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Joseph’s story
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New treatment gives hope to children battling pre-cancerous skin disease
Children’s Cancer Research Fund-backed researchers at the University of Minnesota have published promising results in the August 12 issue of the New England Journal of Medicine, which shows the healing power of stem cells to repair skin. This breakthrough discovery demonstrates that a lethal skin disease, Epidermolysis Bullosa (EB), can be successfully treated with stem cell therapy.
Lead researchers John E. Wagner, M.D., and Jakub Tolar, M.D., Ph.D., are—for the first time—using stem cells from bone marrow to repair the skin of patients with EB.
“This discovery is more unique and more remarkable than it may first sound because until now, bone marrow has only been used to replace diseased or damaged marrow – which makes sense,” says Tolar, associate professor of pediatric transplantation. “But what we have found is that stem cells contained in bone marrow can travel to sites of injured skin, leading to increased production of collagen which is deficient in patients with EB.”
EB is a rare, genetic skin disease that causes skin to blister and scrape off with the slightest friction or trauma. It affects the skin and lining of the mouth and esophagus. Previously, there was no treatment and no chance for cure. In some countries, even euthanasia has been considered for newborns with the severest forms. If children with EB do not die of infection in their early life, many with the disease do not live beyond their 20s or 30s because they develop an aggressive form of skin cancer. While a few will live long term, the severest forms of EB are generally lethal.
Wagner and Tolar initiated the study in the fall of 2007. Since then, ten children with the most aggressive forms of EB have been transplanted at the University of Minnesota Amplatz Children’s Hospital. While all of the children have responded to the therapy, the magnitude of each response has varied.
“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 which eventually scar. 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 for these kids.”
The article, “Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa,” was printed in the August 12, 2010 issue of the New England Journal of Medicine. Their discovery was featured in many national news outlets including USA Today, Businessweek and CNN. To view the latest updates and to learn more about this discovery, visit our website, ChildrensCancer.org/EB.
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