January 11, 2002

ON MDA & THE MD-CARE ACT

As you may know by now, President Bush signed the MD-CARE (Muscular Dystrophy Community Assistance, Research and Education) Act into law on Dec. 18 (www.mda.org/news/011218MDCAREact.html.)

MDA National Chairman Jerry Lewis and former MDA National Goodwill Ambassador Benjamin Cumbo testified before a Senate subcommittee last year, urging allocation of more federal funds for muscular dystrophy research.

MDA and its supporters worked diligently throughout last year to turn this proposed legislation into a reality. Passed unanimously by both houses of Congress and signed by the President, the law will increase the government's emphasis on developing treatments and cures for the nine muscular dystrophies. Among its main provisions are that the National Institutes of Health (NIH) will establish several "centers of excellence" across the country, which will implement significantly greater government-funded muscular dystrophy research.

But our work is far from finished. Since the bill was signed, we've taken several steps to ensure that MDA has a continuing voice in the implementation of this important legislation. We're helping to set up a coordinating committee that will include representatives of government agencies, people affected by muscular dystrophy or their parents, researchers and clinicians.

This month, MDA representatives will meet with scientists and NIH personnel to establish strategic goals for accelerated MD research. The researchers are listing, for each disease, the goals scientists need to reach in order to close the gap between present knowledge and future treatments.

For example, in Duchenne MD, the idea of repairing the muscle cell membrane by substituting a functional muscle protein for one that's lacking in the disorder shows great promise. Finding methods to increase production of proteins that could perform this substitution is high on the to-do list.

In facioscapulohumeral muscular dystrophy (FSHD) research, significant changes at NIH already reflect increased activity. Among the NIH projects recently funded for FSHD are studies of affected versus unaffected muscles; of a gene called D4Z4 that may play a role in the disease; of the muscle cell membrane structure; and of how genes are turned on or off on chromosome 4 because of the FSHD genetic defect.

MDA has never sought funding from any governmental agency and isn't doing so now, as the MD-CARE Act allocates support for NIH, not for MDA. However, at this time, with gene addition and gene correction strategies, stem cell strategies, and many kinds of drug trials in the wings, the curtain is about to rise on projects of unprecedented importance in muscular dystrophy research — but also requiring unprecedented expenditures.

For this reason, MDA can no longer "go it alone." The government must do its part. But — in addition to continuing our own worldwide research program — we're committed to offering our knowledgeable and watchful support in the acceleration of muscular dystrophy research promised by the MD-CARE Act.

With every best wish…

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