A Phase I/II open-label trial of intravenous allogeneic mesenchymal stromal cell therapy in adults with recessive dystrophic epidermolysis bullosa

Abstract Background Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary blistering disorder due to lack of type VII collagen (C7). At present, treatment is mainly supportive. Objective To determine whether intravenous allogeneic bone marrow-derived mesenchymal stromal/stem cells (BM-MSC) are safe in RDEB adults and if the cells improve wound healing and quality of life. Methods We conducted a prospective, phase I/II, open-label study recruiting 10 RDEB adults to receive two intravenous infusions of BM-MSCs (Day 0 and Day 14; each dose 2-4 x 106 cells / kg). Results BM-MSCs were well tolerated with no serious adverse events to 12-months. Regarding efficacy, there was transient reduction in disease activity scores (8 of 10 subjects) and significant reduction in itch. One individual showed a transient increase in C7. Limitations Open label trial with no placebo. Conclusions MSC infusion is safe in RDEB adults and can have clinical benefits for at least 2 months.