|
|
|
|
International collaborations
The setting up of a policy of international networking is a vital step in the development of therapies for diseases characterised by their rarity.
“TREAT-NMD” European excellence network
Created on 1 January 2007, the TREAT-NMD network has for objective to speed up the arrival of cutting-edge therapies. The project is centred on the technological and methodological development of the tools necessary for these therapies. Among these tools are: animal models, data and biobanks, methods of evaluation etc. The participation by small and medium sized companies is actively encouraged. This network does not consist of direct financing research and development expenses, but rather an improvement of the standardisation and better knowledge-sharing and an optimisation of inter-team exchanges. By bringing together 21 partner organisations, this network encourages experts to work together to share best practices of research and define standards of care. Scientists will work in close collaboration with physicians to test and apply new research paths in order to develop innovative therapies as quickly as possible. Close links with pharmaceutical companies will be developed to speed up their availability. Patients and patients’ associations will be involved at all levels. The majority of European neuromuscular disease specialists are taking part in this initiative – the AFM, Généthon, Inserm, Institute of Myology, Italian Téléthon, academic teams from all over Europe, different researcher and patient networks and/or researchers centred around a particular pathology, as well as biotechnology companies such as Santhera, Prosensa, Genosafe etc. The 11 countries involved in the Treat-NMD network are France, the United Kingdom, Spain, Germany, Switzerland, Belgium, Netherlands, Italy, Finland, Sweden and Hungary. The network has received a grant from the European Union of 10 million euros over a period of 5 years. The project leaders are Kate Bushby and Volker Straub, both from the University of Newcastle (UK). They have received wide support from the AFM, the ENMC and ACIES, a French consulting firm specialising in European projects. To begin with, the project will concern 3 flagship projects: Duchenne muscular dystrophy (DMD), spinal amyotrophy (SMA) and congenital muscular dystrophies (CMD). It will consist of several “activities” subdivided into programmes – “workpackages” (see complete list on the ENMC website) for example: - a communication platform
- harmonisation and creation of data, DNA and tissue banks (Eurobiobank is involved, as is Christophe Béroud at Montpellier)
- creation of a European centre for clinical trials
- a programme for animal models
- a programme for standardisation and patient management
- several programmes dedicated to the production of clinical batches and toxicology etc.
The network will be structured by the creation of a Centre which will coordinate European initiatives for the development of innovative therapies in partnership with institutions, industry and patients’ associations. It will act as an essential bridge to the sharing and standardisation at an international level of therapeutic strategies in the field of neuromuscular pathologies. The first meeting of Treat-NMD took place at the Génocentre at Evry on 18 and 19 January 2007. > To know more : http://www.treat-nmd.eu International Muscular Dystrophy Community Forms Historic Research Partnership
The world's leading muscular dystrophy organizations have launched a historic coalition that will pool knowledge and resources in an attempt to accelerate development of promising treatments and a cure for the most common childhood form of the disease. At a meeting in Paris, the organizations - The Association Française contre les Myopathies (AFM) of France, the Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD), both of the United States, and United Parent Projects Muscular Dystrophy (UPPMD), headquartered in the Netherlands - officially signed a memorandum of understanding to launch the Duchenne Research Collaborative International (DRCI), on January 17, 2007.
In forming DRCI, the organizations have made a commitment to cooperate, collaborate and communicate openly in support of projects to accelerate translational research into therapeutic options to treat DMD.
Additionally, while focused on DMD right now, the partners have a broader vision to serve as a model for collaboration in the battle against neuromuscular diseases by improving access to information about rare conditions, creating strong public/private partnerships and, ultimately, increasing the amount of funding available for disease research.
The European Union (EU) recently approved 10 million euros to aid muscle disease patients. The funding will bring together some of the world’s leading doctors and scientists in a network, TREAT NMD, aimed at improving treatment and finding cures for debilitating neuromuscular diseases.
In developing DRCI, leaders of the international Duchenne community have established a first series of three near-term goals: - A Clearinghouse for Research Investments and Research Resources. This will provide the Duchenne research and patient community with one central international repository to track research grants and the location and availability of key research resources.
