A Scout Is Brave: Newly approved treatment helps Cub Scout thrive in face of neuromuscular disease

When Hiram Secrist was 2 years old, his parents learned that he had Duchenne muscular dystrophy, a rare genetic disease that causes progressive muscle damage and weakness.

“If you Google it, it’s not good,” says Hiram’s mother, Kristen. “One of the first things I learned is to stay off Google.”

Indeed, a person with Duchenne muscular dystrophy normally experiences increasingly noticeable muscle weakness between the ages of 3 and 5. Most will be in a wheelchair by the time they turn 13.

And death from respiratory or cardiac failure typically occurs when patients are in their mid-20s or early 30s.

It’s a tough break, to say the least.

Hiram’s parents made it their goal to focus on the day-to-day and worry about the distant future later. Hiram grew to enjoy the things that most kids enjoy: videogames, dinosaurs and playing with Lego bricks.

As he prepared to enter kindergarten, his parents learned to help him save his energy for the playground. If he avoided stairs and used a wheelchair as needed, he could run around the playground like the rest of his friends.

Then something else came into the Secrists’ lives: Cub Scouting.

“We thought it would give him something extracurricular to do, but it’s not too physical,” Kristen says. “It wouldn’t exacerbate his muscles. It’s low impact, but he gets to be with his friends and do fun activities.”

A life-changing phone call

When most people exercise, their muscles undergo microtraumas — tiny injuries that heal quickly and result in muscles that are stronger than they were before. Children with Duchenne muscular dystrophy suffer those same microtraumas, only their muscles never fully heal.

So instead of focusing on growing stronger and building more muscle, they focus on preserving the muscles they already have.

When he was 5½, Hiram began taking prescription steroids that, over time, could help preserve his muscles.

As a member of Pack 1345 in Burke, Virginia, with his mom or dad by his side, he was able to participate in almost all Lion Cub Scout activities, using a wheelchair when needed.

“My favorite thing about Scouts is playing with my friends,” Hiram says.

Then came a phone call from their doctor that changed their lives. Hiram, it turns out, was eligible for a newly approved gene therapy administered at Children’s National Hospital in Washington, D.C.

The treatment, called ELEVIDYS, is a recombinant gene therapy. During one 90-minute infusion, it delivers a gene that leads the body to produce a protein that helps muscles heal.

At the time, the treatment was only available for kids ages 4 or 5. Hiram received the treatment one day before he turned 6, becoming the first child in the world to receive the treatment after it was approved by the FDA.

Growing stronger

Hiram is now a 7-year-old Wolf Cub Scout.

The difference in his life post-ELEVIDYS treatment is remarkable. The change started just a week or two after the treatment and has continued ever since.

“His endurance is off the charts,” Kristen says. “He’s running. He runs to the bus stop every morning. He is able to jump, which is something he’d not been able to do before.”

There’s a Tiger Adventure called Tiger Tag that requires the Cub Scouts to play an active game with their den.

Before his treatment, Kristen probably would have advised her son to skip that activity. After gene therapy?

“You couldn’t tell which kid had muscular dystrophy and which ones didn’t,” Kristen says. “He’s doing everything everyone else is doing. He’s keeping up with everyone.”

Kristen is quick to point out that ELEVIDYS is not a cure. It’s a treatment designed to help kids with Duchenne muscular dystrophy live healthier, more active lives.

“It’s a huge step in the right direction,” she says. “Hopefully it will prolong his life and prolong the years that he’ll have to be ambulatory.”

For now, they’re just taking it one day at a time.

Photos courtesy of the Secrist family