Stem Cell Treatment Shows Promise Against Rare, Deadly Skin Disease


In a study published today in the New England Journal of Medicine, researchers describe a new procedure which offers some hope against Epidermolysis Bullosa (EB).

EB is a skin disorder affecting 1 out of 50,000 Americans. People with EB carry a genetic mutation that affects their ability to produce collagen – the protein which binds together our many layers of skin. In its most sever form, EB causes the skin to tear and blister at the slightest pressure. Life is one of chronic pain and scarring, and those that survive past childhood will likely develop an aggressive form of skin cancer as adults. (Find a dermatologist.)

This week’s study brightens those grim prospects. Researchers at the University of Minnesota Amplatz Children’s Hospital performed bone marrow transplants on a group of children with EB. The bone marrow contained a special type of stem cell which the researchers found could travel to the skin and spur on collagen production. Many children improved notably, and the results in some cases were dramatic – one child was able to play on a slide and jump on a trampoline, which before treatment was “unthinkable.”

While several children died of complications related to treatment, the authors argue that “you have to understand how horrible this disease actually is… It’s a serious treatment for a serious disease.”  Dr. Jakub Tolar, a co-author of the paper, wrote, “Patients who otherwise would have died from their disease can often now be cured.”

And interestingly, this is not just a step forward for patients with EB. While stem cells are an exciting field of research, there are few accepted therapies based around them. According to lead author Dr. John Wagner, stem cells might be used some day to treat other skin conditions or burns: “This tells us more about the potential power of stem cells.”

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