Stem Cell Therapy for Epidermolysis Bullosa—Does It Work?

Katsuto Tamai, Jouni Uitto

Epidermolysis bullosa is a group of heritable skin fragility disorders with considerable morbidity and mortality. It is known to be caused by mutations in as many as 18 distinct genes, but there is no specific or effective treatment. Preclinical developments of gene correction, protein replacement, and cell-based approaches for treatment have suggested new therapeutic avenues, and some of them, including bone marrow transplantation and mesenchymal stem cell therapy, have entered into early clinical trials. Hammersen et al. report on two patients with severe generalized junctional epidermolysis bullosa treated with allogeneic stem cell therapy, but with little success. Careful examination of the existing literature suggests that current approaches of cell-based therapies may be helpful in ameliorating some of the clinical features and symptoms in these patients, but advanced strategies, with improved safety profiles, are required for development of durable therapy for these currently intractable disorders.