Report on the International ALS/MND Symposium Hosted by The ALS Association

Over 800 researchers and clinicians converged on Orlando for the 21st Annual ALS/MND Symposium, sponsored by the Motor Neurone Disease Association, and hosted by The ALS Association.  The meeting is one of the largest meetings in the world devoted to ALS research and treatment.

The ALS Association was represented by President and CEO Jane Gilbert, Chief Scientist Lucie Bruijn, Ph.D., Vice President of Patient Services Sharon Matland, and three ALS Association National Trustees: Ellyn Phillips, Elizabeth Rosenberg and Cynthia Douthat.  During the meeting of the International Alliance of ALS/MND, which was held just prior to the symposium also in Orlando, Sharon Matland was elected to the board of the International Alliance.

The meeting had a dual focus: research into the causes of ALS and development of the best treatments and clinical care for patients.  That focus was reflected in the two-track structure with a full complement of speakers addressing each topic throughout the meeting.

“In ALS, we need to focus on both these important areas,” said Lucie Bruijn, Ph.D., member of the program committee and Chief Scientist at The ALS Association, who opened the meeting with her colleague, Wim Robberecht, M.D., from Belgium, chair of the program committee.  “We need to deliver the best possible care to people currently living with ALS, and we need to find the causes of this disease, in order to develop treatments that can halt the disease.”

On Saturday, research scientists explored new insights into ALS disease mechanisms, with a focus on the cell molecule called RNA Disruption of RNA handling within neurons, which has emerged as a potentially major factor in the disease process.  Clinicians focused on quality of life, the importance of multi-disciplinary clinics in ALS care, and the search for biomarkers to track disease progression.

On Sunday, clinicians discussed the importance of cognitive and psychological issues in ALS patients, and reviewed the challenges of designing clinical trials that give unequivocal results, while remaining short and practical to conduct.  The afternoon brought results from several recent clinical trials, including trials of talampanel, pioglitazone, and a new muscle activator called CK-2017357.  Research scientists, meanwhile, heard about new technologies for drug screening and drug delivery, as well as new discoveries in the genetics of ALS and the role that axons—the long extensions of motor neurons—play in the disease process.

On Monday, basic scientists turned to examining glia, the cells that motor neurons rely on for many of their metabolic needs.  Clinicians discussed multiple issues in care and patient monitoring, telemedicine, physical activity, and caregiver support.  They also focused on the critically important aspects of respiratory and nutrition management.

The final session brought everyone together to hear about an important finding in ALS: the identity of a new ALS gene, called VCP. In addition, there is likely to be another ALS-causing gene mutation on the same chromosome, and the extensive search for that gene was the subject of another presentation.  Finally, the meeting closed with an exploration of the concept of ALS not as a single disease, but as a syndrome, divisible into several distinct entities each with their own prominent features and separate prognosis.  Such a view is contrary to current approaches to ALS but may be more fruitful for design of clinical trials and development of new treatments.

While the formal presentations were divided by major interest, clinicians and basic researchers talked long and intensely with one another throughout the meeting, sharing insights to speed development of new treatments.

“The value of a meeting such as this is exactly in this kind of close collaboration,” Dr. Bruijn said.  “When all is said and done, everyone is committed to finding the best way to treat ALS patients, now and in the future.  That's what drives us all.”

In-depth coverage of select sessions from the meeting will be available soon on the website. Check back often for the latest updates.