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

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Elham Rashidghamat, Tendai Kadiyirire, Salma Ayis, Gabriela Petrof, Lu Liu, Venu Pullabhatla, Chrysanthi Ainali, Alyson Guy, S Aristodemou, James R. McMillan, L Ozoemena, John Mee, Rashida Pramanik, Alka Saxena, Rosamond Nuamah, Emanuele de Rinaldis, Sonia Serrano, Clarisse Maurin, Magdalena Martinez-Queipo, Su Lwin & 10 more Dusko Ilic, Anna E. Martinez, Francesco Dazzi, Ineke Slaper-Cortenbach, Kasper Westinga, Sabrina Zeddies, Marcel van den Broek, Alexandros Onoufriadis, J Mellerio, John A. McGrath

Original languageEnglish
JournalJournal of the American Academy of Dermatology
Early online date28 Nov 2019
DOIs
Publication statusE-pub ahead of print - 28 Nov 2019

King's Authors

Abstract

Background: Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary blistering disorder due to a lack of type VII collagen. At present, treatment is mainly supportive. Objectives: To determine whether intravenous allogeneic bone marrow–derived mesenchymal stromal/stem cells (BM-MSCs) 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 2 intravenous infusions of BM-MSCs (on day 0 and day 14; each dose 2-4 × 10 6 cells/kg). Results: BM-MSCs were well tolerated with no serious adverse events to 12 months. Regarding efficacy, there was a transient reduction in disease activity scores (8/10 subjects) and a significant reduction in itch. One individual showed a transient increase in type VII collagen. 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.

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