A team from NYU Abu Dhabi and the University of Denver announced on Apr. 1 the discovery of a small molecule that could slow or halt the progression of neurodegenerative diseases such as Parkinson's disease.
The study addresses a significant gap in current treatment options for disorders like Parkinson's, Lewy body dementia, and multiple system atrophy, which are caused by the buildup and spread of a neuronal protein that damages brain cells. There are currently no approved therapies that can stop or slow this process.
The research, published in Science Translational Medicine, was led by the Magzoub lab at NYU Abu Dhabi and the Kumar lab at the University of Denver. The scientists developed SK-129, a small molecule designed to block harmful protein aggregation. According to their findings, SK-129 prevents these proteins from clumping together and spreading within the brain.
Tests were conducted using human cells, tissue samples from patients, and living organisms. In each case, SK-129 reduced effects associated with disease progression. The researchers also found that SK-129 is able to cross the blood-brain barrier—a challenge for many drugs targeting neurological conditions—and significantly decreased disease-related damage in mouse models.
"This is an important step toward developing treatments that target the root cause of these diseases," said Mazin Magzoub, Associate Professor of Biology at NYU Abu Dhabi and co-lead author. "Instead of only treating symptoms, we are working toward slowing or stopping the disease itself."
The study also revealed that SK-129 blocks interactions with another neuronal protein linked to Alzheimer's disease. This suggests potential benefits for addressing complex neurological disorders beyond those originally targeted.
Although further research is needed before clinical trials can begin, researchers say their findings represent progress toward effective treatments for neurodegenerative diseases.
