17 January 2007

Four muscular dystrophy organisations have launched a 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 on 17 January (attended by MDC's research team), the organisations:
  • The Association Française contre les Myopathies (AFM) of France
  • Muscular Dystrophy Association (MDA)
  • Parent Project Muscular Dystrophy (PPMD), both of the United States, and
  • United Parent Project Muscular Dystrophy (UPPMD), headquartered in the Netherlands

officially signed a memorandum of understanding to launch the Duchenne Research Collaborative International (DRCI).
 
"This is a monumental day for the entire Duchenne muscular dystrophy community throughout the world," said Lou Kunkel, PhD, Professor of Genetics and Pediatrics at Harvard Medical School, and head of the team that discovered the gene for DMD in 1986.  “This historic collaboration...has the potential to provide a real and major boost to the efforts against this devastating and deadly disease.”
 
In forming DRCI, the organisations have made a commitment to cooperate, collaborate and communicate openly in support of projects to accelerate translational research into therapeutic options to treat Duchenne.
 
Additionally, while focused on Duchenne 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. 
 
"While this is a significant step forward for the Duchenne community, we are confident that the framework we are putting into place can be widely replicated for the benefit of patients with other neuromuscular and rare conditions the world over," said Laurence Tiennot-Herment, President of AFM. "In forming DRCI, we are clearly setting a new direction that hopefully can be used to improve collaboration and accelerate the research agenda for countless other conditions."
 
The amount of funding being invested in Duchenne research has grown significantly over the past five years.  In the United States, this is due in large part to the enactment of the Muscular Dystrophy Community Assistance, Research, and Education Amendments (MD CARE) Act in 2001.
 
That law led to an infusion of new research dollars, the establishment by the National Institutes of Health of six research "centers of excellence," and a greater focus on public health education and awareness through the Centers for Disease Control and Prevention. 
 
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 which is led by MDC-funded scientist, Prof Kate Bushby - 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 Duchenne 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 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 organisations - AFM, MDA, PPMD and UPPMD - merged their research grant databases to form a central reference for funded research grants in Duchenne.