Hope for a first effective treatment for narcolepsy

Hope for a first effective treatment for narcolepsy

According to the results of a study, a promising new treatment could help people with narcoleptics. A major breakthrough that gives hope to the 20,000 French people affected by this disabling disease. All the details of this French study.

If staying awake for some people may seem like an easy task, for others it is simply impossible on a daily basis. Drowsiness is the main symptom of narcolepsy. It can be accompanied by hallucinations, weight gain, sleep paralysis or even cataplexy (a sudden loss of muscle strength). Generally, this pathology occurs between 15 and 20 years. To date, there is no treatment to cure narcolepsy but only treatments to alleviate the symptoms.

However, researchers from the Reference Center for Rare Narcolepsies and Hypersomnias – in Montpellier – could well change the daily lives of these patients. According to the results of their work published in the journal The New England Journal of Medicine, a new treatment could cure people with narcolepsy. For the authors of this study, this is a “big step” in the treatment of this neurological condition.

Patients claiming to be cured

According to Inserm, in narcoleptic patients, 80,000 neurons are destroyed. The latter lack orexin (neurotransmitters). “Narcolepsy is an autoimmune disease of genetic and environmental origin whose symptoms are caused by the destruction of a particular category of neurons, those which synthesize orexin otherwise called hypocretin, a neurotransmitter whose main role is to stimulate the waking state“, say the researchers.

For their study, the researchers therefore tested on the participants of the study, a treatment developed by the Takeda laboratory, “an orexin 2 receptor agonist“. It is a molecule “which acts as a key similar to orexin and therefore has the same effects on the organism”. Inserm adds that “if an equivalent treatment had already been tested by intravenous injection, it was for the first time given to patients in oral form”.

Of the 73 participants in this study, 17 received TAK-994 at a dose of 30 mg twice daily. In a second group, 20 patients received a dose of 90 mg twice a day. Patients in the third group (19 people) received 180 mg twice a day. As for the 17 other volunteers in this study, they received a placebo.

In a press release, Yves Dauvilliers, lead author of this study, welcomed the “spectacular results” of this work: “We didn’t just have an improvement in symptoms, for the first time, patients just felt cured.”

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A clinical trial interrupted because of too much toxicity for the liver

Although great improvements in somnolence and cataplexy were seen over an 8 week period, hepatotoxic effects (liver damage) also occurred in some patients. As confirmed by Inserm, this forced the researchers to prematurely stop their work. However, scientists remain optimistic because this treatment would “to open the door to a real hope of cure for all narcoleptics who struggle to stay awake”. “We are already working on a new agonist with a greater affinity for the orexin receptor 2, and which would therefore fewer side effects”, specifies Yves Dauvilliers.

For this researcher who led the study, even if this treatment will take longer than expected to reach the market, it is necessary to continue to diagnose this disease early. “You have to understand that sleeping in class when you’re young can reveal an underlying disease”, insists Yves Dauvilliers. “Today on average it takes 8 years to diagnose narcolepsy, and only 1/3 of patients have a diagnosis , it’s really too little!”.