Finally a cure for this deadly disease?
An American study carried out on mice unveiled on Tuesday a new treatment full of promise against Charcot's disease, for the moment with no known effective cure.
Also called amyotrophic lateral sclerosis (ALS) affects approximately 30,000 people in the United States.
It causes progressive paralysis of the muscles, creating a state of confinement in the patient, and generally causes death in less than five years.
In the study published by the scientific journal PLOS Biology, a team of researchers says they have studied a way to target and stabilize a protein that protects cells from toxic elements from food or inhaling oxygen.
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In many cases, hereditary mutations in a gene that produces the protein in question are the cause of Charcot disease.
But these mutations can also occur without a family history.
Mutations in this gene, SOD1, lead to poor assembly of the protein which prevents it from carrying out its tasks and disrupts the cellular machinery in the broad sense, leading to a clump of proteins which are also linked to, among other things, cancer. Alzheimer's and Parkinson's.
Restoration of protein functions
The new treatment is a “molecular stabilizer” that acts like a “stitch” and forces the protein to stay in its correct configuration, explained study director Jeffrey Agar, who discovered and tested this with his team. tool after 12 years of research.
The molecule was tested on mice - genetically modified so that they carried the disease - and the researchers found that it not only restored the functions of the protein, but also stopped any secondary toxic effects.
Its safety has also been tested on rats and dogs.
The treatment successfully stabilized 90% of SOD1 proteins in blood cells and 60 to 70% in brain cells.
The researchers now hope to obtain approval to move on to clinical trials in humans.
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Although there is currently no effective neuroprotective treatment for all patients, advance marketing authorization was issued in April 2023 in the United States for a drug (Qalsody from the Biogen laboratory) targeting certain forms only illness.