In November 2022, Pauline Messier20 years old at the time, learned that she had amyotrophic lateral sclerosis (ALS), more commonly known as Charcot disease. The doctors announced to the young woman from Chambly (Oise) that he only has two or three years left to live. In April 2023, Pauline testified in our columns, speaking about her past, her present and her future. Future which was being drawn in pencil, at the time. “I can’t have any more plans. I feel like a time bomb, I’m slowly losing all my limbs», she confided to us.
“In great shape” two years later
Two years later, Pauline is still alive. And is “in great shape“. Invited in December on the set of the show “It begins today” presented by Faustine Bollaert on France 2, for the second time after April 2023, the young woman gave some rather reassuring news, others less so. First not very positive news: the disease is genetic and hereditary. “I learned that my father had the geneshe says on set. A year after my diagnosis, my aunt became ill with ALS. It’s complicated on a daily basis. But the luck we have is that I’ve already been there. She very quickly met my neurologist and benefited from treatment from which I benefit.”
This is where the good news comes. Pauline benefits from treatment which slows the progression of the disease. “This treatment is not for everyone, well not everyone who has ALS, but only for those who have the SOD1 gene which has mutatedshe specifies. It only affects 1% of those who have the disease. In France, there are 50 of us who have it.» Injected via the lumbar route, the treatment is painful, but allows Pauline to dream of a more distant future. “Today, I smile when I talk about itshe continues. Because it is almost unexpected in a disease as terrible as ALS. It’s something exceptional, I can think a little about my future!»
Treatment called into question by the High Health Authority
Problem is, this treatment was recently called into question by the High Authority for Health (HAS). Produced by the American laboratory Biogen, the medicine Qalsody has been authorized since December 2022 for compassionate access. It made it possible to treat 55 patients. It then obtained a centralized European marketing authorization on February 22, 2024. But last October, the French High Authority for Health refused its request for early access authorization. Clearly, the medicine can still be produced, but will not be reimbursed on French territory. Except that a single injection costs €30,000and one per month must be carried out…
For the High Health Authority, several data provided by the Biogen laboratory did not make it possible to conclude favorably on the request. “Indeed, the results of the main analysis of the phase III clinical study did not demonstrate a statistically significant difference between tofersen and placebo on the clinical state of patients after 28 weeks of treatment., indicates the High Authority in a press release dated November 8. Furthermore, the data from the Biogen laboratory study were not sufficiently robust regarding a possible favorable evolution of a biological marker (neurofilament dosage).” Stating in conclusion that the evaluation of the drug was continuing.
But on November 28, a new publication announced an unfavorable opinion on the reimbursement of the drug. “Qalsody has no place in the therapeutic strategy for the treatment of ALS with SOD1 gene mutationindicates the High Authority. The medical benefit provided by Qalsody is insufficient to justify coverage by national solidarity.”
Pauline does not understand this decision
An incomprehensible decision for those first concerned. “For what ? This is a bit the question that we all ask ourselves, whether it is the patients who benefit from this treatment, the neurologists, the various health professionals.reacts Pauline to Faustine Bollaert. We don’t really understand. What are the reasons for taking away a treatment that is incredible? We really noticed a stabilization of the disease…»
While the authorities are fighting over whether or not to reimburse a drug that could save lives, or at least years of life, Pauline continues to enjoy every day, every hour, every minute. In April 2023, she told us she dreamed of continuing her travels. Since then, she has been able to attend a Beyoncé concert, see a car race in Belgium, or even go skydiving. “I have many other dreamsshe smiled. Go see a fashion show, cross the Atlantic on a sailing boat, and learn my favorite cheese!»
