“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 their children with DMD. The only medication currently available for this fatal pediatric disease is a daily dose of steroids, which experts agree may (at best) keep diagnosed children walking for an additional 1-2 years. Steroids as a treatment are far from ideal, as they are often accompanied by negative side effects including rapid weight gain and loss of bone density. More disturbing is that DMD’s bottom line has not changed since the disease was first described in the medical literature 150 years ago. The disease remains 100% fatal, with most boys dying in their teens or 20s.
Several medicines that have been FDA-approved for use in other illnesses show real promise in the animal model for DMD. The safety and toxicity profiles of these drugs are well known. It is widely agreed that these drugs and supplements might help children with DMD. Yet human clinical trials for these treatments are still not underway.
The rate limiting step here is not scientific knowledge. Rather, the stumbling blocks are administrative requirements and lack of time, experience, and resources on the part of those who can initiate a trial.A great deal of administrative work is required just to set up a trial, including drafting a protocol and budget, securing approval from the hospital regulatory body, and sourcing funds.Once a trial is approved and funding secured, patients must be recruited and then followed diligently to ensure compliance.Forms must be collected and filed. Data must be gathered, analyzed and reported.Not surprisingly, most physicians simply lack the time and manpower to set up and monitor a trial in addition to their clinical responsibilities.
Pilot Trials Now is an innovative program conceived of and supported by Charley’s Fund and the Nash Avery Foundation. The goal of Pilot Trials Now is to implement small clinical trials over the next 2 years for FDA-approved drugs and supplements that show promise as treatments for DMD.
The objective is to identify trends and determine if full-scale, robustly populated multi-site clinical trials are warranted. Pilot Trials Now partners with carefully selected physicians and fully supports them as lead investigators in clinical trials of FDA- approved drugs that show promise for DMD.
Now is a crucial time to identify drugs that can keep DMD kids “in the game.” For the first time ever in the history of this notorious disease, groundbreaking new therapies are being developed. While they wind their way through the regulatory process, we must keep children with DMD as healthy and strong as possible so they will still be able to benefit when the new drugs become available.
Please consider supporting our Pilot Trials Now initiative.