2024-02-02 19:30:00
Scientists may have found a cure for Charcot’s disease, according to a recent study. After twelve years of research, the results show very promising data.
A glimmer of hope in the fight against Charcot disease. Nearly one hundred and fifty years ago, the neurologist and professor of anatomy Jean-Martin Charcot discovered amyotrophic lateral sclerosis (ALS), also called Charcot disease. As a reminder, this is a degenerative disease which affects neurons and causes muscle paralysis. To this day, this pathology, which affects approximately 30,000 people in the United States, remains incurable. However, researchers may have found a treatment for ALS. As revealed by a study published in the scientific journal PLOS Biologyexperts have found a way to target and stabilize a protein that protects cells from toxic elements from food or inhaling oxygen.
Charcot disease: a promising new treatment
Often, patients with Charcot disease have inherited mutations in a gene, called SOD1, which causes the body’s inability to perform certain tasks and disrupts cellular machinery. This creates a cluster of proteins that may also be linked to other diseases such as Parkinson’s or Alzheimer’s. Scientists have therefore worked on a treatment which they describe as “molecular stabilizer”. According to Jeffrey Agar, the director of the study, the treatment acts as a “point de suture” allowing the protein to remain in its correct configuration. And if the researchers are so enthusiastic about testing their product, it is because the results shown on mice are very promising. Indeed, the molecule was tested on mice which were genetically modified so that they carried the disease. The researchers then found that the treatment restored the functions of the protein.
Treatment for Charcot disease: clinical trials on humans soon?
But that’s not all ! The treatment showed no side effects on rodents. To verify its safety, the treatment was also tested on dogs and rats. Here again, the results are positive. It has in fact succeeded in stabilizing 90% of SOD1 proteins in blood cells and 60 to 70% in brain cells. In light of these figures, scientists now have only one idea in mind: begin clinical trials on humans. This could give some hope to people with ALS.
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