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

Ellie Rashidghamat 1, Tendai Kadiyirire 1, Salma Ayis 2, Gabriela Petrof 3, Lu Liu 4, Venu Pullabhatla 5, Chrysanthi Ainali 6, Alyson Guy 4, Sophia Aristodemou 4, James R McMillan 4, Linda Ozoemena 4, John Mee 7, Rashida Pramanik 1, Alka Saxena 5, Rosamund Nuamah 5, Emanuele de Rinaldis 8, Sonia Serrano 9, Clarisse Maurin 9, Magdalena Martinez-Queipo 1, Su M Lwin 1, Dusko Ilic 10, Anna Martinez 3, Francesco Dazzi 11, Ineke Slaper-Cortenbach 12, Kasper Westinga 12, Sabrina Zeddies 12, Marcel van den Broek 12, Alexandros Onoufriadis 1, Jemima E Mellerio 1, John A McGrath

Main idea: Mesenchymal stromal/stem cells infusion is safe in recessive dystrophic epidermolysis bullosa adults and can have clinical benefits for at least 2 months.

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.

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.

Source NIH