A first patient with Duchenne muscular dystrophy has received a gene therapy treatment at I-Motion, the paediatric clinical platform for neuromuscular diseases, in the context of a clinical trial conducted by Généthon. A first decisive step!
A young boy with Duchenne muscular dystrophy was treated with a gene therapy at I-Motion, in Paris. He is the first patient in an international phase I/II/III multicentre trial, sponsored by Généthon, the AFM-Téléthon laboratory.
The treatment consists of introducing into the patient’s body a shortened version of dystrophin, the protein that is deficient in patients with Duchenne muscular dystrophy. This gene therapy product, which combines this microdystrophin with a vector designed to express in muscle tissue, was developed by Généthon, in partnership with the teams of Prof. Dickson (University of London, Royal Holloway) and of the Institute of Myology (Paris). It was co-developed with the US biotechnology company, Sarepta Therapeutics.
“The treatment of this young patient is a symbolic step for Généthon, and the realisation of 30 years of pioneering research. This trial embodies the quality of the research conducted in our laboratories, in collaboration with international teams of excellence. Duchenne muscular dystrophy is a particularly complex disease, because it affects all the body’s muscles. Therefore, we must remain cautious, but we are proud that the technologies developed at Généthon are now being transformed into drug candidates that are likely to change the futures of patients” says Frédéric Revah, Généthon’s Chief Executive Officer.
“Treatments for Duchenne muscular dystrophy are cruelly lacking at the moment. We are placing a great deal of hope in this drug candidate” says Prof. Muntoni, of the Dubowitz Neuromuscular Centre (UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital, London, United Kingdom), principal investigator of the trial that will include patients in France, the United Kingdom, the United States and Israel.
Duchenne muscular dystrophy is the most common neuromuscular disease in children. It is emblematic of AFM-Téléthon’s fight, and affects 1 in 3500 boys. Although immense progress has been made in the last few decades in its medical treatment, especially with respect to cardiac problems, it remains incurable. The development of an effective treatment, making it possible to reverse the disease or stop its progress, represents an exceptional challenge!