As the public member of the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center Steering Committee I attended the annual "face to face" meeting recently hosted by the University of Rochester Center in Rochester, NY. A little background might be helpful as many in the Duchenne community may not realize that as mandated by the MD CARE Act of 2001 these Centers are focused on research for the nine known Muscular Dystrophies including Duchenne, Becker, Limb Girdle MD, FascioScapuloHumeral MD and Myotonic MD. The six Centers have a five year award receiving $1 million annually plus a small discretionary fund for travel and other research uses. The three original Centers at the University of Rochester, University of Pennsylvania, Pittsburgh and University of Washington,Seattle are up for renewal or replacement. Collaborative research is strongly encouraged and an emphasis is placed on Translational Research meaning moving research from the lab to clinical trial and therapeutic application.
The first several meetings I attended, by teleconference or "face to face", were more focused on the structure and operation of the Centers. Eventually more understanding was achieved and the meetings began to focus on research and collaboration. The science is challenging and while I understand much of the hypothesis for DMD research the concepts for the other Muscular Dystrophies is more difficult to grasp due to less familiarity for me. As the public member I have a vote on how funds are spent so I spent much time researching the less familiar disorders and base my decisions on whether or not I think the research would be beneficial to affected individuals and families. The first meeting was in Bethesda, MD at the NIH and subsequent meetings have been in Pittsburgh, Seattle and this May in Rochester, NY. This is the first meeting to include Volunteer Health Organizations and there were representatives from PPMD, the FSH Society and Myotonic MD organizations.
The first day for the meeting each Center described their role in the network and current research and future objectives. Again, the research is collaborative among the Centers and other outside Center researchers and focused on all the Muscular Dystrophies. Topics included disrupting the pathways to muscle deterioration, muscle derived stem cells, gene therapy, exon skipping and upregulation of compounds to slow or stop the progress of muscle wasting. Some research discussed was unpublished and each participant was asked to not share specifics to avoid compromising an investigators trust upon sharing this material. What might be interesting to learn is that some approaches that did not work in for one form of MD holds promise for another. While some of the same information is heard at the annual PPMD Conference, much is new. Program Directors John Porter and Glen Nuckolls also spoke about the NIH role in moving research from the bench to bed side. On fact shared was that while the NIH overall budget has not grown in the last year, MD research, has increased due to Translational Research awards. The largest share has been awarded to DMD research. After each Center made their presentation the floor was open to questions and further discussion.
Day two was focused on the advocacy organizations. Pat Furlong spoke about PPMD and moving research forward. Each organization was given time to speak about goals, objectives and focus and there were many similar themes, yet each is focused on a different disorder. The groups all spoke of cooperation and sharing resources.
The Wellstone program is a great opportunity to promote collaboration and share ideas for MD research. Funding is still an area that all agree needs attention, yet the level of cooperation and collaboration is very encouraging. It is my hope that the theories, experiments and practices will soon lead to clinical trials and therapy for those living with MD.
For those who lobby and are working towards the reauthorization of the MD CARE Act I hope this brief update will assure you that your efforts are appreciated by the those in the research community. Eventually much of this research will lead to improvements for those living with DMD and the importance of each of us taking what time we can to write letters, visit Capitol Hill or whatever we are able to do is beneficial.