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U researchers use stem cells to repair the skin of children with a life-threatening disease

Research by Jakub Tolar, M.D., Ph.D., and John E. Wagner, M.D., moved quickly from mouse studies in the laboratory to showing success in patients. (Photo: Emily Jensen)

Physician-scientists at the University of Minnesota have for the first time demonstrated that a lethal skin disease can be successfully treated with stem cell therapy.

Medical School researchers John E. Wagner, M.D., and Jakub Tolar, M.D., Ph.D.—in collaboration with researchers in Oregon, the United Kingdom, and Japan—have used stem cells from bone marrow to repair the skin of children with a fatal skin disease called recessive dystrophic epidermolysis bullosa (RDEB).

It’s the first time researchers have shown that bone marrow-derived stem cells can repair the skin and upper gastrointestinal tract and alter the natural course of the disease. Until now, bone marrow has only been used to replace diseased or damaged marrow.

“To understand this achievement, you have to understand how horrible this disease actually is,” says Wagner. “From the moment of birth, these children develop blisters from the slightest trauma… They live lives of chronic pain, preventing any chance for a normal life. My hope is to do something that might change the natural history of this disease and enhance the quality of life of these kids.”

Since the study began at the University in 2007, 10 children with the most aggressive forms of EB have received transplants at University of Minnesota Amplatz Children’s Hospital. While all of the children have responded to the transplants, the magnitude of each response has varied.

The research results appeared in the August 12 issue of the New England Journal of Medicine.

“Bone marrow transplantation is one of the riskiest procedures in medicine, yet it is also one of the most successful,” Tolar says. “Patients who otherwise would have died from their disease can often now be cured.”

This research is supported in part by grants from the National Institutes of Health, the University of Minnesota Academic Health Center, Epidermolysis Bullosa (Liao Family) Research Fund, Sarah Rose Mooreland EB Fund, Children’s Cancer Research Fund, and agencies in Japan.

Read the news release, watch a video on the research, and meet some of the children who participated in the clinical trial at

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