More than ever this year, as a result to its achievements, AFM-Telethon relies on the French citizen’s generosity to collect all the money requested to accelerate the research in gene and cell therapie. An area where this patient group is an historical precursor.
Téléthon 2015 famous media event will be launched on the French Television public media group and all over the country this December 4th. This marathon combines a 30 hours TV show and a unique public involvement. Thanks to the combined energy from volunteers, searchers, families, partners and donors, AFM-Telethon could create the Biotherapies Institute for Rares Diseases (BIRD) which is at the forefront of the therapeutical innovation in France and worldwide.
That’s the reason why a scientific press conference was held last december the 2nd at the Myology Institute (Paris) which is part of the Biotherapy Institute as a translational research lab and an international reference hub in muscle and its diseases. There, searchers funded by AFM-Telethon drew up an exciting report: innovative biotherapies clinical trials have multiplied all over the world and outcomes speed up.
First, Pr Philippe Ménasché (Georges Pompidou European Hospital, AP-HP, Paris, France) presented the promising results of a cell therapy clinical trial based on human embryonic stem cells conducted in patients with severe cardiac failure. Indeed, this treatment came out from Michel Puceat’s work in 2007 when he was working at I-Stem, another BIRD’s member and international leading R&D lab.
Promising results as well with a gene therapy clinical trial in Wiskott-Aldrich syndrome presented by Marina Cavazzana (Necker Hospital, AP-HP). And promoted by Genethon which is also part of BIRD.
Indeed, 6 children treated for a rare and severe immune deficiency could recover a functional immune system.
And read more : Généthon, Winner of the World Innovation Competition 2030 (french)
Federico Mingozzi, head of the Genethon (Inserm) Gene Therapy and Immunology team dedicated to the liver diseases, hopes to be as successful. This team studies a gene therapy for the patients with Crigler Najjar, a rare liver disease. In this case, it’s about bringing the gene-medicine straight into the patient blood.
Overall, let’s remind that the Telethon donations, notably, allowed for the creation of I-Motion in 2015 : a clinical trial center dedicated to young patients suffering from neuromuscular diseases and located at the Trousseau Hospital in Paris (France). Besides, 37 clinical studies (running or coming soon) have been funded by AFM-Telethon, with improvements showed for patients. As well as more than 230 research plans and young searchers were supported in 2014.
Finally, Telethon 2015 TV Show is to be focused on energy. That’s why the Biotherapies Institute’s scientific teams will dedicate their time and healthy muscles to diseased ones. Meaning that they’ll come and ride bicycles during the TV show broadcast from Paris in order to enlighten the giant 3637 phone number posted on the famous Montparnasse tower facing the Eiffel tower.
