British researchers have just developed a revolutionary gene therapy for people with hemophilia A. According to their initial tests, it has been successfully tested on 13 men suffering from this genetic disorder.
Hemophilia, which affects about 20000 people in the US and 700,000 worldwide, is a rare disease that prevents blood from clotting, which carries a risk of bleeding. Carried in the X chromosome and mainly affecting men, it can be particularly painful and can even cause death if the bleeding is not treated in time.
Caused by the lack or absence of factor VIII, a clotting factor generally present in our plasma that allows the blood to clot, hemophilia A is the most common form: 80% of patients suffering from this genetic disease are affected with the A variant. Because their blood cannot clot properly, patients are exposed to the risk of excessive bleeding at the slightest cut or injury, but also to the risk of spontaneous internal bleeding. Therefore, they are given at least three intravenous injections per week to control and prevent bleeding.
But new research in gene therapy may allow patients to eliminate these regular injections, which affect their quality of life. In a paper published in the New England Journal of Medicine, researchers from Queen Mary University in London, United Kingdom, announced that a single treatment resulted in 13 patients having normal or near-normal levels of blood clotting protein, factor VIII, even after one year.
The treatment is still effective after 3 years
Although this preliminary work had already been published in 2017, this time the authors announced that, three years after receiving treatment, all patients in the study still benefited from a substantial reduction in bleeding rates. The 13 patients No longer needed regular infusions of factor VIII to prevent bleeding.
“Our 2017 paper showed that gene therapy could significantly boost factor VIII levels in men with hemophilia A. Our new data are critical in helping the scientific and medical communities understand this pioneering technology,” explained Pasi. “This latest study confirms both safety and the long-term beneficial impact of the treatment. A long-term treatment that effectively ends the life-long regime of regular injections can transform care and massively improve the quality of life of hundreds of thousands of people born with this challenging genetic condition.”
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