Hospital del Mar seeks 45 volunteers to investigate first drug for people with Down syndrome

  • Health center begins clinical trial of drug that could improve cognitive function in this group

A new clinical trial will seek to validate the safety and tolerability of a new drug for the cognitive enhancement of the people with Down syndrome. would be the first drug for this group, which until now had been “orphaned by medication”. The Hospital del Mar Institute for Medical Research (IMIM-Hospital del Mar) has just launched this study, which will be the first to analyze the feasibility of a new type of approach in this field. To do this, the hospital seeks 45 people between 18 and 35 years old with Down syndrome to participate in this clinical trial. The results of the investigation will be known in spring. In Catalonia there are between 4,000 and 5,000 people with Down syndrome.

This medication is for “cognitive enhancement” of the brain in people with Down’s syndrome, so that they have “better quality of life”. The drug is based on modulation of the functioning of the endocannabinoid system of the brain. According to various studies, people with Down syndrome have CB1 cannabinoid receptor hyperactivity, and its modulation with specific inhibitors significantly improves cognitive performance in animal models. Based on this principle, French society Aelis Pharma has developed an innovative approach, through a new molecule called AF0217, the first of its kind, which acts by mimicking a mechanism of natural defense of the brain to counteract the hyperactivity of this receptor.

Also for autism and ADHD

“With this study, we will not only show that the treatment is safe and well tolerated by people with Down syndrome, but we will have tools to see if the treatment has positive effects on cognition in these people,” says Dr. Raphael de la Tour, who will lead the clinical trial. De la Torre points out that this drug, if it proves effective, could also be useful in the autism spectrum disorder Either Attention deficit disorder and hyperactivity (ADHD).

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The 45 volunteers participating in the study will be distributed among those who take the medicine (an oral solution swallowed with water) and those who will receive a placebo. They will do it for 28 days, period during which monitoring will be carried out. Before starting and at the end they will be tested for cognitive performance and an encephalogram to get a first look at how the treatment affects your cognitive abilities.

Once the results of this phase have been obtained, a new workshop, this time with various centers across Europe and with a larger number of volunteers (150). The intention of the steering team is to launch this European study next summer. The researchers’ goal is to find a treatment that improves the working memory of these people, a key cognitive deficit in this pathology.

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