- A Global Patient Registry. Through a global registry, smaller local and regional registries can be consolidated into one "super database," providing public health officials and researchers with greater ability to study a much larger proportion of the world's DMD cases. This will help ensure patients from all over the world, especially those in remote locations, have the opportunity to be selected for potentially life-saving treatment and therapy trials. A central database will help researchers locate eligible candidates for clinical trials in a more efficient manner.
- A Global Clinical Trial Network. A Global Clinical Trial Network will present a platform of support systems critical to the efficient conduct of clinical research. Through this trial network, the translational research conducted by the global Duchenne research community will be accelerated as researchers draw from shared databases and validated research resources, thus avoiding unnecessary and costly duplication of effort.
The DRCI has also created a Professional Ethics Committee which will recommend guidelines for industry and nonprofit organizations that invest time and resources to help patients with Duchenne. The committee is currently developing an ethical platform for investments in translational research in rare disease, using Duchenne as a model. Concrete actions have already been taken toward these goals. Just last month, all four organizations - AFM, MDA, PPMD and UPPMD - merged their research grant databases to form a central reference for funded research grants in Duchenne. While several promising DMD research pathways have already been identified, a number of challenges remain for the DRCI to address, including: development of superior animal models; establishment of clearer evidence-based standards of care; greater engagement of industry partners; and reduction in fragmentation of research. > Read the complete press release. The Myoamp European project
The AFM is involved in the Myoamp European project, initiated in 2006 and financed by the European Union. Myoamp brings together eight research teams from five European countries with a dual objective: develop new cell therapy strategies for Duchenne myopathy and – at the same time – define recommendations so that clinicians will be able to transfer these therapeutic strategies quickly and effectively. This three-year project should contribute to the setting up of a clinical trial in Europe, in particular within the TREAT-NMD network.
It gathers biologists, clinicians and biotechnicians of France, Italy,Sweden, Germany and England. The Myores network
Myores is a European network of excellence, financed by the European Union and dedicated to the study of the normal and pathological muscle through multi-organisational approaches. It gathers together the best teams of European researchers with the aim of speeding up fundamental research and promoting clinical applications.
The AFM is an official partner of the Myores network.
> To know more : www.myores.org Collaboration with the ICCIn September 2007 the AFM joined the ICC (the International Collaborative Committee for SMA clinical trials). This organisation brings together the main actors – patients, researchers and clinicians – in spinal amyotrophy. The ICC is particularly focused on the harmonisation of its patient database with the international database managed by TREAT-NMD. Major international academic or industrial programmesAn agreement was signed with the University of Pennsylvania (Prof Dreyfuss’s team) to make progress in the understanding of the mechanisms at work in infantile spinal amyotrophy and to develop active molecules for this disease. The fundamental research work of Prof Gidéon Dreyfuss concerns the complex role of proteins interacting with the SMN protein. A screening programme of molecules potentially active in this pathology has been set up with research into different tests capable of locating or confirming molecule activity.
Objectives:
To decipher anomalies in the interactions of the SMN protein complex in patients in order to counter them, and identify one or several active molecules in the cell models developed by the University of Pennsylvania. Programme arrived at term in August 2006 and renewed for six months.
Prospects:
Thanks to this partnership the probability of identifying potentially therapeutic molecules is optimised. Once validated, these molecules will be evaluated in murine models of the pathology before possibly undergoing clinical trials. Preclinical programme of evaluation in a transgenic mouse model of Steinert myotony, aimed at correcting muscle anomalies and validating non-invasive muscle function evaluation techniques (Puymirat project). Programme for the clinical development of myoblast transplant in Duchenne myopathy (Tremblay programme). Industrial research programme within the framework of an agreement passed with Prosensa, a Dutch biotechnology company – clinical development of exon skipping by oligonucleotides on Duchenne myopathy. Cooperation with neuromuscular associationsSeveral agreements have been made with foreign associations: > Luxembourg, ALAN
> Spain, ASEM Federation > EAMDA (Alliance of European Neuromuscular Diseases Associations)
> SMA Europe
> Latin America, MDA Peru (Peruvian Association against Myopathies)
> Mexico, Sociedad Mexicana de la Distrofia Muscular AC > Africa, Tierno and Mariam International Foundation (FITIMA) > Agreement with Handicap International for the Algeria-Maghreb programme
...
|
|
| 
Print.
Send.