Final Update from PPMD's One Voice Advocacy Summit

The third panel for the One Voice Summit, moderated by Debra Lapin, focused specifically on the accomplishments and possible gaps in the MDCC Action Plan.  Members of industry and researchers made up the panel.  Jasbir Seehra and Chris Garabedian represented industry while Se-Jin Lee, and Eric Hoffman represented research.  Bob McDonald is a parent PPMD Board member who also was on this panel.  


The discussion began with Eric Hoffman’s review of the Action Plan.  He looked over the Plan’s 67 Objectives in some cases comparing accomplishments work done by TREAT-NMD in the European Union.  Eric ranked work on mechanisms in DMD high, Diagnostics medium and Therapeutics and Research Infrastructure being done better by TREAT-NMD explaining that due to systems in the EU and with TREAT-NMD making much difference.  The discussion turned to bringing drugs to trial, developing outcome measures and clinical endpoints and standardization of many of these elements and processes to improve success.  Including patent reported outcomes as possible measures in clinical trials, provided it was deemed useful to regulators was mentioned.  Infrastructure needs, engaging big pharma and application of studies in other indications was discussed.  


During the question and answer part of the session there was talk of parent’s concern with some clinicians not being as aggressive and seeking other providers.  The response was the CDC Care Considerations makes recommendations, yet in most cases it doesn’t mandate specifics, so what is important that parents must be confident that what is recommended is safe and effective.  There was discussion of the relevance of stabilization of progression in a clinical trials and developing outcome measures for nonambulatory patients.  The group was told that industry understands the need for urgency and that companies are working to push as much as possible realizing it will never be seen as enough.  


The final panel involved Glen Nuckolls and John Porter who are program directors at NIAMS and NINDS respectively with Sharon Hesterlee moderating the session.  There are limits on what could be discussed as federal employees are not allowed to advocate for funding in any manner.  Glen and John described current work at NIH related to muscular dystrophy, collaboration with PPMD and MDA to support investigators who aren’t able to receive funding due to budget limitations and how this has resulted in successes for some of these investigators who later received large grants from NIH.  They spoke in general terms as to what reduction in budgets might mean for the funding of future grants stating that NIH Director Francis Collins commented that they are currently able to fund about 20% of grants and a reduction would me that figure would drop.  John and Glen are still committed to this community and spoke about revisiting the MDCC Action Plan this year and that they took many notes from this meeting which will help in some of the decisions to be made in the redevelopment of the Plan.


Lee Sweeney wrapped up the Summit meeting saying he thought this was an effective forum for all involved.  He reminded the group that in 2000 little was being done by federal research agencies for DMD and it was very bleak.  Now we have a new set of problems, yet these are reason for optimism as many of these problems are related to advances in lifespan and opportunities for clinical trials.  Lee urged the group to express gratitude to Congress for what has been done, yet there is a real danger that cuts will have a negative impact on progress and as a community we need to convince our Members this is important and it crucial to retain funding to avoid losing ground.


Thank you.


Brian Denger

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Comment by Kimberly Galberaith on February 14, 2011 at 10:45pm

Thanks Brian for taking such great notes and keeping everyone informed on what was happening at the meeting. 

We appreciate all of your help- and I know others appreciated reading your posts. 



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