In France, only one drug is available but its effects remain modest. Yet there are potentially effective new treatments that take too long to reach patients.
With Charcot’s disease, life expectancy hardly exceeds a few years. New treatments could offer invaluable months of life. But their authorization is slow in Europe, to the regret of patients who no longer have time to wait.
«When you’re sure to die, patients and some doctors are willing to take risks“said Olivier Goy, diagnosed with Charcot’s disease in 2020. This entrepreneur, founder of the financial start-up Octoberspends 3,000 euros each month to buy molecules and make its own drug preparation, faced with the almost total absence of innovative treatments authorized in France.
Charcot’s disease is at this stage incurable. It results in a progressive paralysis of the muscles which gradually makes it impossible to walk, eat, breathe or speak, creating a state of confinement for the patient whose brain and intellectual abilities remain intact. Once diagnosed, life expectancy does not exceed three to five years.
But some treatments can give a few more months of life. In France, this is the case of Riluzole, a drug with modest effects authorized since the 1990s and capable of prolonging the life of patients by about three months. But apart from this, no new treatment has been approved for more than twenty years in Europe.
In the United States and Canada, a new treatmentl’AMX0035 was recently approved. It is “the first hope we have had for 20 years: the first molecule which is aimed at everyone and which would give results of a gain in survival» of up to six months, explains Sabine Turgeman, director of the Association for research on amyotrophic lateral sclerosis (ARSLA), another name for Charcot’s disease.
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Inaccessible clinical trials
Conditions deemed unacceptable by patients and families, again with regard to the speed of progression of the disease. “We feel totally abandonedenrages Sophie Garofalo, whose brother has Charcot’s disease. “Diagnosed for five years, he does not fit into any box. He is ready to take anything, try anything. We have contacted structures to integrate clinical trials, but either it does not meet the criteria or the trials have already started“, laments this caregiver.
Still, the interest of the molecule is still uncertain. The American health authorities have chosen to approve it on the sole basis of a clinical trial on a limited number of patients, a priori too small to be certain. Its producer, the American Amylyx, has started larger trials and is waiting to find out more to request authorization in Europe. But for many patients, it will be too late since it takes about two years to carry out studies. “It’s not going fast enough. The time of illness is not that of the administrators“, pleads Sabine Turgeman.
8,000 French patients
Of the approximately 8,000 French patients, some choose, like Olivier Goy, to obtain molecules abroad to manufacture their own drugs. With health risks, black market problems or exorbitant prices. For other patients, the only way to access innovative treatments is to join a clinical trial. But you have to meet very selective criteria and run the risk of receiving a simple placebo for weeks.
Patient associations are demanding, among other things, less selective criteria for access to trials and a relaxation of the rules for early access to new drugs. “The time is very limited. In the case of Charcot’s disease and in the interest of patients, we must be able to have a slightly different device and flexibility“, estimates Alain Moussy, director of the AB Science laboratory, which is developing a treatment, the masitinib, the first results of which show gains in months of survival and beneficial effects on motor functions in some patients. “How much risk should be taken? It’s up to agencies to respond, but they can be guided by policymakers and patients“, he adds.