The treatment of Charcot’s disease, now incurable, has just been considered in a new light.
A new strategy for treating amyotrophic lateral sclerosis (or Charcot’s disease) has just been developed. For the first time, researchers were interested in the purification of a protein responsible for this incurable pathology from which the famous scientist Stephen Hawking suffered, and not only in the anomaly of the gene.
“This has never been done before”
The vast majority of people who develop a rare neurological disease, such as amyotrophic lateral sclerosis, have one characteristic in common: the toxic buildup of the protein TDP-43 within nerve cells. Post-mortem data have shown that 97% of people with amyotrophic lateral sclerosis (ALS) carry these toxic protein deposits.
So the scientists decided to focus on how they work. The study’s lead author, Christopher J. Donnelly, professor of neurobiology, explains: “Instead of targeting the gene that causes the disease in a subgroup of patients, we are targeting the proteins that clump together in almost all these patients. This has never been done before.”
Toxic deposits
The team found that the TDP-43 proteins stick together, forming toxic deposits, when not protected by RNAs. Ribonucleic acid (RNA) is a biological molecule present in practically all living beings, and also in certain viruses. Cells in particular use RNA as an intermediate support for genes to synthesize the proteins they need.
Inspired by what they saw, the researchers therefore developed an artificial molecule that specifically targets TDP-43 and binds to it, like RNA. And it worked, since the TDP-43 proteins didn’t clump together.
A serious neurodegenerative disease
Amyotrophic lateral sclerosis (ALS), better known as Charcot’s disease, is a serious neurodegenerative disease that results in progressive paralysis of muscles involved in voluntary motor skills. It also affects speech and swallowing. It is a disease with a poor prognosis, the outcome of which is fatal after 3 to 5 years of evolution on average. Most often, it is the attack of the respiratory muscles which causes the death of the patients*.
Source: Inserm.
.
