2024-04-25 13:22:58
After decades of research, Charcot patients remain virtually untreated. One of them, which had raised many hopes, ultimately proved ineffective, reigniting speculation regarding the best avenues once morest this disease which gradually locks the patient within his body.
• Read also: Charcot disease: a controversial treatment withdrawn in the United States and Canada
“We have had 30 years of tests (and) all have been negative,” summarized French neurologist Philippe Couratier on Monday during a conference devoted to this disease, also called amyotrophic lateral sclerosis (ALS).
“Don’t imagine that we are insensitive to the fact that we have failure following failure. When you’re on your fortieth or fiftieth, it’s a bit hard to bear,” he said in front of an audience that included several patients.
The latter are condemned to progressive paralysis which spreads throughout the body and results in death within a few years: three to five on average.
Like many neurological pathologies, first and foremost Alzheimer’s disease, treatments are almost non-existent despite decades of research.
Professor Couratier spoke in a context made particularly painful by the failure of a treatment on which patients were counting heavily: Relyvrio, from the American laboratory Amylyx.
This drug has given results considered promising in initial studies on a small number of patients. Without there being any question of curing the disease, these trials suggested the possibility of giving patients a few more precious months.
Starting point ?
On this basis, the American health authorities approved it in 2022. But this decision, taken under strong pressure from patient associations, was far from arousing unanimity, with many neurologists deeming it premature.
In fact, Amylyx announced at the beginning of the year that a larger trial had resulted in negative results and, in the process, logically withdrew its treatment from the market.
In France, the medicines agency (ANSM) had just authorized it, certainly imposing much more restrictive conditions than in the United States but showing itself to be more open to patient associations than its European counterpart, the EMA, which objected to a refusal to accept.
Have we unduly raised the hopes of patients? By approving a treatment without a sufficiently solid basis, are we lowering the standards likely to one day result in truly effective drugs?
But “we deeply regret that the hopes raised are not confirmed,” admits Professor Desnuelle, vice-president of the Association for Research on ALS (ARSLA).
It is difficult for associations not to feel this failure as a return to square one, even if progress is not completely non-existent in terms of treatments.
Complex clinical landscape
One of them, in particular, was recently approved in Europe by the EMA. This is Qalsody, developed by the Biogen laboratory from a molecule called tofersen.
However, this treatment only concerns a tiny portion of patients (less than 2%) who are affected by a very specific genetic form of the disease.
This medication, in fact, acts by rectifying the action of the incriminated gene. It seems able to slow the progression of the disease, or even in certain patients to temporarily interrupt it, even if this last point is still far from being confirmed.
Despite the small number of patients affected, these good results raise hope among neurologists because they validate the interest of so-called gene therapies in the face of hereditary forms of the disease, potentially opening the way to other successes.
The fact remains that these forms are clearly in the minority: barely more than one in ten patients. The others suffer from so-called sporadic forms, the risk factors of which are largely unknown.
What therapeutic responses can be provided to them? The question is all the more complex because Charcot disease varies greatly depending on the patient: age of onset, speed of progression, presence of cognitive problems, etc.
This complex clinical landscape explains, for several specialists, the difficulties of research. This, according to them, should better identify differences between patients.
“What we now know is that there is not one ALS but ALS,” concludes Claude Desnuelle. “You shouldn’t expect a one-size-fits-all treatment.”
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