This year, the members of the jury have unanimously decided to give the international Sisley-Jérôme Lejeune Award to the world renowned professor Mobley to reward him for his innovative work in research on a treatment against Down Syndrome. Professor of paediatrics at the university of San Diego and doctor in sciences, he has been head of the neurosciences department of the Medicine Faculty of California since 2009. He was particularly involved in the research on neurodegenerative diseases and neurobiology in Down Syndrome. William Mobley has been the precursor in research in neurodegenerative anomalies observed in Down Syndrome, in particular GABA and noradrenaline (see lexical).
The work accomplished by William Mobley and his team these last years have enabled them to put forward three leads for a treatment for cognitive disability in Down Syndrome:
- GABA-A receptors inhibitors in the hippocampus
For this project, clinical trials are being prepared with GABA-A receptors inversed agonists, alpha 5 subunit.
- GABA-B receptors inhibitors
William Mobley’s team’s ongoing work is about trying to test its capacity at reversing the effect of the excess of GABAergic neurotransmission by inhibiting the GABA-B receptors (without of course provoking the total cessation of these neurotransmissions).
- Potassium channel Girk2 inhibitors
Recently, the team has noticed that the excess of the Girk2 gene, observed in the Down Syndrome animal models, also existed in Alzheimer’s disease and provoked neuronal degeneration partly responsible for the disorders of patients with Alzheimer. Once he had established this link, William Mobley concluded that by trying to treat people with Down Syndrome, he could also diminish the risk of these patients developing Alzheimer’s disease. This project is still in its first stage of development and the team does not yet have specific inhibitors. However, it hopes to establish a collaboration that will enable them to find and test components on mouse models for Down Syndrome as soon as possible.