Researchers at the Children’s Hospital of Philadelphia (CHOP) have collaborated with the School of Veterinary Medicine at the University of Pennsylvania to explore a new treatment for dogs suffering from hemophilia. Their study, published in the journal Molecular Therapy Methods and Clinical Development, investigates the use of adeno-associated viral (AAV) gene therapy as a potential life-changing treatment for companion animals with severe forms of this hereditary bleeding disorder.
Hemophilia, though rare in dogs, remains the most common inherited bleeding disorder in canines, traditionally managed with blood transfusions. Researchers see promise in AAV gene therapy, already successful in treating human conditions like hemophilia A and B, to make this treatment feasible for dogs.
Benjamin J. Samelson-Jones, MD, PhD, an attending physician in the Division of Hematology at CHOP, emphasized the potential impact of the treatment, stating, “Our findings show that AAV-liver-directed gene therapy could be a promising treatment for dogs with hemophilia.” Samelson-Jones noted further, “While more research is needed before AAV gene therapy can enter veterinary clinics, our research suggests that companion dogs could reside in their homes with their owners and receive regular blood clotting tests and immune system checks.”
The study observed 12 dogs treated with the AAV gene therapy, demonstrating a 94% reduction in bleeding rates and a 61% improvement in quality of life over an average period of 4.1 years. This is the first study measuring quality of life in hemophilic dogs, reporting that after treatment, dogs became more active and faced fewer health issues.
Funding for this research came from the National Heart, Lung, and Blood Institute, the National Center for Advancing Translational Sciences, and the Institute for Translational Medicine and Therapeutics at the Perelman School of Medicine and the School of Veterinary Medicine at the University of Pennsylvania.
Doshi, Benjamin J. Samelson-Jones et al. published their findings in an article titled “AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life,” available online since March 14, 2024.
