HOUSTON – There’s a new type of therapy that can help kids with Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) affects one in 3,600 male births, and it can lead to immobility, and eventually death.
Curtis and Kristen Sommers from Spring said there were signs their son, Carter, had DMD at 18 months old when he wasn’t hitting milestones like pulling himself up.
The diagnosis was devastating, his mother, Kristen, said it was even hard to go home and be in Carter’s nursery, which was decorated with a sports theme.
“You just don’t imagine having, you know, having a child and that the diagnosis is they may be in a wheelchair by the time they’re 10 or 12, and they might not live past 20. You know, you never imagine that in your wildest dreams,” Kristen said. “So, it was hard to walk into that nursery because it was just like everything that we imagined felt crushed.”
However, they may have found a treatment that could change all of that.
This month, Carter became the first Houston child to receive a new gene therapy designed to deliver a protein to his muscles that should improve strength long term.
The treatment is called Elevidys.
Dr. Tim Lotze, a neurologist at Texas Children’s Hospital, said little boys who were part of the study with Elevidys are showing long-term promise.
“This is really a groundbreaking treatment that’s become available,” Dr. Lotze said. “It really looks to dramatically change the long-term outcome for these boys’ strength, and hopefully, truly helping to preserve it so that they don’t lose their ability to walk around age 10 to 12, which is more typically expected.”
The therapy has to be given between four and five years old, that’s the group it’s been studied for now.
Carter’s family said they’re already seeing improvement.
“They’re already seeing the benefits, that he is moving a lot faster around the home, climbing up on things that he might have been hesitant to climb up on, and so, you know, these are things that I’m looking for,” Dr. Lotze explained. “Is he able to run? Because that’s one thing that boys with Duchenne often are not able to do, and just seeing him taking those larger, longer strides down the hall, bending over to pick things up and being able to throw things. I think these are all telltale signs that this treatment is having a beneficial effect.”
So, this therapy may have intervened in Carter right on time.
“We hope that he never sees a wheelchair... We hope for a more active, longer active child,” Kristen said.
Unlike previous patients with Duchenne’s, these are finally realistic hopes for the future of patients.
“I mean, the kids that are taking the gene therapy, in the five or six years they’ve had it, they’re doing awesome. Some kids are playing sports. They’re not in wheelchairs at all. They’re so far doing good. And that’s what we’re looking for: to prolong his muscles and, you know, as we’ve been told, it’s not a cure. But if, you know, I don’t know, 10 years down the road, there’s no telling where we’re going to be in the future,” Curtis said.
For now, Carter may just be able to continue with maintenance steroids and won’t need another infusion.
The family was initially charged $3.2 million for this one-time infusion. However, the Sommers fought insurance and managed to get a lot of it covered. This means, they might’ve just opened the doors for other boys like Carter to receive the same type of treatment.