A collaborative effort from German and Italian researchers allowed a child dying from severe epidermolysis bullosa (EB) to lead a healthy, normal life. EB is a genetic disorder which causes the top layer of the skin (epidermis) to become extremely fragile and easily blister-prone. Patients with EB typically do not live past the age of 30, given the exorbitant risk of infections and other complications of having “paper thin” skin, and there is currently no cure. However, a recent experimental skin graft, made from the patient’s own stem cells, allowed a young boy to return to normalcy. The graft’s success comes from a technique of genetic engineering to correct the defective gene that causes EB in immature stem cells, and then develops those stem cells into layers of epithelial tissue and applies them to the patient’s affected areas. Though the risk of such procedures is high, using the patient’s own cells minimizes the risk of rejection and provides a safer alternative to merely enduring this disease.
Researchers at the University of Minnesota, world leaders in the treatment of Epidermolysis Bullosa (EB), have developed a stem cell treatment [utilizing mesenchymal stem cells – MSCs] to treat the disorder. This devastating condition involves problems with connective tissue, making skin blister and tear with the slightest contact; severe cases impact internal organ tissues as well. Affecting 1 in every 20,000 children, the disorder can lead to severe infections and be fatal. The team at the University utilized a treatment involving complete renewal of the immune system through chemotherapy and a bone marrow transplant, followed by the administration of mesenchymal stem cells to regenerate the skin tissue.