Scientists have developed a small experimental molecule that could point toward a new way of treating Parkinson’s disease and several related brain disorders.
Instead of simply easing symptoms, the molecule is designed to interfere with a process believed to drive damage inside the brain.
The research comes from scientists at NYU Abu Dhabi and the University of Denver.
The study was published in Science Translational Medicine.
It focuses on a molecule called SK-129 and its ability to target abnormal forms of alpha-synuclein, a protein that normally exists in the brain. When alpha-synuclein changes shape and sticks together, however, it can form harmful clumps.
These abnormal protein deposits are strongly associated with Parkinson’s disease, Lewy body dementia, and multiple system atrophy. The conditions differ in their symptoms and progression, but all can involve damaging forms of alpha-synuclein. Scientists sometimes group them together as synucleinopathies.
Parkinson’s disease is best known for movement problems such as shaking, stiffness, and slower movement. It can also cause sleep problems, mood changes, digestive symptoms, and difficulties with thinking. Current medicines can improve many symptoms, but they generally cannot stop the underlying loss of brain cells.
That is why researchers are interested in preventing harmful alpha-synuclein from forming and spreading. SK-129 was designed to interact with the protein and prevent it from folding into the shapes that lead to dangerous clumps. In theory, stopping this process early could protect brain cells and slow the disease.
The researchers tested the molecule using several approaches rather than relying on a single experiment. They studied laboratory-grown cells, material connected with human disease, and animal models. Across these tests, SK-129 reduced several signs of harmful alpha-synuclein activity.
One particularly important result involved the blood-brain barrier. This protective layer separates the bloodstream from brain tissue and blocks many chemicals from entering the brain. While it protects the brain from harmful substances, it also prevents many potential medicines from reaching their target.
The researchers found that SK-129 could reach the brain. In mouse experiments, treatment reduced signs of disease-related damage and helped protect against processes associated with neurodegeneration. This makes the molecule more promising as a possible drug candidate, although animal results cannot show whether it will work in people.
The study also suggested that SK-129 may interfere with harmful changes involving tau, another protein associated with brain disease. Abnormal tau is especially important in Alzheimer’s disease and several other forms of dementia. A treatment capable of affecting more than one damaging protein process could potentially have wider uses.
Still, the findings should be viewed as an early step rather than evidence of a new cure. SK-129 has not yet been shown to safely slow Parkinson’s disease, Lewy body dementia, or multiple system atrophy in humans. Researchers still need to study its safety, suitable dose, possible side effects, and long-term effects.
Human brain diseases are also far more complicated than laboratory or animal models can fully reproduce. A treatment that successfully reduces abnormal proteins in mice may not produce the same benefit in patients. Clinical trials will therefore be essential before scientists know whether the approach can change the course of these diseases.
Even so, the study offers an encouraging direction. Many existing treatments for neurodegenerative diseases mainly help people live with symptoms, while researchers are searching for therapies that attack the biological processes causing brain cells to die. SK-129 is designed with that more ambitious goal in mind.
The Science Translational Medicine study shows that it may be possible to design small molecules that reach the brain and directly interfere with harmful protein formation. If later research confirms the findings in people, this approach could eventually contribute to treatments that do more than manage symptoms and instead slow the damage itself.
If you care about brain health, please read studies that eating apples and tea could keep dementia at bay, and Olive oil: a daily dose for better brain health.
For more health information, please see recent studies what you eat together may affect your dementia risk, and time-restricted eating: a simple way to fight aging and cancer.
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