Pilot Trials Now was an initiative launched by Charleys Fund to get clinical trials underway for Duchenne patients using drugs that have already been FDA approved for other indications. Two clinical trials have been completed with support from several of our fellow nonprofit foundations including the Nash Avery Foundation, Action Duchenne, Cure Duchenne, Hope for Javier and Zubin’s Wish. These clinical trials were the outcome of years of screening, discovery and animal studies financed by Charley’s Fund to identify FDA-approved drugs (alone and in combination) that can help boys with Duchenne.
Sildenafil, a drug that showed promise as a cardiac treatment for Duchenne, was tested in young adults ages 15+ at Johns Hopkins/Kennedy Krieger Institute. Unfortunately, the drug did not improve the heart function in the clinical trial population. However, the pharmaceutical company Eli Lilly is building on what was learned during this research to test a similar drug in younger boys with Duchenne. Click here to read the published results of the study we financed.
Increlex, a drug that may stabilize muscle membranes and offset the egregious side effects of steroids, was studied at Cincinnati Children’s Hospital Medical Center in 30 boys with Duchenne.
There was no difference between the treatment and control groups over 6 months in the six minute walk test and most of the secondary motor outcomes. IGF-I treated subjects grew twice as well as the control subjects. Treated subjects also gained more weight than the controls, but BMI (Body Mass Index) gain was the same in both groups. Based on this result, the gain in weight in the IGF-I treated subjects is likely related to increased lean, rather than fat mass. Furthermore, IGF-I treatment was associated with a lowering of signs of Insulin resistance. Lowered insulin resistance is desirable, as insulin resistance is related to development of type 2 diabetes.