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Pilot Trials Now

Pilot Trials Now is funding clinical trials of existing drugs for other diseases that might be of benefit to boys living with Duchenne. PTN is an international partnership between leading Duchenne non profits.

We have already begun preparing for the first clinical trial supported by Pilot Trials Now. PTN will give boys with DMD a fighting chance until a cure is found.

Pilot Trials Now partners with carefully selected physicians and fully supports them as they conduct clinical trials of FDA-approved drugs that show promise for DMD.

For our first trial, Prinicpal Investigator Kathryn Wagner, MD, PhD will lead a team of five experienced physician-researchers at Kennedy Krieger Institute of Johns Hopkins University. The team will test a drug called sildenafil (commonly known as Viagra). Originally developed by Pfizer as a heart medication, sildenafil has been shown to delay and even prevent heart failure in the DMD animal model.

In a study supported by Charley's Fund and the Nash Avery Foundation, sildenafil significantly improved the cardiac and vascular function of treated mice, and permitted increased blood flow to skeletal muscles.  The study was conducted by Stan Froehner, PhD at the Unviersity of Washington.  Heart failure is a leading cause of morbidity in DMD patients. Thirty boys ages 16+ will participate in the year-long trial, which is now recruiting.

Pilot Trials Now was established by Charley's Fund and the Nash Avery Foundation in the USA. To learn more, contact Pilot Trials Now at (413) 298-4300,or send an email to Tracy Seckler at tracy@charleysfund.org. 

See also:   www.charleysfund.org and www.nashaveryfoundation.org

For further information in the UK contact Nick Catlin at Action Duchenne nick@actionduchenne.org phone: +44 (0)208 556 9955