Using stem cells from umbilical cord blood and bone marrow, researchers apparently have cured a fatal genetic disease in a 2-year-old Minneapolis boy, a feat that could open the door for a variety of stem cell treatments.
For the first time in his life, Nate Liao is wearing normal clothes, eating food that has not been pureed and playing with his siblings.
"Nate's quality of life is forever changed," said John Wagner of the University of Minnesota Medical School, who performed the treatment. "Maybe we can take one more disorder off the incurable list."
Nate suffers from recessive epidermolysis bullosa, a genetic disease that affects one in every 100,000 children. Such children lack a critical protein that anchors the skin and lining of the gastrointestinal system to the body.
Their skin is fragile. Tearing and blistering occur with minimal friction. Solid food erodes the esophagus. Death usually results from malnutrition, infections or skin cancer.
The only treatment previously has been to keep the children wrapped in bandages.
The idea of using circulating stem cells to treat the disorder was developed by Angela M. Christiano of Columbia University Medical Center. This is the first time cells from bone marrow and cord blood have been used to treat a condition that does not involve blood.
Nate was the first to receive the treatment.
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