This Friday, February 7th, at 10am, Congressional Staff will be briefed regarding a big decision the FDA is about to make that could greatly affect the futures of kids with Duchenne Muscular Dystrophy (DMD.)
According to Charley’s Fund, a non-profit started by Benjamin and Tracy Seckler who are parents of a child with DMD, based on steps being taken this week, the FDA will decide whether to grant accelerated approval for a treatment drug that is the best bet ever for kids with DMD, and they are urging their followers to write their congressman and senators showing support so that the FDA will not hold the treatment back from kids who have no time to wait.
In case you’re unfamiliar with the sobering facts about DMD, here are just a few that can help you understand what’s at stake for these kids — and why time is of the essence. DMD is currently 100% fatal. Most kids with DMD are in wheelchairs by age 10 or 12 and die in their late teens or early 20s. It’s the most common fatal genetic disorder worldwide. Approximately 15-20 thousand children in the US alone are afflicted.
However, this amazing statement was just posted on the Charley’s Fund website, “We have a drug that is safe and effective in a small clinical trial that has lasted more than two years. Some of the boys taking the medicine are even IMPROVING, a word that is never uttered in relation to Duchenne. Yet the drug is only available to the 12 kids in the trial, and it could be YEARS before other kids with Duchenne are allowed access.”
This is a big moment for the Secklers. As Tracy Seckler explains, “This drug cannot help Charley. But the company developing it has plans to develop 8 new drugs for Duchenne, and Charley can benefit from one of the next ones. The new drugs are all based on the same chemical backbone as this one [Eteplirsen, which alone can help combat the effects of Duchenne in 13% of kids while the 8 new drugs developed thereafter collectively would be able to treat 85% of kids with Duchenne.] So if it takes years to approve Eteplirsen, it will take a decade or more to do the rest. If Eterplirsen is granted accelerated approval, the others can get moving now.”
If a drug is to help Charley, and all other boys like him, there is no time to spare. And that has been the driving force of Charley’s Fund, which the Secklers started in 2004 when Charley was first diagnosed at the age of 3. Run with their tireless effort with the goal of saving their child’s life, the Fund has already directed 25 million dollars into medical research for DMD. What they’ve done on behalf of their son — and all sons who have this destructive disease — is unprecedented.
Two years ago, I was in the audience of a TEDx talk given by Benjamin Seckler and was struck by the focus and relentless drive he had to race against time in pursuit of a cure for his son. What I walked out feeling was that prior to learning of his son’s illness, he was just a parent, just someone looking to the bright future of his three kids before everything changed with one phone call from a doctor. I invited him to sit and tell me more about what it was like for him, as a parent — that horrific life altering moment when he learned of his son’s diagnosis, and the journey he has found himself on ever since.
The following are his words:
“I remember the day that I found out. Tracy called me. I was at work in Hudson [New York.] Charley had gotten the official diagnosis for Duchenne. Of course, I left work immediately to go home, and I remember crying hysterically all the way home. And maybe this was kind of a weird thing to do — I’m sort of a weird person though — but I remember as I was crying uncontrollably, I looked at myself in the rear view mirror. I wanted to look at what a man who just found out his son had a fatal illness looked like. Like as if I were sitting in a restaurant and motioned to someone and said “See that guy over there? He just found out his son has a fatal illness.” It was as if I had just become someone else. Everything had just changed. I wanted to see what this other person I had become looked like.
That was a bad day.
Right after they receive such news is when most parents are paralyzed by the emotions that go along with it. Many marriages don’t survive. And since this disease affects mainly boys, sons, I think particularly of their fathers. I think it’s safe to say that dads lean toward their sons in a certain way. In the same way that mothers think of their daughters, fathers think of their sons as a way of remaining immortal. Their sons are little versions of themselves.
In boys, the diagnosis for DMD generally comes between the ages of three and six. Charley was three. Sam is just 16 months older than Charley, and my wife, Tracy, was expecting Maizy when we found out about Charley’s diagnosis.
Both Tracy and I are the youngest in our families: Tracy is the youngest of four, and I’m the youngest of five. And my siblings are quite a bit older than me. So I always had that notion of looking toward the future, consciously planning out my life: Grow up, do all the right things that would get me into a good college, plan well, go to medical school, meet my wife, start a family.
Everything had always played itself out as I had expected, more or less. I did lose my father when I was 17, but the news about Charley was different. I suddenly knew, from that moment, my whole life was going to revolve around trying to save my child’s life. I suddenly had a completely new horizon.
I really think the worst feeling is now, having things in my future I don’t want to think about. The painful thought of having a funeral and burying my child. The thought that this is really going to happen. One of the most comforting things for a parent is the thought that your child will be here after you’re gone. That is gone, too. It’s like a double whammy.
One of the ways I explain what the pain feels like is this: It’s like your child is in the middle of the road — tied to the middle of the road — and there’s a car coming. The car is coming from a long way away. And although you’re racing to get to the middle of the road, you can never get there. And you see that car in the distance getting closer and closer, and still you can’t do anything to stop this accident that’s going to happen.
So the work we do at Charley’s Fund refocuses those obsessive thoughts. Instead of thinking about Doom’s Day, I’m constantly thinking of the opposite. Tracy and I did manage to overcome the crucial crossroads of feeling paralyzed after the diagnosis. We formed Charley’s Fund just four months later. Some of it has to do with our personalities, and some of it has to do with the silver lining of my being a young physician who understands the language of genetic medicine.
I was able, in those early stages, to mine though the research I found online. I would work all day as a radiologist, then I would be up all night studying the research. When the stakes are so high as in our situation, where you’re fighting to save your child’s life, you look at every angle; it forces you to examine the Rubik’s cube in so many different ways. I think that’s why we’ve been so successful. We had to be innovative. We had to think of possibility and hope. We have to keep our thoughts there.
I remember in those early days calling the clinicians and scientists in the labs directly until I formulated the early strategy of what we needed to do. The breakthrough was when we decided to hire a Ph.D-level scientist to work full time for us. All of a sudden we went from a small parent foundation to the next level. And eventually, over time, we even outgrew the brain power we had with that Ph.D. We decided to create an all-star team of drug development experts. Now we have formed our own virtual biotech company. By taking matters into our own hands, we are literally changing the way drug development happens in this country.
Because I was the youngest in my family, my siblings would always joke that everything was taken care of for me. I was ‘the golden child.’ The nickname my siblings gave me was ‘Fog,’ because I was oblivious to so much. It’s interesting that after growing up with a nickname like ‘Fog,’ I am now involved in something that requires so much clarity. And I have that clarity.”