Caution should be exercised. But if the essay is transformed, it could change the lives of many families. A team of researchers from Inserm, CNRS, Inrae and the University of Tours has just published promising results on a drug that would be effective in improving the interactions of patients with autism. There is talk today of Autism Spectrum Disorder (ASD), so this disease that affects neurodevelopment is heterogeneous. These TSAs reached about 700,000 people in France, not two levels of adults. However, there are no drugs to help these patients today. 20 minutes explains why this post has something optimistic (may be cautious).
What treatment is this?
In April 2022, the research team published an article in the journal scientifique Neuropsychopharmacology revealing that bromide ions represent an interesting therapeutic pathway for relieving the symptoms of autism. Knowing that potassium (which belongs to bromide ions) is already used for epilepsy in veterinarians. “We tested the effect of bromide ions on mice,” said Julie Le Merrer, co-author of the study. In autism, there is a change in the balance between arousal and inhibition of the brain. We tested this brain-inhibiting molecule on three models of autistic pathology with different mechanisms. We then performed the most accurate behavioral analysis possible of these mice. And we found a part, a restoration of the contact lengths of the nose. On the other hand, a decrease in the stereotyped behavior of animals: mice made fewer circles on themselves. The treatment also reduced the anxiety of the mice.
In summary, what treatment has allowed the animal to significantly improve three often common symptoms of ASD: difficulty interacting, repetitive movements (stereotyping) and anxiety. And this in all three types of ASD and over time.
What are the limitations of the study?
We speak well for the moment of preclinical study, so only for the animal. It remains to be seen whether human patients will react in the same way as mice. “We have a big challenge, recognize Julie Le Merrer. So far, the research on autism has not been very successful, so these first results are not a guarantee of success. »
Indeed, one study looked at the effect of balovaptan on the socialization and communication skills of adults with ASD, but the treatment was not effective. Another tried oxytocin intranasally, without success. Third failure, this time on the bumetanide. “There have been a lot of disappointments in the last two years,” said Frédérique Bonnet-Brilhault, head of the psychiatry department at the CHU de Tours and head of the Autism Center of Excellence. This discourages labs from investing in autism research, when there is a real need. »
Second limitation: Bromide ions should be used with caution. “There is no addictive effect, but after a certain dose there is a sedative effect, a disturbance of alertness, rashes,” says Julie Le Merrer. As soon as we see the first signs, we stop and they disappear. The problem is that with this drug the margin between effective and toxic dose is low. »
This means that this treatment requires close monitoring, with regular blood prices. “Not all patients with ASD are willing to accept that,” she said. More about a cherchée solution, which could be combined with bromide ions with a mGlu4 glutamate receptor activity facilitating molecule. This will improve the efficiency, give five times less doses of each molecule, so as to limit the side effects and reduce the contraction of the pursuit. »
What are the next steps?
“He will have to mount a clinical trial on adult patients,” says Julie Le Merrer. But the research team is not ready yet and has no start date for this trial.
This clinical trial, s’il était conclusive, will allow to define the prescription dose and the means of administration.. At the moment, what is being considered is an oral treatment, with drops. So easy to take. “In children under 6, it can’t be tablets, but drops,” adds Frédérique Bonnet-Brilhault. At another time, those looking for it can also test the combination of two molecules.
Why is this result hopeful?
“Autism is a field of medicine where therapeutics is about rehabilitating language and motor skills, but we are sorely lacking in a therapeutic arsenal,” recalls Frédérique Bonnet-Brilhault. The interest with this molecule is that it has a significant effect on the social aspect, an area in which one is very destitute, especially since the interventions must start early and are heavy. »
If this therapeutic path is confirmed, “this pharmacological treatment will not replace the behavioral approaches used today, but complement them,” insists Jérôme Becker, a researcher at the University of Tours. We are not going to cure the disease but make life easier for patients, especially adults ”for whom early care is rarer. “We will first target children and adults with a major disability,” said the head of the child psychiatry department.