A team of American researchers has developed a treatment for Charcot’s disease with very promising results.
- Researchers have developed a new treatment for Charcot disease.
- It stabilizes the protein disrupted by the SOD1 gene mutation, which when it functions well, protects cells from toxic elements from food or the inhalation of oxygen.
- In tests carried out on mice, the treatment stabilized 90% of SOD1 proteins in blood cells and 60 to 70% in brain cells.
New hope is offered to people affected by Charcot disease and their loved ones. American researchers have developed a new treatment with promising results on mice. Their work on this fatal degenerative disease was presented in the scientific journal PLOS Biology the 30th January 2024.
ALS: a treatment that aims to counter the mutation SOD1
Charcot disease, also called amyotrophic lateral sclerosis, is characterized by a progressive death of the motor neurons that control voluntary muscles. Which causes progressive muscular paralysis. To date, no treatment can stop its progression and patients die within 3 to 5 years.
If neurodegenerative pathology is multifactorial, genetics can play an important role in its development. For example, in many cases, gene mutation SOD1 is in question. The latter causes poor assembly of the protein which protects cells from toxic elements from food or the inhalation of oxygen. The protein fails to perform its protective functions. It also causes a disruption of “cellular machinery”, leading to a cluster of proteins which are also linked to Alzheimer’s and Parkinson’s disease.
The researchers had the idea of targeting and stabilizing this protein. After 12 years of research, the leader of this study, Jeffrey Agar of the Northeastern University, developed with his team a “molecular stabilizer”. The expert explained to AFP that the new treatment, baptized S-XL6, forces the protein to stay in the correct configuration.
Mutation SOD1 : up to 90% of stabilized proteins
This new treatment was tested on mice carrying Charcot disease. And the results are very encouraging. Analyzes showed that the proteins of the rodents receiving the product became capable of correctly carrying out their protective functions. Furthermore, the molecule stopped the secondary toxic effects of the mutation. In detail, the new drug stabilized 90% of proteins SOD1 in blood cells and 60 to 70% in brain cells.
The team of the Northeastern University hopes to soon obtain the green light from the authorities to launch clinical trials on humans.
