For many months, we at MDA have been using the word "progress" to talk about the incredible period we now find ourselves in, with more new drugs in the pipeline and more clinical trials underway than ever before. Last month, we experienced a potential game changer in our urgent race to find treatments and cures for patients and families affected by neuromuscular disorders.
After years of MDA research investment and learning, advocacy work, and follow-up development by biopharmaceutical companies, the Food and Drug Administration (FDA) announced on, April 21, 2014, that it will consider accelerated approval for eteplirsen. This is a drug specifically designed and developed for about 15 percent of boys fighting Duchenne muscular dystrophy (DMD) — those with a DMD-causing abnormality in a specific part of the dystrophin gene.
You can read a great story published recently in The Washington Post that captures the history and excitement of this development. I've annotated it to highlight MDA's role. You can also read about MDA's reaction to the news at MDA Thrilled FDA Will Consider Accelerated Approval for New Muscle Disease Drug Aimed at DMD.
This landmark development comes as a result of MDA’s willingness to take a risk by funding early-stage research on a novel strategy, known as "exon skipping," in the late 1990s. Our steadfast funding for exon skipping, which allows cells to "skip over" a piece of genetic instructions so that functional dystrophin protein can be made, allowed Australian molecular biologist Steve Wilton, one of the pioneers in this field, to pursue his ideas and concepts.
Wilton, who has been funded by MDA since the 1990s and has a current MDA research grant to further develop exon-skipping technology, has repeatedly said that, “MDA funded his groundbreaking work when no one else would touch it.”
If this next round of human clinical trials shows eteplirsen is safe and effective, it may be the first of many drugs in its class that have the potential to modify the course of Duchenne muscular dystrophy, preserving muscle strength and improving the quality and length of life for thousands of boys and young men facing this currently fatal diagnosis. And exon skipping as a general strategy could have implications for many genetic disorders beyond Duchenne dystrophy.
You, our community of supporters, are playing an important role in making this new research development and potential breakthrough drug possible. Your dedication to save and improve lives of the millions worldwide fighting muscle diseases like DMD has helped bring help and, especially today, hope to the families we serve.
I encourage you to share this news and information with your circles to thank them for their part in making this incredible development possible.
Together, we are progress.
Steven M. Derks
President & CEO
Muscular Dystrophy Association