U of M Sets Course For Cure of Fatal Childhood Skin Disease
Physicians at the University of Minnesota and University of Minnesota Children’s Hospital, Fairview have set the path to a cure for a young boy’s fatal genetic skin disease, recessive dystrophic epidermolysis bullosa (RDEB), by using a cord blood and bone marrow transplant. Nate Liao, a 25-month-old from Clarksburg, New Jersey, underwent the experimental therapy in October 2007.
“We have established a new standard of care for these EB patients, beginning with Nate,” said John Wagner, M.D., the lead University of Minnesota Medical School physician who developed the clinical trial. “Nate’s quality of life is forever changed.”
Because they lack collagen type VII, children with RDEB have skin that is exquisitely delicate. The skin and lining of their GI tract is fragile; tearing and blistering occur with minimal friction. Coughing and vomiting often result in tears in the lining of the esophagus and stomach. Those affected must have their entire body continuously wrapped in bandages. Those who do not succumb from malnutrition and infection in childhood will acquire a uniformly fatal, aggressive cancer of the skin in young adulthood.
