Treatment found for Dystrophic Epidermolysis Bullosa ?

Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa

Wagner J.,  Ishida-Yamamoto A., McGrath J., Hordinsky M.,  Keene R.,  Riddle M., Osborn M., Lund T., Dolan M., Blazar B, Tolar J.

N Engl J Med 2010; 363:629-639. August 12, 2010

Epidermolysis Bullosa is a genetic condition in most cases and different mutations affect the epidermis, basal membrane and dermis according to the molecule mutated and thus dysfunctional. In one type of recessive dystrophic epidermolysis bullosa, collagen VII, which is an anchoring protein of the basal membrane to the dermis is mutated, with severe consequences which include respiratory problems in addition to the recurrent and blistering skin lesions. The disease is disabling, with infections, feeding difficulties, spinous cell carcinomas (SCC) among other complications.

The University of Minnesota has been doing extensive research on the condition,  but the results on the efficacy of bone marrow transplantation (BMT) (allogenic BMT) + myeloablative chemotherapy in a clinical trial are very promising.

6 patients out of seven 7 (clinical documentation and presence of collagen VII deposited with the donor cells present (5patients))

We encourage you to read the full article on this new potential application of stem-cell therapy.