Promising New Treatment for Charcot Disease Discovered in American Study on Mice

An American study conducted on mice has revealed a promising new treatment for Charcot disease, a fatal condition that currently has no known effective treatment, reports AFP.

Charcot disease, also known as amyotrophic lateral sclerosis (ALS), affects about 30,000 people in the United States.

This condition causes a progressive paralysis of the muscles and generally leads to death in less than five years.

In the study published in the scientific journal PLOS Biology, the researchers stated that they have analyzed a way to target and stabilize a protein that protects cells from toxic elements.

In many cases, hereditary mutations of a gene that produces the protein in question are the ones at the origin of Charcot disease. However, these mutations can also occur without a family history.

Mutations in this gene, SOD1, lead to defective assembly of the protein, which prevents it from carrying out its tasks and disrupts the wider cellular machinery, resulting in a group of proteins that are also associated with Alzheimer’s and Parkinson’s diseases, among others .

The new treatment is a “molecular stabilizer” that acts as a “suture” and forces the protein to maintain its correct configuration, explained study leader Jeffrey Agar, who discovered and tested this tool with his team after 12 years of research. .

The molecule was tested on mice – genetically modified to be carriers of the disease – and the researchers found that it not only restored the functions of the protein, but also stopped any toxic side effects.

Its effectiveness has also been proven in research on rats and dogs. She managed to stabilize 90% of SOD1 proteins in blood cells and 60 to 70% in brain cells.

The researchers now hope to obtain approval to conduct clinical trials on human subjects.

Although to date there is no effective neuroprotective treatment for all patients, in the United States an early marketing authorization was granted in April 2023 for a drug (Biogen’s Qalsody) that targets only certain forms of the disease. AGERPRES

2024-01-31 14:26:33
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