Promising results against ALS in mice

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Professor Jeffrey Agar works in his laboratory researching treatment for ALS.

Agence France-Presse

A treatment against amyotrophic lateral sclerosis (ALS, Charcot or Lou Gehrig's disease) tested on mice has given encouraging results, shows an American study published in the journal PLOS Biology (New window) ( in English).

This deadly disease affects 3,000 people in Canada. It causes progressive paralysis of the muscles, creating a state of confinement in the patient, and generally causes death within five years.

The Northeastern University research team says it has studied a way to target and stabilize a protein that protects cells from toxic elements from food or inhaling oxygen. p>

In many cases, hereditary mutations in a gene that produces the protein in question are the cause of the 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 sense large, leading to a cluster of proteins which are also linked, among others, to Alzheimer's and Parkinson's diseases.

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The new treatment is a molecular stabilizer which acts like a stitch and binds the protein to remain in its correct configuration, explained the director of the study, Jeffrey Agar, who discovered and tested this tool with his team after 12 years of research.

The molecule was tested on mice – genetically modified so that they carried the disease – and the researchers found that not only did it restore the functions of the protein, but it also stopped any toxic effects secondary.

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

The researchers now hope to obtain the x27;authorization to proceed to clinical trials in humans.

If there is no treatment to date effective neuroprotectant for all patients, advance marketing authorization was issued in April 2023 in the United States for a drug (Qalsody from the Biogen laboratory) targeting only certain forms of the disease.