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

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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.

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