‘Focused, Intense’ MDA Conference Advances Neuromuscular Disease Research

Turning neuromuscular disease research into treatments as quickly and effectively as possible was the overarching theme of dozens of formal presentations, nearly 200 scientific posters, and countless informal conversations at the MDA Scientific Conference, April 21-24.

A palpable sense of excitement pervaded the sold-out event thanks to the unprecedented number of experimental treatments in clinical trials for neuromuscular diseases, and the unique opportunity the conference provided for information-sharing and collaboration among scientific professionals from many disciplines.

“This is one of the few times that researchers from the different fields of muscular dystrophy get together and compare notes in one place at one time,” commented Ken Hensley, an ALS researcher from University of Toledo Medical Center. “It’s a very focused, intense meeting where we can share the latest in applied and basic biology.”

Conference attendees included a number of younger researchers.

The biennial conference, held for the first time in Washington, D.C., drew more than 500 participants from the academic, corporate drug development and clinical arenas. (The conference was not open to the general public.) A larger-than-ever number of young researchers were among the attendees this year — a statistic cited by more than one participant as a positive sign for the future of neuromuscular disease research. 

Sharing ideas across diseases a priority

Capitalizing on MDA’s unique status as an umbrella organization covering more than 40 neuromuscular diseases, the tightly packed agenda focused on common research themes rather than individual diseases.

Sessions were built around such themes as targets for drugs, genetic modifiers, therapeutic modalities, biomarkers, animal models, preclinical work and clinical trials.

Each session featured presentations from researchers working in different neuromuscular diseases, enabling the widest possible sharing of knowledge and ideas.

There was “a real sharing of unpublished data,” said Kay Davies of the University of Oxford, who is working on a treatment for Duchenne muscular dystrophy (DMD). “People [were] willing to give their ideas and really fight each other to find out which is the best way. Good competition for the same goal. That’s the way we’re going to succeed.”

A rapidly changing research landscape

A significant amount of research data, published and unpublished, was shared during the two-and-a-half day conference, both from completed trials and trials-in-progress.

“[Research] is going exhilaratingly fast,” said Christopher Pearson of the Hospital for Sick Children at the University of Toronto, who is studying a type of genetic mutation underlying several diseases in MDA’s program, including myotonic and oculopharyngeal muscular dystrophies and Friedreich’s ataxia. “In fact, it’s going so fast that I could spend much of my time reading other people’s work instead of doing my own work. It’s a very exciting time.”

"Cross talk" among researchers studying a variety of neuronmuscular diseases was a hallmark of the conference.

Many speakers made reference to the rapidly changing landscape of research, which has been altered by such factors as technological advances, improvements in clinical trial design, interest by the drug development industry, and the increasing power of the patient’s voice. A commonly heard refrain was “we couldn’t have held this conference five years ago.”

“Many pharmaceutical companies unexpectedly are now targeting ALS,” said Jeff Rothstein, director of the Brain Science Institute, and the MDA/ALS Center, both at Johns Hopkins University. “That wasn’t the case a few years ago. They’re very interested in the better science that we can do in ALS today — that is, using cells to discover therapies, using cells to find biomarkers so they can intelligently design trials.”

“It’s a very hopeful time,” agreed Hensley. “Not only is there incredible scientific knowledge that’s blooming forth about the basic origins of these diseases … but there really is a change in the landscape in the way clinical trials are designed, the way that regulatory committees and agencies look at orphan disease therapy development — a whole ‘sea change’ in the way pharmaceutical companies are viewing orphan diseases as an attractive target for new therapeutic technologies.”

Poster session award winners

In addition to more than 70 session presentations, some 200 scientific posters were displayed at the conference, detailing the results of experiments and trials in a wide range of diseases.

Several posters by research trainees were selected for awards:

About the MDA Scientific Conference

Conference co-chairs Eric Hoffman (left) and C. Frank Bennett presided over the event.

Co-chairing the conference were C. Frank Bennett, CEO of the biomedical company Isis Pharmaceuticals, and Eric Hoffman, director of the Center for Genetic Medicine Research at Children's National Medical Center in Washington, D.C.

The event was made possible by the generous support of a number of sponsors.

To learn more about the conference, see:

MDA holds scientific conferences in odd years and clinical (health-care-focused) conferences in even years. (Visit the MDA annual conference series home page for an overview of the series.)

In summary

The many reasons for holding an MDA scientific conference were underscored on the last day of the event in a speech made by Vance Taylor, 35, a homeland security consultant in Washington, D.C., who has LGMD.

Taylor showed conference attendees photos of his wife and two children, and then declared: “I want you to give me 50. Not 50 push-ups. Fifty years. I need to make it to my 50th birthday.”

In order to cover the large amount of science reported at the conference, in the coming weeks MDA will be posting new articles and updating older articles with new information. Be sure to check mda.org for the latest neuromuscular disease research news.

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