January 09, 2003

ON A NEW DIRECTION:
TRANSLATIONAL RESEARCH

In 1941, faced with the challenge of World War II, Winston Churchill declared, “Give us the tools, and we will finish the job!” In 2003, MDA is determined to give our researchers the tools they need to conquer neuromuscular disease by expanding our research program to tackle some of the logistical problems that have slowed the development of therapies.

Since MDA’s founding, we’ve sponsored research to uncover the causes of neuromuscular diseases and subsequently to develop treatments and cures for each disorder in our program. That goes for the well-known MDA-covered disorders such as the muscular dystrophies, spinal muscular atrophies and amyotrophic lateral sclerosis (ALS)… and applies equally to numerous diseases that may not be as familiar to the public but which are nonetheless formidable and potentially life-threatening, such as Friedreich’s ataxia, acid maltase deficiency (Pompe’s disease) and the mitochondrial myopathy that affects our 12-year-old National Goodwill Ambassador, Mattie Stepanek.

From our Association’s beginning, MDA looked to the real experts in the field to determine how research funds should be allocated. MDA created two advisory committees. The Scientific Advisory Committee is composed primarily of academic scientists and helps direct MDA’s commitment to basic research, seeking new understanding of the cellular and molecular processes underlying muscle-wasting disorders. The Medical Advisory Committee fulfills a complementary function, steering MDA’s preliminary clinical investigations of potential new treatment methodologies.

Now the success of these ongoing investments in basic and clinical science has led us to the point where cutting-edge techniques to replace faulty genes, or “re-seed” muscles with new cells, for example, have crossed the line from the theoretical to the possible. In 2003, it’s time to make these ideas, not just possible, but practical.

Recognizing the need for a new type of research program to meet the needs of a new type of research, this year we’re implementing a translational research initiative — a research program that will work outside the box to deal with the practical realities of translating good ideas from the laboratory into therapies available widely to those served by MDA.

The translational research initiative will be overseen by a Translational Research Advisory Committee, or TRAC. Members of the TRAC will be seasoned individuals possessing expertise in the business side of the pharmaceutical and biotech industries as well as in scientific and clinical research. Their goal will be to complement the ongoing work of the Scientific and Medical Advisory Committees by guiding funding of the development of specific tools and infrastructure needed to turn the work generated by these groups into viable therapies.

For instance, this new translational research initiative may be used for such diverse projects as generating mouse models of various neuromuscular diseases, funding the development of a facility at a university to produce modified viruses for gene therapy, or contracting directly with industry to conduct the regulatory studies required by the Food and Drug Administration before approving a new therapy. A key difference between the new translational research program and the more traditional approaches of the Scientific and Medical Advisory Committees is that the TRAC will identify needs proactively and solicit grants rather than evaluate the projects that investigators choose to propose to MDA.

We hope that the combination of this new approach to funding research along with our more traditional approach will minimize the gaps in understanding that exist among academia, the government and industry and thereby maximize the speed with which we advance toward treatments and cures. In the coming months you’ll read more in Quest magazine and on our Web site about the TRAC and the new projects that this group will inspire.

And while our research program may continue to evolve and adapt over time, one thing that remains rock-solid is MDA’s commitment to explore every avenue to seek definitive answers for families fighting neuromuscular diseases. We’re giving our researchers the tools they need to finish the job.

With every best wish...

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