Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions
Cell therapy closes large wounds in epidermolysis bullosa
Key clinical point: Recurrent and chronic wounds are different; the latter may be healed using a novel gene therapy.
Major finding: Larger wounds were associated with a significantly greater impact on quality of life in adult and pediatric with the recessive dystrophic type of EB (RDEB); and in adults with RDEB, treated with gene-corrected autologous epidermal grafts, 95% of chronic open wounds healed by 6 months, 68% at 1 year and 71% at 2 years, compared with 0%, 17%, and 17% of control wounds.
Study details: A global survey of 85 individuals (47 children and 38 adults) with RDEB, and a phase 1/2 trial of gene therapy in seven adults with RDEB.
Disclosures: Dr. Gorell did not provide a conflict of interest statement. Dr. Tang disclosed receipt of honoraria or consultation fees from Abeona and Menlo Therapeutics and being a stock shareholder in PellePharm (BCC company) and BridgeBio. Dr. Tang also acknowledged receiving research grants from the EB Research Partnership, the Epidermolysis Medical Research Foundation, and the California Institute for Regenerative Medicine.
Gorell ES. EB World Congress, poster 29. Tang JYl. EB World Congress, oral presentation.