brain cells to send messages that control movement, motivation, and decision-making. When dopamine levels fall, people may develop tremors, stiffness, slow movement, and balance problems. Over time, symptoms usually worsen.
In some cases, Parkinson’s disease is linked to a genetic mutation that affects an enzyme called LRRK2. Enzymes are special proteins that control chemical reactions in the body. When LRRK2 becomes too active, it disrupts how brain cells communicate, especially in the dopamine system. This damage plays a major role in the development of Parkinson’s symptoms.
Dr. Suzanne Pfeffer, one of the lead scientists, explained that if doctors could block this enzyme early, it might slow down or even stop the disease from progressing. To test this idea, researchers used a compound called MLi-2 that attaches to the enzyme and reduces its activity. They wanted to see whether this treatment could protect brain cells and restore lost functions.
In a healthy brain, dopamine-producing neurons send signals to another area called the striatum. These signals depend on tiny structures on cells known as primary cilia. These structures act like small antennas, helping cells receive and send important messages.
However, when the LRRK2 enzyme is overactive, many brain cells lose these antenna-like structures. Without them, communication breaks down, and cells become vulnerable to damage.
One of the most important signals comes from a protein with the unusual name “sonic hedgehog.” This protein helps nearby brain cells produce protective substances that keep neurons alive. When cilia are missing, cells cannot receive this protective signal, which leads to fewer protective proteins and greater risk of cell death.
At first, the scientists treated mice carrying the Parkinson’s mutation with the inhibitor for only two weeks. They saw little improvement. Later, inspired by research showing that cilia can grow and shrink over time, they tried a longer treatment period. This time, they treated the mice for three months, and the results were striking.
After three months, the brain cells in the affected mice looked much more like those in healthy animals. The primary cilia had grown back, allowing communication between brain cells to resume.
Signals from dopamine neurons were once again reaching their targets, and the protective responses were restored. The researchers also observed signs that damaged neurons were beginning to recover, suggesting that the brain may have some ability to heal if given the right conditions.
This discovery is especially important because early warning signs of Parkinson’s disease can appear many years before movement problems begin.
Symptoms such as loss of smell, constipation, and sleep disturbances may occur up to 15 years earlier. If people with the LRRK2 mutation could be treated during this early stage, it might be possible to delay or even prevent serious symptoms.
The research team now plans to investigate whether this treatment could help people with other forms of Parkinson’s disease that are not linked to the LRRK2 mutation. Several clinical trials testing similar enzyme-blocking drugs are already underway, offering hope that new therapies may soon become available.
The study was supported by major organizations dedicated to Parkinson’s research, including The Michael J. Fox Foundation and other international groups. While more research is needed before this approach can be used in humans, the findings provide an encouraging step forward in the fight against this challenging disease.
If future studies confirm these results, blocking the overactive enzyme could become a powerful strategy to protect brain cells, restore lost communication in the brain, and improve the lives of people living with Parkinson’s disease.
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.
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