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Parent Project Muscular Dystrophy: A Wake-Up Call
Timothy P. Cripe, MD. PhD
Associate Professor of Pediatrics
Division of Hematology/Oncology
Director, Translational Research Trials Office
Cincinnati Children’s Hospital Medical Center

“Your child has cancer” is devastating news and until today I thought it was the worst news that a parent could receive. I now believe that “your child has Duchenne Muscular Dystrophy (DMD)” needs to sit at the top of the list. I am a pediatric oncologist and cancer researcher and unfortunately have had the opportunity to witness the ravages of a cancer diagnosis to both the child and the family. There is no question that the diagnosis of cancer is bad news, but I am proud to say that there has been significant progress in the field. We are at least running a reasonably effective offensive attack against many pediatric malignancies. Unfortunately, the same cannot be said for males affected by Duchenne Muscular Dystrophy.

Because my wife is a pediatric cardiologist, our family recently attended the closing banquet of the Parent Project Muscular Dystrophy annual meeting. At the meeting there were ~250 families affected by muscular dystrophy gathered together to promote research and education in DMD. At our table was an adorable, blond six-year-old boy named Charlie. He had that generic “healthy boy-look,” with no outward difference apparent from my two sons, who were seated across the table. However, there was an important difference. Charlie is a ticking time bomb as he was recently diagnosed with DMD. If there are no significant research advances made soon, Charlie will face certain death sometime within the next 10-15 years. And it won’t be pretty. It will be a slow, painful, downhill spiral. Charlie will eventually lose the ability to walk followed by the ability to breathe. His heart will slowly lose its ability to function. At this moment in time, there is little that anyone can do about it. Unfortunately, medical science has not orchestrated any effective treatment strategies against the disease that is slowly destroying the muscles in his body.

Thirty years ago, that’s the way it used to be with many types of childhood cancer: almost guaranteed death. We can now cure nearly 70% of children with cancer. Forces in the U.S. were mobilized with a 1972 Presidential declaration of war on cancer, and legions of scientists and doctors worldwide have since spent countless years and dollars investigating the biology and treatment of cancer. Childhood cancer afflicts 1 in 300 children < 20 years old in the United States (data from the National Cancer Institute SEER Program). That number includes many readily treatable forms of cancer. Because of hundreds of clinical trials conducted by multiple cooperative groups, only about 1 in 1,000 children die from cancer. As a result of these efforts, hearing that your child has cancer is no longer a death sentence.

The incidence of DMD is 1 in ~3,000 male births. However, in contrast to cancer, DMD currently is uniformly fatal. As a result there is only a 3-fold difference in the number of childhood deaths due to cancer vs. DMD, but DMD research is relatively scarce. A search of the NIH website for open clinical trials (www.nih.gov, searched 7/18/03) revealed 2,368 for cancer, including 936 that were open to children. There were only 3 for DMD, a 300-fold difference. The paucity of research may be the reason that there is no cure for this devastating neuromuscular disease. Without research, there is no hope. Shouldn’t all people at least been given the chance to hope?

Why the disparity in resource allocation? Why has no war been declared on muscular dystrophy? Whatever the reason, it’s time to change. Every person, even a blonde little boy, should at least be able to face tomorrow with hope. Having the opportunity to meet Charlie and his family was a wake-up call. Thank you, Parent Project Muscular Dystrophy.

To learn more about Duchenne muscular dystropy, please visit the Parent Project Muscular Dystropy web site

This article originally appeared on the Parent Project Muscular Dystrophy web site, www.parentprojectmd.org. Reprinted with permission.  

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