Autism: a promising treatment

Autism: a promising treatment

In the often polemical context surrounding the management of autism, French researchers presented the results of a promising clinical trial to reduce the severity of cognitive disorders in patients with this syndrome. According to their study, published in the scientific journal Translational Psychiatry, the administration of a diuretic to children with autism leads to an improvement of some disorders characteristic of this deficiency for three-quarters of them, such as difficulties in interaction Social and communication.
“Today there are only treatments that act on agitation and aggression, which are secondary symptoms of autism, but we have nothing to treat the principal,” says Dr. Eric Lemonnier, Psychiatrist at the Brest hospital, co-author of the study.

Treatment that improves brain activity and facilitates communication

A high level of chlorine in immature neurons
The priori idea of diverting the use of bumetanide, a diuretic normally prescribed to treat arterial hypertension or edema in the newborn, was born in 2007 from the meeting of this autistic clinician with the Pr Yehezkel Ben-Ari, researcher at Inserm. 
In the past, the work of this scientist working on the developing brain has highlighted a high level of chlorine in immature neurons. “We know that anxiolytics cause, in this situation, a paradoxical reaction of excitation,” says Dr. Lemonnier. I noticed that they also have this effect on autistic people. We therefore assumed that neurons are also immature in autistic individuals, suggesting a high concentration of chlorine. “Hence the attempt to administer a diuretic, known to block the entry of chlorine into the cells.

Scientists hope to eventually develop this drug in syrup form

Efficiency in almost 80% of cases
In March 2010, the researchers launched their randomized, double-blind trial of 60 children aged 3-11 years with autism or Asperger’s syndrome. The severity of their disorders was measured, both before and after treatment, using the behavioral scale Cars. Researchers concluded that bumetanide was more or less effective in almost 80% of cases (compared to 30% in the placebo group). After three months, the mean score of the children improved significantly. The severity of autistic disorders in the treated group increased from high to medium.
“We have seen an improvement in social relations,” says Dr. Lemonnier. These children have more capacity to be in the exchange, they look better in the eyes, they do more easily what is proposed to them. “
In support of these findings, Inserm presented yesterday a small film showing an example of the progress that can be made. We see a 5-year-old boy playing the doll with a psychologist, visibly serene, while he showed himself to be fleeing and stressed before the treatment.
Over time, researchers have made further improvements. Thus, according to Dr. Lemonnier, “children are less embarrassed by sounds and are less sensitive to pain. The treatment also facilitates the usual psychological support and schooling “.
After several months of testing, Dr. Lemonnier indicated, however, that treatment had no effect on about 10% of the children. It also does not affect certain disorders, such as attention deficits, mental retardation or problems of falling asleep. It is also reversible.
The hope of commercialization
“This is not a cure of autism, but a treatment that improves brain activity and facilitates communication,” says Prof. Yehezkel Ben-Ari, aware that the announcement of these results will raise a great hope Among parents.
Indeed, although this is a very interesting first step, this clinical trial has yet to be confirmed by a multicenter study that should be launched in the coming months.
Scientists hope to eventually develop this drug in syrup form, easier to take for a child. If the efficacy of bumetanide in autism is confirmed, an application for marketing authorization will be filed for marketing in 2015.

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