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

• Published in: Journal of the American Academy of Dermatology Vol. 83; no. 2; pp. 447 - 454
• Main Authors: Rashidghamat, Ellie, Kadiyirire, Tendai, Ayis, Salma, Petrof, Gabriela, Liu, Lu, Pullabhatla, Venu, Ainali, Chrysanthi, Guy, Alyson, Aristodemou, Sophia, McMillan, James R., Ozoemena, Linda, Mee, John, Pramanik, Rashida, Saxena, Alka, Nuamah, Rosamund, de Rinaldis, Emanuele, Serrano, Sonia, Maurin, Clarisse, Martinez-Queipo, Magdalena, Lwin, Su M., Ilic, Dusko, Martinez, Anna, Dazzi, Francesco, Slaper-Cortenbach, Ineke, Westinga, Kasper, Zeddies, Sabrina, van den Broek, Marcel, Onoufriadis, Alexandros, Mellerio, Jemima E., McGrath, John A.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.08.2020
• Subjects: Adolescent; Adult; Aged; BM-MSC; epidermolysis bullosa; Epidermolysis Bullosa Dystrophica - complications; Epidermolysis Bullosa Dystrophica - diagnosis; Epidermolysis Bullosa Dystrophica - therapy; Female; Humans; Infusions, Intravenous; Male; Mesenchymal Stem Cell Transplantation - methods; mesenchymal stromal cells; Middle Aged; Prospective Studies; Pruritus - diagnosis; Pruritus - etiology; Pruritus - therapy; Quality of Life; RDEB; Severity of Illness Index; Transplantation, Homologous - methods; Treatment Outcome; Wound Healing; Young Adult; epidermolysis bullosa; CI; mesenchymal stromal cells; BM-MSC; RDEB; BM; SD; SCC; C7; SEM; MSC; EB; HMGB-1
• ISSN: 0190-9622; 1097-6787; 1097-6787
• DOI: 10.1016/j.jaad.2019.11.038

Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary blistering disorder due to a lack of type VII collagen. At present, treatment is mainly supportive. 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. 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 × 106 cells/kg). 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. Open-label trial with no placebo. MSC infusion is safe in RDEB adults and can have clinical benefits for at least 2 months.