A randomized controlled trial of a novel skin substitute on epidermolysis bullosa wounds

Summary: Abstract Body: Background: Recessive Dystrophic Epidermolysis Bullosa (RDEB) exhibits severe manifestations including extensive areas of wounds, increased risk of squamous cell carcinoma, anemia, skin infections, pain, itch, and early mortality. The topical drugs, beremagene-geperpavec and birch-triterpenes, have been approved for RDEB wound healing but are not curative. We have developed a cell therapy approach utilizing genetically corrected patient-derived stem cells to heal RDEB wounds. Application of cell therapy requires a matrix that can suspend and allow cells to attach to the wound bed. RDEB patient skin is extremely fragile and prone to injury from adhesive dressings. We tested a nano spun polymer spray matrix as a vehicle to ensure safety, tolerability, and wound healing in RDEB wounds. Method: RDEB patients, age 6 years or older with at least six wounds of 10cm2 were enrolled at a single site. The primary endpoint was 90% wound closure from baseline to Month 4. Secondary endpoints included wound pain, itch and adverse events (AEs). Results and Discussion: We evaluated 42 wounds in 6 RDEB patients (mean age of 21 years). Wounds were matched by size and location, and were randomized either to Spincare matrix or control. Subjects applied the nano spun matrix weekly and did not report increased blistering, pain, or itch compared with untreated wounds. Importantly, Spincare matrix did not significantly change wound healing over 4 months using monthly evaluations/photos and did not increase wound infection. No AEs were related to Spincare® and its discontinuation. Results from this pilot trial show that Spincare matrix treatment on RDEB wounds is a safe and tolerable cell therapy delivery method for a future Phase 1 trial of gene-edited induced pluripotent stem cells. Yuri Ikeda¹, Pirunthan Pathmarajah¹, Ricardo V. Gaona¹, Jenny Deng¹, Estephannie Alvarez¹, Vaishali Mittal¹, Jessica Torkelson^{1, 2}, Hanson H. Zhen^{1, 2}, Irene Bailey¹, Dawn H. Siegel¹, Anthony Oro^{1, 2}, Jean Y. Tang¹ 1. Department of Dermatology, Stanford University School of Medicine, Stanford, CA, United States. 2. Center for Definitive and Curative Medicine, Stanford University School of Medicine, Stanford, CA, United States. Clinical Research: Interventional Research