There are a lot of frustrations in the Duchenne community—one of the most common themes I’ve heard is “why isn’t all this research and drug development coordinated better?” The problem, of course, is that there are a lot of companies and funding groups trying to help at once—sometimes it may feel like too many cooks in the kitchen, but in the long run, I think this is a much better problem to have than a lack of interest in therapy development for Duchenne. And of course both companies and nonprofit organizations have their own goals and agendas—not all of which are duplicative. In the best of worlds these groups should fit together like an ecosystem, with each filling in gaps that wouldn’t be covered otherwise. The question is, are we a healthy ecosystem or an uncoordinated mob?
Well, if you don’t know what all the other players in the ecosystem are doing it’s hard to find your niche. In the interest of promoting the ecosystem model, at the recent CIRNG network meeting for Children’s National Medical Center in Washington D.C., PPMD held its second “Duchenne Drug Development Round Table” meeting to bring together representatives from different companies (Prosensa, Sarepta, Pfizer, PTC Therapeutics and Shire) working on therapeutics for Duchenne. At the table were also MDA, DART Therapeutics (spun out of Charley’s Fund and the Nash Avery Foundation), and CureDuchenne, and several National Institutes of Health (NIH) representatives. Because the companies present are technically business competitors, it really takes a neutral third party like a patient advocacy organization to get them in the same room and at the same table, but it’s clear that the companies see this as a welcome opportunity to compare notes to the extent that they are able. Ditto for the funding groups.
And so what did we accomplish? Jane Larkindale, Director of MDA’s Translational Research program described MDA’s efforts to develop newborn screening, a “societal burden of disease” study the organization has submitted for publication, and the new MDA patient registry which will be launched through their clinic network. I described PPMD’s advocacy efforts with the FDA, including the two meetings we’ve held with the Neurology Division regulators and I described the methodology we will use for our soon-to-be-launched risk/benefit survey. Finally, representatives from the National Institute of Health who are working with the FDA presented the draft agenda for a meeting on non-ambulatory endpoints that will take place this spring. We all received very active interest and feedback from our industry colleagues. Elliot Goldstein, COO of DART Therapeutics and Mike Kelly, Scientific Director for CureDuchenne made significant contributions to the discussion as well.
In addition to hearing about projects underway by the various funding agencies present, the industry representatives also brought up topics that were of greatest concern to them in their efforts to develop new drugs. Understanding where industry perceives barriers in the process is essential for funders who are designing programs meant to circumvent these barriers. I think most of us left that meeting feeling like we had a better understanding of all the moving parts in the “drug development landscape” in Duchenne and where each of us could play a significant role.
It wasn’t actually a round table and no one was wearing chain mail, but the essence of the idea is there—different groups collaborating around a single purpose. The group will meet again in the spring to compare notes and once again take the pulse of drug development efforts in Duchenne.
Sharon Hesterlee, Ph.D.
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