Hoping for relief by participating in a dupilumab clinical trial

“Mom, I need you upstairs,” Jonah said.

“What is it?” I asked.

“I just need you,” he responded. “Come right now.”

Typically, exchanges like this one don’t happen nearly as much as they used to. As Jonah, my son with epidermolysis bullosa (EB), has gotten older, he’s become more independent (and less clumsy), and our emergency situations are a bit fewer and further between. But two weeks ago was one of those emergency moments.

Jonah, in getting out of bed, accidentally caught his foot on the leg of his metal bed frame. And just like that, he went from fine to really, really not fine. I got upstairs and saw what we were dealing with: Jonah’s fourth and fifth toes on his left foot were completely degloved, in this case meaning the top layer of skin was shorn. He was in excruciating pain, and there was little I could do.

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Just like that, his pain was to the point of him almost vomiting, and he couldn’t go to school. And it was two days before we were to leave for Chicago, where he’d be on his feet much more than normal.

That’s one of the worst things about EB — the way it can completely change the trajectory of your day (and even your week) and make typically fun things horrifically painful. And it all happens in a blink.

Clinical trials (and the Chicago Cubs)

We were heading to Chicago to participate in Jonah’s first clinical trial, which is testing the medication dupilumab. The treatment, also known by its brand name Dupixent, is a prescribed injection to control the itch that comes with certain skin conditions. It’s approved for eczema and a few other conditions by the U.S. Food and Drug Administration.

Folks at Northwestern University are now testing it on patients with inherited skin conditions. We have an EB friend in the trial who’s experienced incredible results — not just for her level of itch, but also for the overall health and strength of her skin. Like Jonah, she’d always been full-body bandaged, but now she’s able to spot-wrap. Her dressing change time has gone from three hours every day to one hour every other day.

It’s not a cure, for certain. But dupilumab has radically changed her quality of life (and her mom’s!). That may not happen for Jonah, but they have very similar cases of EB and are missing the same protein, so we’re hoping for relief. When EB can deglove a quarter of your foot in a literal second, it makes you want to do whatever you can — as fast as you can — to possibly make life just a little bit better.

We left for Chicago two days later and had one of the most chaotic airport experiences we’ve ever had. Jonah had refused to take his wheelchair. (Teenagers. Sigh.) But when we walked in, wall-to-wall lines of people were snaking back and forth across the front half of the airport. We couldn’t even see the luggage counters. We had to walk around for more than 20 minutes, back and forth, just trying to figure out where the lines went, which one we needed to be in, and where the back of it was. We arrived the recommended two hours early, but still ended up running to our gate.

All of this while Jonah winced, struggled, and teared up, walking on his bandaged but still very raw foot.

Chicago, despite Jonah’s pain, was great. It wasn’t easy for him, but we still enjoyed touring the city by bus and by boat. And to Jonah’s ultimate delight, we attended a Cubs game at Wrigley Field. (It’s quite possible that the promise of a Cubs game is what got him to agree to the study in the first place.)

From left, Jonah at Wrigley Field; with his mother, Patrice; and with his brother, Gideon, and father, all touring Chicago while in the area for Jonah’s dupilumab trial. (Photos by Patrice Williams)

I don’t know what the future holds for Jonah. We’re hoping for vast improvement in the next 16 weeks. If nothing else, we’re doing something about it, which is a feeling I’ve never had in these 15 years. I’m so thankful to have something we can do. Something that might bring some relief. Something that might make us worry a bit less and hope a bit more. I’m just so thankful for something.

Note: Epidermolysis Bullosa News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Epidermolysis Bullosa News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to epidermolysis bullosa.