Summer’s Almost Here and Hopefully, So Are Some FDA Approved Drugs

By May 30, 2007October 28th, 2016Tracy's Blog

When I view my six-year-old child executing his morning and evening routine through the eyes of a visitor, it is truly an amazing sight. Charley downs 13 pills every morning and 11 pills every night. In the evening, he chases down the capsules with a mixture of seltzer and glutamine, a powdered muscle-enhancing supplement. When guests are over, Charley inevitably becomes the evening’s entertainment as he swallows three pills in one gulp, or makes all 13 pills disappear in 20 seconds, or vacuums up the muscle drink through 15 straws at a time.

We’re lucky that he is so good-natured about taking all these pills and powders. It would be very difficult to force the issue with a resistant child, especially knowing that only one of the 24 pills is actually proven to be effective by empirical evidence. The others are all herbal supplements and one heart medication that may or may not help slow the degeneration of muscle in DMD.

Despite that fact that several FDA-approved drugs have shown promise in mice with muscular dystrophy, there is still not one drug — other than steroids — that is being prescribed for kids with DMD to slow the muscle destruction. At Charley’s Fund, one of our main priorities is to identify FDA-approved drugs that have shown promise as a treatment for DMD and to follow up on every one of these leads. While scientists work their way toward a cure, these drugs could buy time for Charley and all the other boys who have no option right now but to suffer the significant side effects of chronic steroid use. The more muscle mass we can preserve, the better off all young boys with DMD will be when a cure or treatment is eventually developed.

In line with that goal, we recently signed on to support a one-year research project being conducted by Dr. Stanley Froehner at the University of Washington. Dr. Froehner is testing phosphodiesterase inhibitors as potential drugs to treat DMD. PDE inhibitors may reduce inflammation, improve blood flow in the muscle, upregulate utrophin and inhibit myostatin, a negative regulator of muscle mass. We know that PDE inhibitors are safe, because they are approved to treat other conditions (for example, sexual impotence!!).

We are also taking advantage of the World Wide Web in our search for pre-approved drugs that may help boys with DMD. We recently posted a challenge on Innocentive, a virtual community of scientists from all over the world. The challenge asked scientists to suggest a drug that may be helpful for DMD boys with a rationale explaining why. One particularly interesting submission suggested resveratrol, a supplement derived from grape skins that may work as an antioxidant, NFkB inhibitor, and antifibrotic. We are collaborating with a scientist to test resveratrol in mice with muscular dystrophy and, ultimately, would like to see a clinical trial in children with DMD.

Losartan, UDCA, sulfasalizine and HCT 1026 are some of the other FDA-approved medications that are “on deck