Scientists have developed a special small molecule capable of preventing the buildup of harmful proteins in the brain; this molecule works by preventing proteins from clumping together and spreading.
A team of researchers from New York University-Abu Dhabi (United Arab Emirates-UAE) and the University of Denver (USA) have recently identified a promising small molecule that could slow or stop the progression of serious brain diseases such as Parkinson's, opening up hope for treatments that go beyond symptom management.
According to research published in the journal Science Translational Medicine, the Magzoub Laboratory at NYU Abu Dhabi and the Kumar Laboratory at the University of Denver, along with international collaborators, have developed a special small molecule capable of blocking the accumulation of harmful proteins in the brain.
This molecule, called SK-129, works by preventing proteins from clumping together and spreading in the brain.
The research team tested SK-129 on various disease models, including human cells, tissue taken from patients, and living organisms. The results showed that the molecule significantly reduced the harmful effects associated with the disease in all tested models.
Notably, SK-129 also has the ability to cross the blood-brain barrier—a protective layer that makes it difficult for many drugs to reach the brain. In tests on mice, this molecule significantly reduced the development of disease-related lesions in the brain.
Mazin Magzoub, Associate Professor of Biology at NYU Abu Dhabi and co-lead author, emphasized that this is a significant step towards developing treatments that target the root causes of disease.
According to him, instead of just treating the symptoms, the current goal is to slow down or completely stop the progression of the disease.