The social interactions of children with autism have been greatly improved thanks to a diuretic that has been used for over 40 years.
Hopes for treatment for autism are growing. French researchers have just shown that a diuretic, bumetanide, used since the 1970s can reduce the severity of autistic disorders in children. The results of their clinical trial are published in the medical journal Translational Psychiatry.
Dr Eric Lemonnier, child psychiatrist and director of the Limousin autism expert center at Limoges University Hospital, and Prof. Yehezkel Ben-Ari, Inserm emeritus research director, had the idea of using this drug more than 7 years ago. This reduces the concentration of chlorine in the cells. In children with autism spectrum disorder, neurons contain a large amount of chlorine, which interferes with the functioning of the neurotransmitter GABA. An imbalance which would generate the troubles of social interaction. In previous work, scientists had already shown the benefits of bumetanide in a small group of children.
Convinced by these first results, European health authorities asked the team to continue their trial. Thus, nearly 90 children aged 2 to 18 with autism or Asperger syndrome were selected in 6 French centers (Brest, Limoges, Lyon, Marseille, Nice and Rouen). These were divided into 4 groups, including a placebo group. The other 3 received different doses of bumetanide (0.5 mg / 1 mg / 2 mg) to determine the most optimal. During the 3 months of the experiment, neither the families nor the doctors knew who was being treated and who was receiving the placebo.
Authorization request in 2022
At the end of this experiment, the doctors and the parents report a clear reduction of the symptoms in the treated children. All noted improved visual communication and stereotypical disturbances. Children who have received bumetanide are more present and it is easier to get them to do activities. Observations already made during work carried out in 2012 as shown in the video below. The researchers add that the children in each group responded to the treatments, regardless of their age and autistic disorder.
Based on these results, the doctors gathered within the Neurochlore company are preparing to launch their phase 3 trial in partnership with Servier laboratories. It is expected to last a year and include more than 300 children from around the world. If the study is conclusive, the researchers hope to be able to file a marketing authorization application in 2022.
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