Perhaps (finally) good news for all people with Charcot disease and their entourage. As a reminder, Charcot disease, or amyotrophic lateral sclerosis (SLA) is a progressive neurodegenerative condition which mainly affects motor neurons. The latter command voluntary movements, and their progressive destruction leads to generalized paralysis, making everyday gestures, walking, speaking, breathing, more and more difficult.
In France, around 5,000 people live with SLA, and 1,000 new cases are identified each year, according to public health figures France.
For a long time, the causes of this incurable disease have remained vague, which considerably complicated the development of effective treatments.
Until recently, research focused on genetic or environmental tracks, without any unique trigger factor being formally established. This new discovery, published in April 2025 by CNRS, Inserm and Sorbonne University teams, may well change the situation.
But then, what are the causes of Charcot’s disease?
What researchers have discovered: the role of microglies
So far, we have known risk factors. Certain genetic mutations (such as those of SOD1 or C9orf72 genes), environmental factors (exposure to pesticides, heavy metals, etc.), but nothing made it possible to designate a direct cause of the disease.
However, researchers from the CNRS, Inserm and the Sorbonne University, have identified a very serious track: the microglies. These brain immune cells are normally responsible for protecting neurons. But in people with ALS, they become too active, and this overactivation triggers chronic inflammation in the central nervous system. Worse: they end up attacking the motor neurons they were supposed to defend.
The causes of Charcot’s disease finally understood?
This discovery is crucial, because it is the first time that a precise immune mechanism has been identified as a potential trigger of the ALS. We therefore understand better how some people, without family history, develop this pathology. We are talking about the sporadic form of the disease here, which represents around 90 % of cases.
-For researchers, this deregulation of microglies would be linked to a combination of genetic and environmental factors, which could explain why some people are more vulnerable than others.
A scientific advance that gives hope
Towards a targeted treatment?
Until today, therapeutic solutions against Charcot’s disease were very limited. Two drugs are available in France, including riluzole, but their effectiveness remains modest: they only slightly slow down the evolution of the disease. Faced with this, this new track opens much more concrete perspectives.
By specifically targeting dysfunctional microglies, researchers hope to develop molecules capable of slowing down, or even interrupting the neurodegenerative process. Clinical trials are in preparation, and could start in the coming years. If they are conclusive, this could radically change the lives of people with ALS.
A human mobilization at the height of the stake
Meanwhile, solidarity around SLA continues to grow. In Isère, five friends climbed the 4,061 meters of Grand Paradis, in the Italian Alps, to raise awareness of Charcot’s disease and collect funds for research.
These actions are precious. Because if research is advancing, it needs means. And visibility. The scientific advance of CNRS researchers, Inserm and the Sorbonne University is likely to justify these means …
NAMELY
Inserm researchers have discovered that sleep disorders can precede the appearance of the first motor symptoms of SLA. In question: a dysfunction of orexin neurons, involved in the regulation of sleep.
