Stem cells appear to cure boy's disease
Stem cells appear to cure boy's disease
By Thomas H. Maugh II
Los Angeles Times
Posted June 07, 2008
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 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.
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 M. 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.
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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