At the 2013 MDF conference, Dr. Tom Cooper of Baylor College of Medicine presented a summary of the ninth meeting of the International Myotonic Dystrophy Consortium (IDMC). The meeting, which was held in San Sebastian, Spain, takes place every two years, bringing together scientists, physicians, clinicians and patients from around the world whose common aim is to help those with myotonic dystrophy.
We asked Dr. Cooper to answer a few questions so we could share with you what the IDMC meeting is and why it’s important.
MDF: Tell us a bit about the IDMC meeting
TC: “IDMC held its first meeting about 18 years ago and has held them every other year since. Although I wasn’t involved in the beginning, it was a natural evolution for me to get involved since my lab works specifically on research related to DM.
“The conference addresses both DM1 and DM2 and investigators working on both types attend. The sessions are focused on both research and clinical characterization of the disease, but there’s a bit more of a focus on the research side and the biology of the disease.”
MDF: What is unique about the IDMC meeting?
TC: “What I like about this meeting is that it brings together researchers, clinicians and patients. Researchers and clinicians deal with different aspects of the disease and they learn a great deal from each other at meetings like this. It’s very important to have the researchers and clinicians talking to each other to really understand what’s going on with the disease. Researchers need to know what the patient is experiencing and clinicians want some understanding of why they’re seeing different features of the disease in their patients.”
MDF: Do you find it valuable to have patients in attendance at these meetings?
TC: “Definitely. Having patients there is great because researchers who are dealing with mouse models and test tubes may not have had one on one interaction with a patient. That’s very valuable and can lend enormous insight. It is particularly important that the trainees in the research labs, the graduate students and postdoctoral fellows, meet patients and their families. This puts the work they do at the bench in the broader perspective and has a major impact on their outlook.”
MDF: What was the general focus for the meeting this year?
TC: “Much of the meeting talked about characterizing the multiple aspects of the disease, how the muscle weakness progresses, how to evaluate heart arrhythmias, issues with daytime sleepiness. It may seem surprising that the disease still hasn’t been fully characterized but that’s not uncommon. There are a select set of signs and symptoms that are used for diagnosis and to determine what aspects of the disease need to be addressed in each individual.
"Now that the strong potential for therapy is coming, it is important to have well-characterized parameters to understand the natural progression of the disease and ultimately to determine whether or not a therapy is having an effect.”
MDF: Can you tell us what you found particularly interesting at IDMC-9?
TC: “As a molecular biologist, I was particularly interested in the approaches people are taking with different types of antisense oligonucleotides (ASOs). These are small pieces of DNA that attach to the toxic RNA that cause the problems in the disease. There were some interesting presentations and discussions about the testing of these ASOs in cell culture and in mice.
“There were also presentations about research testing different chemicals for the potential for future therapies. This ranges from testing specially designed chemicals in cell culture—and even in fruit flies—to screening thousands of chemicals in cultures of cells from DM patients, with researchers looking for the right molecular changes.
“Research is like a funnel: try as many things as you can and a few things will make it through. There are many steps. We start with very basic beginning steps then we move through all the steps up to the mouse model and see if it has the potential to move into a clinical trial. There are always a lot of hurdles.”
MDF: Any final thoughts you’d like to share?
TC: “Definitely. I find the attitude at these IDMC conferences to be particularly noteworthy. The DM field is very collaborative, interactive, and congenial. We don’t always agree with our colleagues’ hypotheses but we’re willing to help test them. It’s the way science should be done. It’s impressive and not all fields this level of collaboration.”