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Pediatric Blood and Marrow Transplant Center

Pediatric BMT Program doctors publish a new treatment for a painful and lethal skin disease via BMT

University of Minnesota physician-researchers have demonstrated that Epidermolysis Bullosa, a lethal skin disease, can be successfully treated with stem cell therapy.

In a study published in the August 12th edition of the New England Journal of Medicine, the Pediatric BMT team of Dr. John Wagner, Director, and Dr. Jakub Tolar, reported the amazing results from their first BMT clinical trial to treat Epidermolysis Bullosa (EB), a rare, genetic skin disease where even the slightest friction causes the skin and the lining of their throats to blister and scrape off.

In this trial, Drs. Wagner and Tolar performed BMT to replace the children's defective skin cells with ones from a healthy donor that would create the crucial protein lacking and responsible for anchoring the skin to the body. Seven children were enrolled in the study and the five children who survived showed remarkable improvements. Sadly, two enrolled pioneering patients died as a result of treatment complications.

The same encouraging results were seen for the five additional children who have been treated since the report was written. Drs. Wagner and Tolar are continuing the work and have opened a new study using a mixture of bone marrow and mesenchymal stem cells to further improve the treatment.

Read The Full Article (opens in new window):

"Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa." John E. Wagner, Akemi Ishida-Yamamoto, John A. McGrath, Maria Hordinsky, Douglas R. Keene, Megan J. Riddle, Mark J. Osborn, Troy Lund, Michelle Dolan, Bruce R. Blazar, and Jakub Tolar. N Engl J Med 2010; 363:629-639; August 12, 2010.

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Looking for information for adult patients? Visit The U of M Adult Blood and Marrow Transplant Program