Expert Says Muscular Dystrophy Research Today ‘Is a Frontier Unlike Any Other’

Contemporary Clinic® interviewed Donald Wood, PhD, the recently elected president and CEO of the Muscular Dystrophy Association, on how the COVID-19 pandemic has impacted the muscular dystrophy field.

Alana Hippensteele: What are your hopes and plans for the future in terms of research and care for patients with muscular dystrophy?

Donald Wood: Well, let me tell you the same thing I tell my kids, my family: I'm here. [I’m here] to continue to move forward on treatments and advances.

We have seen so much growth and progress in the last few years. We're in the clinical trials now for gene therapy. When we first discovered the gene, I remember getting asked by reporters at the New York Times and all the rest of it, “So when do we get a genetics cure?” And I would say, “Well it’ll probably take 25 years,” because we didn't know enough.

But guess what folks, we are now at the point of being able to take the genetic makeup of a single individual, identify the specific genetic defect, and start to develop treatments that would repair that defect. We have people with spinal muscular atrophy—in the history of the world with spinal muscular atrophy—no patient ever walked who had the childhood form of it. Now, we got kids that are walking.

I have a friend with spinal muscular atrophy, and he's in his 30s, and I was talking to him the other day, and he's in one of the clinical trials. It brought tears to my eyes. He says, “I'm feeling better, Don. I'm feeling stronger. I'm for the first time feeling better.”

Folks, you have no idea—this is a frontier unlike any other. To be able to talk to a patient with a genetic disease in gene therapy treatment who says, “I am feeling better,” and he's an adult? Oh my gosh, well that's where we are. We're in a miracle place; we're in a magic place. We're in a place that I always wanted to get to.

What am I looking at 2021? I'm looking at medical history; I'm looking at scientific history. We are making history. I expect to continue that progress as long as I'm allowed to lead this wonderful organization.