Parkinson’s disease is a long-term brain disorder that slowly damages nerve cells involved in movement. It affects millions of people around the world and becomes more common with age.
The disease is best known for causing shaking, stiffness, slow movement, and balance problems. However, many people experience other symptoms years before these movement problems appear. These early signs can include a reduced sense of smell, constipation, sleep problems, depression, and changes in mood.
At present, available treatments mainly help control symptoms, but they cannot stop the disease from getting worse. That is why scientists are searching for ways to protect brain cells before they are permanently damaged.
A new study from researchers at Stanford Medicine has uncovered a promising approach that may help rescue brain cells in one form of Parkinson’s disease.
The research was carried out in mice and suggests that blocking an overactive enzyme may allow damaged brain cells to recover their ability to communicate with one another. The findings were published in the journal Science Signaling.
The study focused on people who carry a mutation in a gene called LRRK2. This mutation is one of the most common known genetic causes of Parkinson’s disease. In these patients, the LRRK2 enzyme becomes too active. Instead of helping cells work normally, the overactive enzyme interferes with the brain’s communication system and gradually damages important nerve cells.
One of the most important chemicals involved in Parkinson’s disease is dopamine. Dopamine allows brain cells to send messages that control movement, learning, motivation, and decision-making.
As dopamine-producing nerve cells become damaged or die, these signals become weaker. This leads to the movement problems that many people associate with Parkinson’s disease.
The Stanford team discovered that another tiny part of brain cells also plays an important role. Many brain cells have microscopic structures called primary cilia. These tiny projections act like antennas, allowing cells to receive important chemical messages from nearby cells.
One of these messages comes from a protein known as sonic hedgehog. Despite its unusual name, this protein is essential for healthy brain function. It tells nearby support cells to produce protective proteins that help keep dopamine-producing neurons alive.
When the LRRK2 enzyme becomes too active, many brain cells lose their primary cilia. Without these tiny antennas, the cells cannot receive protective signals. As a result, fewer protective proteins are produced, leaving dopamine-producing neurons more vulnerable to damage over time.
To test whether this process could be reversed, the researchers used an experimental drug called MLi-2. This compound blocks the activity of the LRRK2 enzyme. At first, the scientists treated mice for only two weeks, but they saw little improvement.
They then decided to continue treatment for three months after learning that brain cells naturally grow and shrink their primary cilia over time.
The longer treatment produced remarkable results. After three months, many brain cells in the affected mice had regrown their primary cilia. This allowed important protective signals to reach the cells again.
The researchers also found evidence that damaged nerve cells were beginning to recover and that communication within the dopamine system had improved. The treated mice showed brain changes that looked much more like those seen in healthy animals.
According to senior author Dr. Suzanne Pfeffer, these findings suggest that blocking the LRRK2 enzyme early enough could slow, stop, or possibly even reverse some of the early changes caused by Parkinson’s disease.
Because warning signs may appear 10 to 15 years before movement symptoms develop, future treatments might one day begin long before permanent brain damage occurs.
The researchers also believe this strategy may not be limited to people with LRRK2 mutations. They plan to investigate whether similar treatments could benefit other forms of Parkinson’s disease. Several clinical trials of LRRK2 inhibitors are already underway, although much more research is needed before this approach can become a routine treatment for patients.
While the results are exciting, this study was performed in mice, so scientists must still determine whether the same benefits occur in people. Even so, the research provides new hope that future therapies may do more than simply ease symptoms. They may actually help protect brain cells and preserve brain function.
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.
The study was published in Science Signaling.
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