Strategic Planning for a Muscle Research Policy in Canada
(July 2002)


Executive Summary

Canadians are international leaders in muscle research and have helped elucidate the molecular basis of a variety of muscle diseases. However, the community is scattered and communication is poor. On July 10, the Institute of Musculoskeletal Health and Arthritis (IMHA) - one of 13 virtual institutes spearheaded by the Canadian Institutes of Health Research (CIHR) - undertook to address this issue by spearheading the “Strategic Planning for a Muscle Research Policy in Canada” workshop.

Held during the Xth International Congress on Neuromuscular Diseases, the workshop was designed to bring stakeholders and members of the scientific community together to develop a Muscle Research Committee charged with the task of spearheading a National Muscle Research Strategy. The session attracted the interest of approximately 35 individuals including new investigators, scientists, patients and representatives from various granting agencies and several of Canada’s top researchers including Dr. David MacLennan, Dr. Ron Worton and Dr. George Karpati.

Agency Overview

IMHA Board member, Dr. Ilona Skerjanc, opened the workshop with an overview of the Institute of Musculoskeletal Health and Arthritis (IMHA) and the current Requests for Applications (RFAs) that the Institute recently announced for 2002.

Muscle is one of six equally important foci areas within IMHA – arthritis, musculoskeletal (MSK) rehabilitation, bone, muscle, skin and oral health. In order to move the science of muscle forward, Dr. Skerjanc emphasized the critical need to develop a strategic plan that would build upon the strategic agendas of other agencies involved in funding muscle research. Ultimately, this would culminate in the creation of a Muscle Research Committee.

Yves Savoie, National Executive Director of the Muscular Dystrophy Association of Canada (MDAC), followed with an overview of the Association and further details regarding a partnership formed with CIHR and the Amyotrohic Lateral Sclerosis Association (ALS) to fund Post-Doctoral Fellows and operating grants. Given the number of areas of overlap between IMHA and MDAC, and the fact that MDAC expects to have enhanced research funding in place in the near future, it was recommended that a more targeted approach be taken to catalyze collaborations. Through a series of iterative meetings involving members of the Advisory Committees of both ALS Canada, MDAC and representatives of CIHR, Mr. Savoie noted that two areas of focus have already been identified including Gene and Cell Therapy and Protein Misfolding and Aggregation.

Sharon Hesterlee, Director of Research and Development of the U.S. Muscular Dystropy Association (MDA), gave a succinct overview of the Association noting that the majority of their international funding goes to Canadian scientists. She also pointed out that MDA is currently involved in a stem cell initiative designed to identify the factors leading to stem cell integration into muscle with the hope of future successful trials in a canine model leading to human phase 1 clinical trials. MDA’s research needs/priorities include:

  • Shared Resources – mouse models and other reagents; tissue banks; data sharing platforms;
  • Better Training opportunities for basics researchers and clinical investigators;
  • Mechanisms to enhance translational research by overcoming barriers - academic labs are not usually suited; industry in not motivated because the profit potential is low; and there is a lack of clinical investigators to conduct trials.

Dr. Richard Lymn, Muscle Biology Program Director for the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), National Institutes of Health (NIH) outlined NIAMS’ strategic plan for muscle biology research. (nih.gov/niams/strategicplan/musclebio.htm)

One of NIAMS’ major goals is to understand skeletal muscle as an integrated whole-body system. In the next several years, it is expected that combined use of cell physiology, genetic techniques, biophysics, whole animal measurements, and computer modeling will help generate fundamental new theories of how muscular systems are designed and function to maintain health. Highlights of their research needs and opportunities include: Muscle Genetics and Disease, Muscle Injury, Repair, and Health, Molecular Biophysics of Movement and Control, Experimental Therapeutics and Clinical Trials, Infrastructure: Foundations for the Future.

Nominations for a Canadian Muscle Research Subcommittee

Over the course of the discussion, a number of recommendations were made regarding the formation of a Muscle Subcommittee. First off, the group agreed that the committee should be comprised of representatives from British Columbia – possibly Dr. B. Bressler and Dr. Andrew Eisen from the University of British Columbia and Dr. W. Parkhouse from Simon Frasier University - as well INMA representatives and new investigators. Given that Muscular Dystropy is one of four priority research areas for The Stem Cell Centre of Excellence, the Centre expressed a keen interest in being involved in the Muscle Research Subcommittee. The Muscular Dystrophy Association of Canada was equally keen to be involved. Patients should also have a voice.

All four granting agencies agreed that the muscle community in both the United States and Canada would benefit from the synergy that would flow out of the formation of a Muscle Subcommittee. In this regard, it was recommended that a joint International muscle meeting be organized to bring together a broad range of stakeholders including IMHA, MDA, MDAC and other appropriate institutes, the Stem Cell Centre of Excellence, NIAMS, researchers and the patient public. The group also identified the need for small focused Canada-based workshops, spearheaded by external stakeholders around specific muscle disorders/issues, that would lead to the development of Requests For Applications.

Another important meeting objective was to provide IMHA with recommendations for muscle research in Canada. It was noted that there is a critical need for better coordination of RFAs and a simplified explanation of the tools – NET, ICE, IHRT. One way to achieve this goal would be to assign a contact person to answer questions about the programs. The fact that the overall direction of MDA (US) and NIH-NIAMS is similar to IMHA’s was noted and will be taken into account in future endeavors. The following recommendations were also made for potential future RFAs:

  • New emerging teams (NET) in muscle
  • Animal trials preparing for clinical trails for neuromuscular disorders
  • Drug trial in muscular dystrophies
  • Molecular Diagnosis of muscular dystrophies in Canadian founder/regional populations
  • Muscular disability of the Elderly (collaboration with aging institute)
  • The role of muscle wasting in cancer cachexia (collaboration with Cancer Institute)

From the Patient’s Perspective

Diagnostics

At least some of the prospective therapies will be gene-specific and will require that the specific gene or genes and their related proteins be identified on a molecular level before these therapies can be applied. It is important that the means to accomplish this be developed to a level that will provide comprehensive, inexpensive, and geographically available procedures - all within a reasonable timeframe. People with neuromuscular diseases often have difficulty travelling to distant centres for diagnostic or treatment purposes. Additionally, such travel is usually very costly. Gene chips or micro-arrays that are specific to neuromuscular abnormalities may need to be developed and made available to physicians and laboratories in Canada.

Delivery of Therapeutic Modalities

Some of the same considerations of distance and difficulty of accessing resources will apply to the delivery of therapies as they are developed. These can be quickly resolved if such therapies consist of taking oral medications. However, where appropriate treatment requires the infusion of genes or stem cells, the approach becomes much more complex. To address this issue, there should be a central Canadian source of modified genes or stem cells. Where, when and how the modification process will takes place, as well as how “medicines” will be made available to patients must also be addressed.

Given the degree of complexity, it will take substantial time to plan possible avenues of delivery. And because time is of the essence for many patients, we need to start planning potential delivery scenarios before the therapies are actually available.

Conclusions

The “Strategic Planning for a Muscle Research Policy in Canada Workshop” was successful in clarifying the fact that there is sufficient interest and expertise in Canadian muscle research to justify the formation of a Muscle Research Subcommittee. Next steps to achieve this goal will involve finalizing the composition of the Muscle Research Subcommittee, through discussions with the IMHA’s IAB and key muscle researchers. In order to realize the recommendations from the workshop, and to enable committee meetings to take place, a budget should also be formulated.

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