Four of my favorite days of the year are the two Fridays each spring and fall when MDA’s Scientific and Medical Advisory Committees make their pilgrimage to MDA's national headquarters in Tucson to discuss and debate the merits of hundreds of research grant proposals submitted for MDA funding. To a science nerd like me, sitting in a room packed with such brainpower is like a kid’s day at Disneyland! By the end of the day, I’ve gotten a good preview of new research projects and new researchers I’ll be following over the next several years. I’ve also had a chance to catch up with the people I admire most in the world: the scientists who are ushering the therapies for all of the neuromuscular diseases I care so much about. How lucky I am to be an eyewitness — and in a small way, maybe even a contributor — to history!
If SAC and MAC Fridays are my day at Disneyland, then the MDA Scientific Conference is my long weekend on a tropical island! No, no, don’t worry — I haven’t skipped out to Virginia Beach to down Coronas. But I confess, it’s a little intoxicating to be immersed in the sheer volume of amazing science at this meeting. It’s the one chance I get every two years to meet with all the researchers whose work we’ve been following and supporting. For me, the MDA Scientific Conference is a celebration of how far we’ve come, and even more exciting, a glimpse of where we’re going in our race toward cures and therapies.
And oh, how far we’ve come! At the last MDA Scientific Conference two years ago, keynote speaker Professor Steve Wilton reported on how exon skipping, the therapeutic technology he pioneered 15 years ago, revealed a glimmer of promise in early-stage clinical trials for Duchenne muscular dystrophy. Boys with DMD whose shin muscles were injected with an exon-skipping drug began to show dystrophin protein in their muscle fibers, where none had been before. This year, we’re hanging on every word reported from clinical trials for two different exon-skipping drugs, which both show signs of preserving walking ability in young trial participants! Almost daily, we’re seeing positive reports on therapies being tested for so many other diseases like spinal muscular atrophy, ALS, myotonic dystrophy, Friedreich's ataxia and myotubular myopathy. I can’t wait to hear the latest on these efforts at the conference.
Looking at the agenda, this conference is packed with science aimed toward developing therapies. And yet, I know that the talks and poster presentations are just the tip of the iceberg. The best part of these conferences happens between the talks, over lunch and dinner, or maybe during a polite whisper between audience members in the midst of a talk. It’s the individual conversations between scientists, following perhaps different lines of research — maybe even different diseases — who have discovered that they may have some mutual interests. Maybe a lab technique that one uses routinely and the other needs to start using. Maybe a quizzical observation made by one scientist, which rings a bell in the other. That’s what this conference is all about. Our goal at MDA is to stir the pot — to spark collaborations and get researchers to think outside of their comfort zone. I’ve watched that happen, and it is SO cool!
See you around at the meeting and in my next post …