Charcot disease: After a twelve-year wait, a promising treatment finally sees the light of day
According to a recent study, scientists have discovered a cure for Charcot disease. After twelve years of research, the results show very promising data.
A glimmer of hope in the fight against Charcot disease. About one and a half hundred years ago, neurologist and professor of anatomy Dr Jean-Martin Charcot discovered amyotrophic lateral sclerosis (ALS), also called Charcot disease. As a reminder, this is a degenerative disease that affects neurons and causes muscle paralysis. To date, this pathology, which affects approximately 30,000 people in the United States, is incurable. However, researchers may have found a cure for ALS. A study published in a scientific journal found PLoS BiologyExperts have found a way to target and Stabilize proteins that protect cells from toxins from food or from inhaled oxygen.
Charcot disease: a promising new treatment
Often, Charcot disease patients have inherited mutations in a gene called SOD1, which causes the body to be unable to perform certain functions and disrupts cellular machinery. This forms a cluster of proteins that may also be associated with other diseases such as Parkinson’s or Alzheimer’s. So scientists have worked on a treatment that they describe “Molecular Stabilizer”. According to study director Jeffrey Agar, treatment a “stitch” Allows the protein to stay in its proper 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 that had been genetically modified to carry the disease. The researchers then found that the treatment restored the protein’s functions.
Treatment of Charcot disease: human clinical trials soon?
But that’s not all! The treatment did not show any side effects in the rats. To verify its safety, the treatment was also tested on dogs and rats. Here again, the results are positive. It actually succeeded in stabilizing 90% of the SOD1 protein in blood cells and 60 to 70% in brain cells. In light of these figures, scientists now have only one thought: Begin clinical trials on humans. This may offer some hope to people with ALS.
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