How does AFM decide to sponsor a particular trial ?
AFM’s mission is to stimulate innovative clinical research by helping conduct clinical trials and by financing part, or all of them. Each year, AFM launches a Call for Proposals dedicated to the development of therapeutics. This program may concern the development of therapeutic products, or preclinical and clinical trials. All projects are evaluated by the Scientific Council.
However, AFM’s action is not limited to financing projects. In order to provide an impetus to clinical trials, the Association has created a service whose role is to identify innovative scientific strategies, stimulate and monitor projects. For example, as a result of studying the potential of cell therapy in FSHD, AFM received several preclinical research proposals in this field. AFM’s role then consists in helping those teams who wish to elaborate their projects before they are submitted to the Scientific Council during the Call for Proposals period.
Increasing the number of trials in humans
A total of more than 30 clinical trials either under way or in preparation are receiving AFM funding. They concern different neuromuscular diseases, but also neurological diseases and diseases of vision, blood, immune system and skin, as well as rare genetic diseases, each a “model” for a given therapeutic avenue.
These trials use different approaches: gene therapy (transfer of healthy genes or correction of genes), cell therapy (genetically modified autologous cells) and pharmacological (to act on a genetic anomaly or slow down the progression of a disease).
Within this framework, Généthon is continuing its reorientation towards clinical trials. In 2005 the laboratory became a recognized establishment for cell and gene therapies after receiving the authorisation of AFSSAPS (the French Agency for the Safety of Health Products) to produce clinical batches. The laboratory is pursuing the preparation of four gene therapy trials in humans for neuromuscular diseases and one for immune deficiency.
Généthon began its first trial in humans as a promoter on a neuromuscular disease, gamma-sarcoglycanopathy in 2006. The laboratory is setting up three other gene therapy trials for neuromuscular diseases and one for immune deficiency.
Also, about twenty trials are being carried out at the Institute of Myology. They concern both the natural history of genetic muscular disease and pharmacological, cell, gene and enzyme therapies as well as exercise retraining.
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