Sylvie Brandicourt knew that the threat was hanging over her head: her father died of Charcot’s disease and several members of her family suffered froma very rare and hereditary form of this neurodegenerative disease. The diagnosis made in 2019 therefore did not surprise her. She had arranged her house in Boën-sur-Lignon accordingly. On the other hand, the very latest decision of the High Health Authority (HAS) dismayed her: authorization refused for this treatment that she has been following for two years.
She receives this treatment in the form of an injection into the spinal cord every month at the Saint-Étienne University Hospital. It was developed by a American laboratory and is based on a molecule which acts on a protein responsible for the degeneration of neurons.
“It is indisputable, the disease has stabilized for two years. I do the same things as before,” says this 63-year-old woman who still drives her car and travels dozens of kilometers on her electric tricycle. Of course, she measures her efforts, uses her wheelchair or her crutches. Of course , she knows that amyotrophic lateral sclerosis, the medical name for Charcot’s disease, is still there. But in fact, she remembers very well the end of her father’s life, diagnosed in the mid-1980s. “He was already there. completely stuck in her chair after 5 years”, remembers Sylvie who can compare with what she experiences. “It’s been 5 years for me too but I’m still very active. Morally, it still does something to know that we are gaining years of life expectancy and quality of life.”
So when she learned that the HAS refused to authorize her treatment, “the world collapsed”, confides Sylvie, who also thinks of her two children and her two grandchildren. The French health authorities point out the insufficiency of the studies to demonstrate the interest of the drug, developed by the Biogen laboratory “not likely to fill a medical need insufficiently covered”.
A petition with more than 46,000 signatures
The only published study focused on patients at a very advanced stage and this form of Charcot disease affects very few patients. “I am living proof that it works. I don’t understand why we don’t take patients’ words into account. If only there were another drug that would be more convincing. But no, there is nothing. That’s what’s terrifying.”
The laboratory formed a appeal which will be examined at the end of November. The association for research on amyotrophic lateral sclerosis (ARSLA) counts more than fifty people benefiting from this treatment. She started a petition where she addresses Emmanuel Macron directly: “Mr. President, hope is there, within reach, and France, until now a leader in the field of rare diseases, is in the process of losing it. divert”.
For her part, Sylvie wrote to all the parliamentarians of the Loire. Some responded very quickly, others did not. THE senator Jean-Claude Tissot planned to raise the subject during questions to the government.
