
A new study from Stanford Medicine has found a promising way to protect brain cells in a form of Parkinson’s disease linked to a genetic mutation.
The discovery may also help scientists better understand other types of Parkinson’s disease and similar brain conditions.
Parkinson’s disease is a long-term brain disorder that mainly affects movement. It happens when certain brain cells, especially those that produce a chemical called dopamine, begin to die.
Dopamine is important for smooth and controlled movement, as well as motivation and decision-making. When these cells are damaged, people may develop symptoms such as tremors, stiffness, and slow movement.
In this study, researchers focused on an enzyme called LRRK2. Enzymes are substances in the body that help control chemical reactions. In some people, a mutation in a gene causes the LRRK2 enzyme to become too active. When this happens, it interferes with how brain cells communicate with each other.
The researchers found that this overactive enzyme disrupts communication between dopamine-producing neurons and nearby support cells in a part of the brain called the striatum. This area plays a key role in movement, habits, and decision-making.
The study, published in Science Signaling, was led by Dr. Suzanne Pfeffer and involved collaboration with scientists in Scotland. The team used mice that carried the LRRK2 mutation to study what happens inside the brain.
They discovered that one of the main problems involves tiny structures on cells called primary cilia. These structures act like small antennas, helping cells receive signals from their surroundings. When the LRRK2 enzyme becomes overactive, many cells lose these cilia.
Without these “antennas,” the cells cannot receive important signals. One of these signals is called the “sonic hedgehog” signal, which helps cells release protective substances that keep brain cells healthy. Without this signal, brain cells become stressed and are more likely to die.
To try to fix this problem, the researchers used a special molecule called MLi-2, which blocks the activity of the LRRK2 enzyme. At first, they treated the mice for two weeks, but they did not see any improvement.
Instead of giving up, the team extended the treatment to three months. This longer treatment made a big difference. The primary cilia grew back on the cells, allowing them to receive signals again. Communication between brain cells improved, and the protective signals were restored.
As a result, the stress on dopamine-producing neurons decreased. Some signs even suggested that damaged neurons were beginning to recover. This is important because it shows that the treatment may not only slow the disease but could also help repair some of the damage.
Dr. Pfeffer explained that this finding could change how Parkinson’s disease is treated in the future. If doctors can block the harmful enzyme early enough, it might be possible to slow down or even stop the disease before severe symptoms appear.
This is especially important because early signs of Parkinson’s disease can show up many years before the well-known movement problems begin. These early signs can include loss of smell, sleep problems, and digestive issues. If people with the genetic mutation can be identified early, they might benefit from treatment before major brain damage occurs.
The researchers are now planning to test whether this approach could help people with other forms of Parkinson’s disease, not just those with the LRRK2 mutation. There are already several clinical trials testing similar enzyme-blocking drugs in humans.
This study brings new hope to people living with Parkinson’s disease. It suggests that protecting and restoring brain cells may be possible, which could lead to better treatments and improved quality of life in the future.
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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