“There is not one FDA approved drug I can offer”
Those are the words Dr. Brian Tseng must utter when families come to his clinic at Massachusetts General hospital seeking a treatment for thier children with Duchenne Muscular Dystrophy (DMD). This disease is the number one genetic killer of children worldwide. For the first time ever in the history of this notorious disease, groundbreaking new therapies are being developed.
Now is a crucial time to identify existing drugs that can keep DMD kids “in the game” until thse new treatments become available. Several medecines that have been FDA-approved for usin other illnesses have demonstrated exciting resuls in DMD animal testing. Pilot Trials Now is an innovative program that supports small clinical trials for FDA-approved drugs that show promise as treatments for DMD.
Trial #1
Who: Dr. Kathryn Wagner and Dr. Daniel Judge with 30 DMD boys ages 16+
What: sildenafil (Revatio)
When: July 2010 – August 2012
Where: Johns Hopkins University/Kennedy Krieger Institute
Why: Animal studies show that sildenafil can delay and possibly even prevent heart failure in DMD.
Drug and placebo provided by Pfizer.
Trial # 2
Who: Dr. Meilan Rutter with 40 DMD boys ages 5+
What: IGF-1 (Increlex)
When: Oct 2010 – Nov 2011
Where: Cincinnati Children’s Hospital Medical Center
Why: IGF-1 offers potential as a therapeutic agent for DMD, as it may improve or preserve motor function and reduce the side effects of chronic steroid use.
Drug provided by Ipsen.