A drug that works on symptoms of autism gives hope

Caution remains in place. But if the article is changed, it could change the lives of many families. A team of researchers from Inserm, CNRS, Inrae and the University of Tours has published promising results on a drug that may be effective in improving the interactions of autistic patients. Today we are talking about autism spectrum disorders (ASD), because this disease that affects neurodevelopment is heterogeneous. This TSA will affect about 700,000 people in France, two-thirds of whom are adults. However, there is currently no medication to help these patients. 20 minutes Explains why this post contains something to make you optimistic (but cautious).

what is the cure?

The research team published an article in April 2022 in the scientific journal Psychopharmacology and Neuroscience Revealing that bromide ions represent an interesting therapeutic method for alleviating the symptoms of autism. Knowing that potassium (which belongs to the bromide ions) is already used in the treatment of epilepsy by veterinarians. “We tested the effect of bromide ions on mice, explains study co-author Julie Le Merrier. In autism, there is a change in the balance between excitation and inhibition in the brain. We tested this molecule, which depresses the brain, on three models of autism with different mechanisms. Then we performed Then the most accurate possible behavioral analysis of these mice was. On the one hand, we observed that the duration of nose contact was restored. On the other hand, there was a decrease in the typical behavior of the animals: the mice made fewer circles on themselves. The treatment also reduced the state of anxiety in the mice.

In summary, this treatment provided the animal with significant improvement in three emblematic symptoms of autism spectrum disorder: difficulty interacting, repetitive movements (stereotypes), and anxiety. This is in the three types of ASD and in the long term.

What are the limits of the study?

We’re talking about preclinical studies at the moment, so only on animals. We will now have to find out if human patients react in the same way as mice. “We are facing a huge challenge, admits Julie Le Merrier. So far, autism research has not been very successful, so these early results are not a guarantee of success.”

In fact, one study looked at the effect of palovaptan on the socialization and communication skills of adults with autism, but the treatment was not effective. Tried another intranasal oxytocin, to no avail. The third failure, this time on bumetanide. “There have been many disappointments over the past two years, summarizes Frédéric Bonnet Briault, Head of the Department of Psychiatry at the University Hospital of Tours and Head of the Center of Excellence for Autism. This discourages laboratories to invest in autism research, when there is a real need.”

Second limitation: Bromide ions should be used with caution. “There is no addictive effect, on the other hand from certain doses there is a sedative effect, disturbance of wakefulness, rash, Julie Le Merrer list. As soon as the first signs appear, we stop and they disappear. The problem is that with this drug the margin between an effective and a toxic dose is small. »

This means that this treatment requires close monitoring, with regular blood tests. She acknowledges “what not all autistic people are willing to accept”. But we looked for a solution that could be to combine bromide ions with a molecule that facilitates mGlu4 glutamate receptor activity. This will improve efficiency, administering five times lower doses of each molecule, thus reducing adverse effects and reducing control limitations. »

What are the next steps?

“We will have to do a clinical trial in adult patients,” Julie Le Merrere confirms. But the team of researchers isn’t ready yet and doesn’t have a date to start this trial yet.

This clinical experience, if conclusive, will make it possible to determine the dose to be prescribed and the means for its administration.. At the moment, what is envisaged is an oral treatment, with drops. Very easy to take. “In children under 6 years old, it can’t be tablets, it just drops,” adds Frédérique Bonnet-Brilhault. In a second step, researchers can also test the mixture of the two molecules.

Why is this result so hopeful?

“Autism is the only area in medicine where treatment is concerned with the rehabilitation of language and motor skills, but we seriously lack the therapeutic arsenal,” recalls Frédérique Bonnet-Brilhault. The benefit of this molecule is that we have a huge impact on the social aspect, an area where we are very poor, especially because interventions have to start early and be heavy. »

If this therapeutic approach is confirmed, “this drug therapy will not replace the behavioral methods in use today, but complement them,” insists Jerome Becker, a researcher at Tours University. We will not cure the disease but make life easier for patients, especially adults “who rarely receive support early. “We will first target children and adults with a significant disability,” explains the chief of child psychiatry.

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