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

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 LwinDusko Ilic, Anna E. Martinez, Francesco Dazzi, Ineke Slaper-Cortenbach, Kasper Westinga, Sabrina Zeddies, Marcel van den Broek, Alexandros Onoufriadis, J Mellerio, John A. McGrath

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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.

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

Keywords

  • BM-MSC
  • RDEB
  • epidermolysis bullosa
  • mesenchymal stromal cells

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