Using stem cells from umbilical cord blood and bone marrow, researchers apparently have cured a normally 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 Dr. John Wagner of the University of Minnesota Medical School, who performed the treatment. “Maybe we can take one more disorder off the incurable list.”
The team subsequently has treated Nate's 5-year-old brother Jacob and is preparing to treat 9-month-old Sarah Rose Mooreland of Folsom, Calif., and have high hopes for them as well.
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Nate suffers from recessive epidermolysis bullosa, a genetic disease that affects one in every 100,000 children. Such children lack a critical protein called collagen type VII that anchors the skin and lining of the gastrointestinal system to the body.
Their skin is extraordinarily fragile. Tearing and blistering occur with minimal friction, leading to painful wounds and scarring.
Solid food produces erosion of the esophagus. Death usually results from malnutrition, infections or aggressive skin cancer.
The only treatment previously has been to keep the children wrapped in bandages to protect the skin as much as possible.
The idea of using circulating stem cells to treat the condition was developed by Dr. Angela Christiano of Columbia University Medical Center. This is the first time that cells from bone marrow and cord blood have been used to treat a condition that does not involve blood.
After perfecting the approach in mice, the team treated Nate seven months ago. Nate went first because one of his brothers was a good genetic match. Jacob received cells from a non-familial donor.
Lab tests show that Nate's body is now making collagen type VII, Wagner said at a news conference Tuesday.
More important, his face is plumped up and he has fewer blisters. New skin now covers his right leg, which once had only a translucent purple-and-red coating over his internal tissues and veins. And he is eating Oreos and chicken noodle soup and begging for more.
“I have watched Nate improve every day,” said his mother, Theresa Liao. “It has been slow, but I believe we are on the road to him getting better.”
The results will be published in a future issue of The New England Journal of Medicine.