Mel Kelly of Torrington was the subject of this Mommy Minute in February.
The Torrington mom has two sons with Duchenne muscular dystrophy (DMD), a life-threatening, genetic disease that mainly affects boys. Both Liam (15) and Jacob (19) are in wheelchairs, robbed of their ability to walk, sit-up and bathe themselves.
When we first met Kelly, she was eagerly awaiting a date to be set to testify before a Food and Drug Administration Advisory Committee. She - and other parents - were ready to implore the FDA for accelerated approval of an experimental drug called eteplirsen, currently in clinical trials. They believe this drug will save lives.
Eteplirsen treats a subset of those with DMD. It is designed to produce a protein that’s missing in patients, preserving muscle function. Parents involved in the trials say the drug has helped their kids' ability to move and has slowed progression of debilitating symptoms.
On Monday, Kelly got her day in Maryland during an eleven hour meeting.
According to a piece on Fox News: "More than 1,000 people packed a hotel conference room to push a deeply skeptical FDA..."
Members of the FDA spoke first, focusing on a preliminary briefing document which did not go in favor of the families, as it questioned the small size of the clinical trials.
Advocates - including Kelly - testified in the afternoon. "There were two boys who said, 'It's up to you whether I die or not.' It was really, really intense," says Kelly. "The testimonies were very powerful and several members changed their vote by the end of it."
7 members voted "no" for accelerated approval, 3 voted "yes" and 3 abstained from voting.
At first, the families were devastated. "As a community we were heartbroken. The atmosphere in that room was tough. You had all these children with long faces and mothers hugging each other and crying. We were scared. We were scared that this drug was not going to get accelerated approval," explains Kelly.
But, after more investigation, they are now encouraged. Kelly has learned that the "abstained votes" count as "yes votes". Also, according to Kelly, a key player from the FDA is now supporting accelerated approval of the drug.
There has also been support from senators, such as Marco Rubio, and a wave of recent media attention.
No matter what happens, Kelly finds great value in the awareness that has been raised.
"This was not in vain," says Kelly. "We believe this drug will get approved...we believe it's going to go through. It may not go through this time but I do feel that our testimonies will be the reason this drug gets approved."
The FDA will issue a decision on May 26th. "Can't come soon enough," says Kelly.