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BMT therapy sets course for curing fatal skin disease

An experimental treatment performed at University of Minnesota Children’s Hospital, Fairview, has brightened the days of New Jersey toddler Nate Liao. The 2-year-old was born with a rare genetic disease that caused his skin and the lining of his digestive tract to slough off—making even a gentle touch or swallow of food painful.

Children with the disease, recessive dystrophic epidermolysis bullosa (RDEB), often need to be wrapped in bandages for protection, and many require feeding tubes to ensure adequate nutrition. In those who reach young adulthood, the disease usually leads to an aggressive, fatal skin cancer.

But a novel therapy used by a team of physicians and researchers at University of Minnesota’s Children’s Hospital, Fairview, appears to have stymied the disease. In October 2007, the team performed an experimental umbilical cord blood and bone marrow transplant (BMT) to combat RDEB. They hoped the procedure would introduce new cells into Nate’s body to produce a missing protein, called collagen type VII, that holds the skin in place.

Since the transplant, Nate has shown signs of dramatic improvement. Multiple biopsies following the procedure showed that his skin and intestinal lining are starting to attach. Although he is still partially wrapped in bandages to prevent skin damage, he doesn’t suffer from wounds as easily or as frequently as he did before.

This is the first time physicians have treated RDEB using marrow-derived stem cells to correct the underlying defect. “We have established a new standard of care for these EB patients, beginning with Nate,” says John Wagner, M.D., director of pediatric hematology-oncology and BMT at the University and the physician-scientist who led development of the treatment. “Nate’s quality of life is forever changed.”

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