«The International Jérôme Lejeune Days, a unique congress on therapeutic research for genetic intellectual disabilities ».
Pr. William Mobley, University of California, Head of the Neurosciences Department
The entry in the era of clinical trials, great interest shown by pharmaceutical industries, a high level of participation: as the interventions of the speakers of the congress, which ended on Saturday the 26th of March, are being put online, the Jérôme Lejeune Foundation paints a positive picture of the progress in research for treating genetic intellectual diseases.
Over 200 international experts presented promising results at the Pasteur Institute. From modern genetics (study of the role of DNA, exploring RNA and micro RNAs, roles of proteins and their interactions) to the creation of new targeted therapeutic options and the development of new model mice, the contacts were dynamic and fructuous.
The tenacity of researchers in the exploration of these new leads enabled to identify mechanisms that are common in various mental disabilities such as Down Syndrome, Fragile X or Rett Syndrome.
The highlight of this congress was the confirmation that research in entering a new era: the era of clinical trials. It can be seen as a turning point in history, a time when it is no longer about keeping to experimental research on animals but about the beginning of the testing, on humans, of products on genes’ expression and neurotransmitters’ anomalies.
For example, for Down Syndrome, Pr. Mara Dierssen of the Genomics Centre of Barcelona, who had launched the phase I of a clinical trial in January 2010, revealed the first results during the congress: after a month of treatment only, positive effects on the patient’s memory and psychomotricity were observed. The trial is being financed by the Jérôme Lejeune Institute. Other clinical trials were announced during the congress: One, in Denvers, was launched by Pr. Alberto Costa on a NMDA receptors antagonist (which results are expected to come out in a year’s time), the other, aiming at inhibiting GABA-b neurotransmitters with new molecules in San diego, is being prepared by Pr. William Mobley.
What’s more, the 2nd day of the International Jérôme Lejeune Days enabled to show that when envisaging any treatment, it will be necessary to carry it out along with innovating educational programmes to ameliorate Synaptic connections and enable a better action of the medicine.
One thing that is quite noticeable is the proximity there is between clinics and research but also something new: The pharmaceuticals industry’s great interest for the development of new medicine in this field. In 2004, during the 1st congress, it was not yet the case.
Finally, the last session, open to the public, brought together around 500 families and caregivers to whom was presented the progress achieved in global caring of people with genetic itellectual disabilities, in particular regarding the amelioration of sleep disorder.
In the view of these promising perspectives, the next International Jérôme Lejeune Days cannot wait another 7 years. It has therefore been decided they will take place in 2014