Treatments and therapeutic innovation are born out of the knowledge of genes and the understanding of the mechanisms causing disease. Beyond its three pioneering laboratories combined within the Biotherapies Institute for rare diseases, AFM-Telethon has been developing collaborations for many years with scientists from all over the world.
Funding as part of a call for proposals policy
AFM-Telethon organizes a yearly scientific and medical research call for proposals to allow it to finance research programs and young researchers (PhD students and post-docs) after assessment by its scientific advisory board. Each year the Association supports over 150 projects within this call for proposals.
AFM-Telethon also organizes theme-based calls for proposals on occasion to strengthen research in certain areas of interest. The latest theme-based calls for proposals involved “Myotonic Dystrophies”, “RNA as a therapeutic target or product”, “Breakthrough technologies in Bioproduction”.
Associative partnerships: as part of the public interest strategy, AFM-Telethon supports research programs from calls for proposals organized by partner associations and dedicated to the development of innovative biotherapies. In recent years, the association has been able to provide support to the charities Vaincre la mucoviscidose (cystic fibrosis), Retina France (eye diseases), Gêniris (pathologies of the eye), the IRME (Institute for research on bone marrow and the brain), the IFCAH (International fundraising for congenital adrenal hyperplasia), Debra France (epiderlolysis bullosa), the ARSEP (multiple sclerosis research foundation), the Kidney Foundation.
Overseas the association is also developing research collaborations as part of calls for proposals organized by patient associations (SMA-Europe, Alliance Collagene VI, Cure CMD) with financial support for these programs usually in the form of co-funding.
Furthermore, AFM-Telethon is partner to the Atip-Avenir program, a call for proposals within a partnership between Inserm and the CNRS, whose goal is to promote the mobility of young, high-level researchers and allow them to set up a team within a French structure.
At the interface of calls for proposalss and strategic programs, this new project category helps provide the support needed by teams developing innovative approaches with a higher risk level to enable them to approve a proof of principle. Support for projects that have reached this stage may continue with other types of funding. These projects may be identified in annual and theme-based AFM-Telethon calls for proposals.
Multi-year strategic plans
The association supports some twenty strategic programs that receive significant multi-year funding of up to €500 K per pear and which are generally conducted within a network of several French and international partners. They are monitored by strategic and therapeutic steering committees.
AFM-Telethon supports three strategic centers with hundreds of researchers for a renewable period of 5 years. These structures are monitored by assessment committees.
- MoThard, headed by Frédérique Magdinier located in Marseille, studies the molecular mechanisms involved in rare diseases to develop new diagnostic tools and therapeutic tools for diseases of the nervous system (sensitive and nervous neuropathies), of the muscle, and pathologies related to proteins of the nuclear matrix, such as premature ageing syndromes. Ten research teams are at work on this.
- Translamuscle, headed by Frédéric Relaix in Créteil. Devoted to degenerative neuromuscular diseases (Duchenne, centronuclear, inflammatory muscular dystrophies, SMA, FSHD), this center with 62 employees relies on the additional expertise of basic, preclinical and clinical research teams to develop new therapeutic pathways to treat these diseases. It has 41 projects and 7 technological tools.
- MyoNeurALP, headed by Laurent Schaeffer, has 18 teams working on more than 45 research projects within 4 research centers in the Rhône-Alpes region (Lyon, Clermont-Ferrand, Grenoble, Saint-Etienne). Created in 2016, it studies the basic aspects of biology of the muscle and the nervous system in normal or pathological condition from embryo development to ageing. It aims to understand the physiopathology of neuromuscular diseases and uncover new therapeutic strategies.
Databases to facilitate the organization of clinical trials
The Association supports 16 genetic and clinical data warehouses for each disease, which are vital for human trials. The establishment and development of clinical and genetic registers are essential in order to better understand the progression of the disease and to recruit patients who can take part in clinical trials.
The AFM-Telethon is also member of various international research networks:
- EURO-NMD: a leading European network for neuromuscular diseases;
- IRDiRC: an international consortium launched by the European Commission and the NIH (National Institutes of Health) in the United States to promote the development of therapies and diagnostic tools for rare diseases;
- ENMC (European neuromuscular center);
- EJP-RD (European Joint Project on Rare Diseases), a research program for rare diseases financed by the European Union