An American study conducted on mice has found a promising new treatment against the deadly Charcot disease, for which there are no known effective treatments to date.
This disease, also called amyotrophic lateral sclerosis, affects about 30,000 people in the United States, and causes gradual muscle paralysis, forcing the patient to remain at home, and causing his death less than five years after the infection.
In the study published by the scientific journal PLOS Biology, a team of researchers indicated that they studied a way to target and stabilize the protein that protects cells from toxic elements coming from foods or from inhaling oxygen.
In many cases, genetic mutations in the gene that produces the protein in question are the causative agent of Charcot disease. But these mutations can also occur even without a family history associated with the disease.
Mutations in this gene, called SOD1, lead to a defect in the process of protein synthesis, which prevents it from fulfilling its role and disrupts the cellular machinery in a broad sense, which causes the appearance of a clump of proteins linked to other diseases, including Alzheimer’s and Parkinson’s diseases.
The person responsible for the study, Jeffrey Agar, who discovered this tool and tested it with his team after 12 years of research, explained that the new treatment is a “molecular stabilizer” that acts as a “stitch” and forces the protein to remain in its correct shape.
The molecule was tested on mice that had been genetically modified to be tolerant to the disease. The researchers found that it not only restored the protein’s functions, but also stopped any secondary toxic effects from occurring.
The effectiveness of the molecule was proven on rats and dogs as well, and it succeeded in making 90% of the “Sud1” proteins in blood cells and 60 to 70% in brain cells stable.
Researchers hope to obtain approval to conduct clinical trials on humans.
Despite the lack of effective treatment to protect nerves for various patients, in April 2023, the US authorities issued prior approval for the sale of the drug “Kalsodi” (from Biogen Laboratories), which targets certain forms of the disease only.
2024-01-31 11:46:53
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