Doctors Use Stem Cell Therapy For Genetic Skin Disease
ScienceDaily (Nov. 5, 2007) — University of Minnesota Children's
Hospital, Fairview physicians have performed the first bone marrow
and cord blood transplant to treat recessive dystrophic epidermolysis
bullosa (RDEB).
Children with RDEB lack a protein that anchors skin to the body,
resulting in fragile skin that sloughs off with little movement or
friction. They suffer painful wounds and must be bandaged at all
times to protect their skin from further damage and infection. The 18-
month-old boy who was transplanted has the most severe form of RDEB,
which also causes skin to slough off on the inside of the body,
affecting the mouth, esophagus, and gastrointestinal tract. EB is
genetic and severe forms are always fatal. Those who live to be young
adults get an aggressive form of skin cancer called squamous cell
carcinoma.
With the help of an EB mouse model and in collaboration with
investigators at Columbia University, University of Minnesota
researchers were able to correct the disease in mice using bone
marrow. They tested various types of adult stem cells to determine
which would give rise to the development of type VII collagen -- the
protein people with RDEB lack. One type of immature cells from bone
marrow proved to be the best at producing anchoring fibrils that bind
the skin to the body.
This is the first time physicians have approached EB from a systemic
perspective, using transplant as a means to rid the body of the
defective blood system and replace it with a healthy blood system
that produces type VII collagen.
"Our goal is to determine the usefulness of stem cells, whether from
the umbilical cord blood or adult tissues like bone marrow, in the
treatment of human disease," said John E. Wagner, M.D., professor of
Pediatrics and director of the Division of Hematology, Oncology, and
Blood and Marrow Transplantation and director of clinical research of
the Stem Cell Institute at the University of Minnesota. "There are
hundreds of thousands of children and adults waiting for new
breakthroughs in stem cell research, and time is never enough. In two
years, the team was able to move this project forward remarkably fast-
-from testing in animal models to treating patients. Time will tell
whether this risky treatment will work as effectively in humans. But,
RDEB is a horribly debilitating, life-threatening disease with no
existing curative therapy."
The boy received both umbilical cord blood and bone marrow from a
perfectly matched sibling. If the results mimic the animal model,
doctors anticipate the healthy blood system will aid in the skin's
ability to produce type VII collagen necessary to anchor the skin and
lining cells of the gastrointestinal tract to the body. Doctors
anticipate in early 2008--approximately 100 days after transplant--
they will be able to judge whether this the treatment helped.
"This represents a real change in thinking within the dermatological
community. The possibility of this approach compels us to explore
more broadly the way some skin diseases are typically treated," said
Maria Hordinsky, M.D., head of the Department of Dermatology at the
University of Minnesota and member of the care team.
The transplant was done as part of a clinical trial funded by
donations made to the University of Minnesota EB Fund and Children's
Cancer Research Fund in Minneapolis.
Adapted from materials provided by University of Minnesota.
http://www.scienced
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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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