For several years, the scientific community has been interested in the GABA allosteric neurotransmitter. For example, the studies led ex vivo and then in the mouse model for Down Syndrome by Pr. William Mobley, chairman of the scientific committee of the US Jérôme Lejeune Foundation, indeed showed the determining role of the inhibition of the GABA receptors in the improvement of the animal’s cognitive capacities. New therapeutic perspectives have thus been found.
The Roche laboratory is therefore carrying out a project with one of the inhibiting molecules of the GABA receptors. After the preliminary phases destined, among other things, to ensure that the molecule is not toxic, a phase carried out over several years is now starting; that of the clinical study, strictly speaking, in which the Jérôme Lejeune Institute will be taking part.
This multi-centred, international clinical study will be carried out simultaneously in France, Spain, Great-Britain, Island, Canada, Mexico, Argentina, Singapore, and New-Zealand.
Half of the patients with Down Syndrome will be between 12 and 13 years old and the other half between 14 and 30 years old: there will be 3 groups of 60 patients. Taking part in the study will include about 10 visits for each patient, spread out over forty weeks with an average of seven neuropsychological consultations. This stage will focus on measuring the level of tolerance depending on the dose and the effectiveness of the molecule.
Work memory, social interactions, speech, the neurological state, and sleep are all criteria which will be studied during regular tests.