Other centres :

In order to encourage the application of innovative therapies in humans, the AFM supports the emergence of new centres and the development of competence clusters around gene and cell therapies while associating both public and private initiatives. Moreover, it continues its support for existing centres (Généthon, Institute of Myology and I-Stem).

• Production platforms

The AFM has invested heavily in the setting up of vector or cell production centres, particularly for clinical use. Thus in 2007 Généthon obtained approval from Afssaps  (the French Drugs Safety Agency) for its second GMP (Good Manufacting Practices) production site. The laboratory, more than 90% financed by the AFM, is now structured for the preparation, transformation, conservation, liberation and distribution of gene and cell therapy products for therapeutic use in humans. Généthon was the first non-pharmaceutical laboratory to benefit from this approval and is manager of an ambitious project for the creation of a preindustrial production centre (Gamma Project) at the Evry site. Given that there is a widespread need at a European and international scale, the AFM is also supporting the construction of Atlantic Bio GMP (ABG), a viral vector production platform at Nantes for phases I/II gene therapy trials with particular emphasis on rare diseases. The start-up date for this platform is early 2009.

This investment is in line with the AFM’s long-standing support for the gene therapy laboratory of the University Hospital of Nantes and its vector production centre for research (2 600 batches produced in 10 years).

Furthermore, following a partnership with Myosix, myoblast batches for cell therapy have been placed at the disposal of research teams.

Also, at the Institute of Myology the AFM supports a platform of cell culture capable of producing immortalised lines of myoblasts for research.

• Centres of clinical investigation

The AFM has also contributed to the setting up of biotherapy-integrated  Clinical Investigation Centres (CICs), launched at the initiative of Inserm and the DHOS (Department for Hospitalisation and Organisation of Care). Five of these centres (at Nantes, Montpellier and the Necker, Saint Louis and Henri-Mondor Hospitals) are financed by the Association for the reception of clinical trials for rare diseases.

• Screening platform

The AFM supports a major pharmacological screening platform for neuromuscular diseases (CNRS in Lyon and Inserm in Paris). This uses the C. elegans model (a nematode parasite worm) – which shares more than 50% of its genome with humans – in order to research in the existing pharmacopoeia and chemolibraries for its active ingredients and characterise its mode of action. Thus in 2007, twenty potentially useful molecules for Duchenne myopathy were identified and are being validated in the mouse.
Furthermore, I-Stem – one of the spearheads of the AFM – is equipped with an automatic system of cell culture which enables the screening of pharmaceutically-useful chemolibraries. This robot can do 10 000 to 40 000 tests per screening campaign.

• Veterinary schools

Partnerships have also been formed with the Nantes and Maisons-Alfort veterinary schools for the preclinical development of rare diseases (myopathies, mucopolysaccaridoses, retinitis etc) on animal models.

• Databases

The Association supports the development of databases for gathering clinical and genetic information about the different mutations in neuromuscular diseases. These bases constitute a vital tool in the choice of eligible diseases and patients to include in a therapeutic trial, as well as in the elaboration of evaluation criteria for the trial. Thus the Universal Mutation Database (UMD), created with the AFM’s support, has become the system of reference used by the TREAT-NMD network of excellence.

• GIS-Institute for Rare Diseases

Apart from financing therapeutic trials on “model” rare diseases, the AFM also supports the Institute for Rare Diseases, a scientific interest group that it helped to create in 2002 and to which it is still the major financial contributor. In particular, the Association plays a role in joint calls for tender for rare diseases, launched with the National Research Agency. Among the projects selected by GIS-Rare Diseases the AFM has contributed to financing programmes related to Crigler-Najjar disease, WAS immunodeficiency, Duchenne myopathy, congenital myasthenic syndromes and dysferlinopathies.

• Imagine Foundation

Finally, in 2007 the AFM was one of the founder-members of the Imagine Foundation. This was created at the Necker Hospital to encourage bridge-building between research and care with the aim of improving the diagnosis and treatment of children affected with genetic diseases.