WHERE DOES THE MONEY GO?

The Foundation to Eradicate Duchenne (FED) raises money to support the world's only clinical trials network for Duchenne Muscular Dystrophy (DMD). The clinical trials network known as CINRG works with scientists all over the world on therapies for DMD - a fatal disease which scientists now say is tantalizingly close to being cured.

For now, this clinical trials network prolongs the trajectory of the disease until a cure can be found. Funding this clinical trials network and the scientists with whom it works means that every dollar is critical in this race against time. The FED puts an overwhelming majority of the money it raises directly into research, which means that each dollar raised brings the boys suffering from DMD closer to the cure.

In pursuit of the cure, FED has raised millions of dollars for the best DMD scientists and doctors around the world. Their efforts have launched clinical trials with drugs such as prednisone, deflazacort and pentoxifillin - drugs upon which Duchenne boys now rely to slow the progression of the disease. These trials have been made possible by a number of small fundraisers and the opportunities for Congressional intervention that they have fostered. Consequently, the District of Columbia Appropriations Bill provided $400,000 to the clinical trials this year and the Energy and Water Appropriations Bill provided an additional $125,000.

In Ohio, Dr. Jerry Mendell is working with $1.2 million on new therapies and solutions for DMD as a result of the work of FED in the Labor and Health and Human Services Appropriations Bill. Similarly, this bill has provided $650,000 for Duchenne research at the Universitiy of California in Los Angeles and San Diego to explore high-throughput drug screening and $50,000 for a gene therapy scientist in Seattle, Washington named Jeff Chamberlain.

Dr. Chamberlain has discovered new approaches to gene therapy that have cured this same disease in mice and very recently, dogs. In recognition of his work, Congress appropriated an additional $125,000 from the Energy and Water Appropriations Bill and, along with FED, is endorsing Dr. Chamberlain's $2 million research grant with the Department of Defense this spring.

In Charlotte, NC, one of the newest members to the CINRG clinical trials network, Dr. Qi Lu is working to manipulate the components of genes. This scientific advancement operates in pursuit of a process that would mitigate the effects of DMD. This process, called "exon-skipping" would allow the flawed gene to "read through" and create dystrophin where previously it could not. If successful, Dr. Lu's technique could produce a genetic process that could change DMD into a much milder, and thus less fatal, form of muscular dystrophy. This approach is another that could be available in mere years, rather than decades.

Managing all of these efforts from his lab in Washington, DC, Dr. Eric Hoffman, Children's National Medical Center in Washington, DC, works closely with FED. As a member of the team 17 years ago that discovered the gene that causes DMD, Dr. Hoffman well understands the stakes involved in this deadly disease. Presently, his work involves identifying the pathways of DMD that could create multiple new targets for medical intervention that could dramatically slow the progression of this disease. In addition, he serves as the lead scientific advisor to these fundraising efforts in coordinating these and many other scientists throughout the world. As a result of the efforts of FED, Dr. Hoffman manages a $3.5 million fund provided by the Department of Defense to promote the most promising therapies available today.

FED is making all of this happen as a result of personal contributions and fundraising events. With FED's efforts, over $2 million has been raised to date. Scientific advancement is progressing as rapidly as the FED can fund it with new breakthroughs being discovered every day. Every dollar counts, as each goes to expanding the foundation's efforts, adding new clinical trials and basic research, and bringing thousands of boys closer to the cure.

Despite all the successes and major scientific breakthroughs, time is of the essence, and this research can only continue to progress if there are funds to support them. While the best scientists in the world agree that a cure for this disease is inevitable, the only unknown is whether it will arrive in time for today's sufferers of DMD.

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