Breakthrough Treatment Unveiled for Fatal Neurological Disease

Washington, DC - A potential development in the fight against the deadly neurological disease amyotrophic lateral sclerosis (ALS) has been suggested by a recent study carried out on mice. ALS, also referred to as Lou Gehrig's disease, affects about 30,000 Americans at any given time and severely damages nerve cells in the brain and spinal cord. The majority of patients pass away from the illness within five years after diagnosis, and the ailment causes a progressive loss of cognitive and physical abilities.

Photo by Tima Miroshnichenko

The study, led by Jeffrey Agar of Northeastern University, was published in the journal PLOS Biology. It targets and stabilizes an enzyme that is essential for protecting cells from toxic byproducts produced during respiration and metabolic activities. Many cases of ALS are caused by mutations in the SOD1 gene, which codes for this enzyme. Some mutations in this gene occur occasionally.

Mutations in SOD1 genes cause the corresponding protein to misfold, reducing its effectiveness and causing protein aggregates to build up—a characteristic that is common to both Parkinson's and Alzheimer's disorders.

For twelve years, Agar and colleagues discovered and evaluated S-XL6, a "molecular stabilizer" that functions like a "stitch," maintaining the proper shape of the protein. The task was to create a molecular repair tailored to SOD1 that wouldn't damage other proteins.

Tested on genetically modified ALS-affected mice, the experimental chemical reduced the secondary harmful effects of the protein while simultaneously restoring its functionality. Positive results from safety evaluations in rats and dogs were encouraging, as S-XL6 was able to stabilize 90 percent of SOD1 proteins in blood cells and 60–70 percent in brain cells.

The team's goal is to obtain approval for human clinical trials, as an investor just purchased the intellectual rights. Agar sees S-XL6 as a possible supplemental in the future to Biogen's Qalsody, an FDA-approved medication that lowers the generation of copies of the SOD1 gene. If effective, this novel treatment may give ALS patients all around the world fresh hope.

 

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Source/credits:

The Manila Times / MSN
New treatment for fatal neurological disease
Story by Agence France-Presse