If you’ve taken a look at the agenda for this year’s MDA Scientific Conference (and if you haven’t, take a look), then you know that the scientific community has a great deal to talk about.
Did you know those talks happen not only during researcher presentations, but before, after, in between ... in the elevators, on the escalators, in the hallways of the hotel, at the breakfast and lunch tables, and during coffee breaks? Some even begin before the conference starts, on the airplane or in the cab on the way to the hotel, or when two researchers find themselves standing next to each other in baggage claim. Many conversations will continue on the phone and via email after the final presentation ends and everyone heads home.
Conferences like these are dynamic from start to finish. People come to talk, yes. And not only to talk, but also to listen. To share, and to teach and to learn.
Today, as will be the case throughout the conference, talks centered on developing therapies for neuromuscular diseases. We all learned about a number of things — about “druggable” targets for FSHD, about drug screening in CMT, about genetic modifiers in BMD, DMD, SMA and MMD. We learned about different types of therapeutic strategies in a number of the neuromuscular diseases in MDA’s program, and we learned about the importance of biomarkers in determining the present status of disease, in predicting its course and for use in clinical trials. And that’s only a small sampling!
So, you might wonder … how can listening to a presentation about one disease be valuable to a researcher whose area of expertise is in another, completely different disease? I asked two ALS researchers that very question.
Jeffrey Rothstein, who spoke today about his work to develop an antisense-based therapy for ALS that’s caused by the C9ORF72 repeat expansion mutation, said there’s “lots” to be learned. Among other things, he said, “We learn about the wide range of rigorous approaches to translation [turning research into treatments], and we learn about new biology that may have relevance to our particular topic — for example, different ways to knock down genes, and different viral vectors.”
Clive Svendsen also spoke today, about the potential for stem cells in ALS. When I asked him about the wide range of topics, he pointed out that there are “overlaps.” For example, antisense oligonucleotides are being tested as a therapeutic strategy in muscular dystrophy, he said, “but lessons learned will be taken forward in ALS.” He also mentioned the similarities that have been identified in C9ORF72-associated ALS and myotonic muscular dystrophy, suggesting the possibility that the two diseases may have similar mechanisms. “We will use data from both,” he said, “to cross compare.”
There’s a great deal more to talk about in the next two days, and you can bet everyone here is taking full advantage of the opportunity to share and to network, to ask and discover and analyze and learn — all of which is sure to benefit neuromuscular disease research.
MDA President and CEO Steve Derks said in opening remarks this morning, MDA’s hopes for this year’s Scientific Conference are that it brings researchers together, that it generates collaboration and discussion, and that it encourages networking and cross-pollination of ideas.
If what I’ve seen already is any indication, then there can be no doubt the conference will be nothing short of a phenomenal success!
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