A recent study conducted on mice has revealed a new treatment that shows promise in combating the deadly neurodegenerative disease ALS. Amyotrophic lateral sclerosis, commonly referred to as Lou Gehrig’s disease, is a condition that severely affects nerve cells in the brain and spinal cord. Currently, approximately 30,000 Americans are afflicted with ALS, which leads to progressive loss of motor and cognitive functions. Sadly, most patients do not survive more than five years following diagnosis.
Published in the journal PLOS Biology, the groundbreaking research was led by Jeffrey Agar from Northeastern University. Agar’s team focused on targeting and stabilizing an abundant enzyme responsible for safeguarding cells against the toxic byproducts produced during food consumption and oxygen breathing. Inherited mutations of the SOD1 gene, responsible for producing this enzyme, are frequently associated with ALS. However, these mutations can also occur sporadically without any family history.
When the SOD1 gene malfunctions, the resulting protein assembles into an incorrect shape, hindering its intended functions. Additionally, this misshapen protein can lead to the formation of clumps, characteristic of other diseases such as Alzheimer’s and Parkinson’s.
Agar explained that over a span of 12 years, he and his colleagues identified and tested a molecular stabilizer called S-XL6. Acting as a “stitch,” this stabilizer forces the protein to maintain its correct configuration. One significant challenge involved finding a molecular stitch that selectively targeted SOD1 without affecting off-target proteins, as these could harm the host.
The researchers tested their molecule in genetically modified mice with ALS-like symptoms. They discovered that not only did the stabilizer restore proper function to the SOD1 protein, but it also prevented the secondary toxic effects associated with the disease. Furthermore, safety tests conducted on rats and dogs yielded positive results.
The treatment successfully stabilized 90% of SOD1 proteins in blood cells and 60-70% in brain cells. The researchers are now seeking approval to commence clinical trials in humans. An investor has already acquired the rights to the patent, indicating promising financial support for the development of this potential breakthrough.
In the future, if proven effective, the researchers hope this treatment could be used in conjunction with Biogen’s Qalsody, a groundbreaking therapy that received accelerated approval from the Food and Drug Administration in 2023. Qalsody functions by reducing the number of copies of the SOD1 gene produced by the body.
The prospect of this new treatment brings hope to ALS patients and their families, with the possibility of extending survival rates and improving quality of life. Continued research and development in this field could lead to significant advancements in tackling neurodegenerative diseases like ALS.
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- Source: Coherent Market Insights, Public sources, Desk research
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