Antiviral Drugs Prolong Survival in Murine Recessive Dystrophic Epidermolysis Bullosa

March 2024 in “ EMBO molecular medicine ”

Grace Tartaglia, Ignacia Fuentes, Neil Patel, Abigail Varughese, L Israel, Pyung Hun Park, Michael H. Alexander, Shiv Poojan, Qingqing Cao, Brenda Solomon, Zachary Padron, John A. Dyer, Jemima E. Mellerio, John A. McGrath, Francis Palisson, Julio C. Salas‐Alanís, Han Chieh Lin, Andrew P. South

daclatasvir recessive dystrophic epidermolysis bullosa RDEB fibrosis TGFβ pathway extracellular matrix pruritus-induced hair loss

TLDR Antiviral drugs, especially daclatasvir, may be a new treatment for a rare skin disease, improving survival and reducing symptoms in mice.

The study "Antiviral drugs prolong survival in murine recessive dystrophic epidermolysis bullosa" investigated the potential of antiviral drugs, specifically daclatasvir, as a treatment for recessive dystrophic epidermolysis bullosa (RDEB), a rare inherited skin disease. The researchers screened 1443 FDA-approved compounds and found 43 drugs that normalized the disease phenotype. Daclatasvir showed potent pro-survival and anti-fibrotic effects in a preclinical mouse model of RDEB, improving fibrosis, overall quality of life, and survival, while also increasing weight gain and activity, and reducing pruritus-induced hair loss. The drug was found to target the TGFβ pathway and reduce total collagen retained in the extracellular matrix. This suggests that daclatasvir could be a potential therapeutic option for patients with RDEB and possibly other fibrotic diseases.

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