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U of M performs first systemic therapy for fatal childhood disease
Date:11/2/2007

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 fastfrom 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 skins 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 2008approximately 100 days after transplantthey 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.


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Contact: Molly Portz
mportz@umn.edu
612-625-2640
University of Minnesota
Source:Eurekalert

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