PARIS - Transgene has taken a gene therapy for Duchenne's disease into a feasibility study at the Institute of Myology in the La Pitii-Salpjtrihre hospital in Paris. The French Muscular Dystrophy Association (AFM - Association Frangaise contre les Myopathies) is financing the trial under the terms of a cooperation agreement signed with Transgene in November 1998.
The study, which will be followed by a full-fledged clinical trial if successful, is designed to determine whether the direct injection into a muscle of the dystrophin gene - the protein that is missing in muscular dystrophy sufferers - enables the patient to start producing the protein, and if so, whether the protein triggers an immune response. As well as determining the local and general tolerance of this gene transfer, the study will test the feasibility of the chosen delivery vehicle, naked DNA, which is thought to be better tolerated than more conventional adenovirus or retrovirus vectors.
Nine patients aged 15 and over have been recruited for the study. No treatment currently exists for Duchenne's disease, the most common form of genetic neuromuscular disease. Transgene's therapy is designed to restore muscle power and function, and subsequent clinical trials will determine, firstly, how effective it is at a local level, and later on whether it can have a broader effect.
The November 1998 agreement between Transgene and the AFM actually renewed an earlier three-year pact signed in July 1995. The current contract, which runs to June 30, 2001, and is also likely to be renewed to enable this research program to continue, provides for the AFM to provide Transgene with funding of FFr84 million (US$14.5 million), including FFr20 million in the form of an interest-free loan that will be repayable only in the event that Transgene commercializes a product arising from the collaboration. The agreement covers not only Duchenne's disease but also another form of muscular dystrophy, Becker's dystrophy.