Before the Labor, Health and Human Services Subcommittee of the Senate Appropriations Committee
Testimony of Dr. Leon Charash - Biography
02/27/01
Thank you Mr. Chairman, Senator Harkin and members of the Subcommittee for this opportunity to address you. I'm here today to call your attention to a matter of life and death for tens of thousands of children and adults affected by muscular dystrophy. Muscular dystrophy is the name given to a group of disorders caused by genetic defects and characterized by weakening and eventual wasting of voluntary muscles. The muscular dystrophies can weaken the muscles of the heart and those required for breathing. By profession, I'm a pediatric neurologist and I've treated many children with muscular dystrophy.
In 1950, a group of parents and other relatives of muscular dystrophy patients, concerned that virtually nothing was being done to combat these diseases, organized to form what is now the Muscular Dystrophy Association. For over 50 years, the Association has provided help through medical services and hope through research for youngsters and adults with any of the nine forms of muscular dystrophy. And it's thanks to hundreds of millions of dollars in research funded by MDA that tremendous developments in understanding the causes of these disorders have occurred. Today, virtually all of the genetic defects that cause the muscular dystrophies are known.
The obvious next step is the development of effective therapies. A number of strategies are being pursued to correct the gene defects in the muscular dystrophies. Some are in the early stage of development and have shown encouraging results. Others are in the clinical trial phase. One approach now in clinical trials involves the use of antibiotics. MDA-funded studies of how cells make proteins led to the discovery that some antibiotics can override a "stop" message in genes that make the critical muscle protein dystrophin. Also, therapies using resident stem cells have shown encouraging results in early studies and, based on those results, will eventually be applied in clinical trials. Another strategy is gene therapy designed to replace missing or defective muscle proteins.
Scientists are optimistic about the therapeutic potential of these new techniques. However, the price tag is high. MDA funded and initiated the first gene therapy clinical trial in a muscle disease in September 1999. The project was a phase-one trial in which only six individuals were slated to receive gene therapy. The budget for this initial trial alone was nearly $5 million. It's clear that continuation and expansion of these studies depends upon an increased financial commitment. For the first time in the history of these terrible diseases, we are not limited by science.
While MDA will not only continue to press ahead but will increase its efforts in search of treatments and cures, we believe strongly that a major investment through the National Institutes of Health will be essential to advance to the next level in muscular dystrophy research. To this end, we propose that the projected $19.9 million NIH annual commitment for muscular dystrophy research be increased by $100 million. We also propose that an NIH study group be established for neuromuscular disease research.
An analysis of NIH expenditures on diseases that affect a number of people similar to that affected by muscular dystrophy shows a great disparity in spending. In fact, spending on some of these disorders, and even on some that affect far fewer people, is many times the amount allocated for muscular dystrophy research. We seek this Subcommittee's support in our effort to meet the challenge of taking muscular dystrophy research to the next level - the development of effective treatments for 250,000 American children and adults.
Thank you.
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Read the other testimonies, [Jerry Lewis] [Christopher Rosa, Ph.D.] |
