Potential new treatment for haemophilia developed by Cambridge researchers

A new treatment that might one day help all patients with haemophilia, including those that become resistant to existing therapies, has been developed by researchers at the University of Cambridge. Within three years, we hope to be conducting our first-in-man trials - Trevor Baglin Around 400,000 individuals around the world are affected by haemophilia, a genetic disorder that causes uncontrolled bleeding. Haemophilia is the result of a deficiency in proteins required for normal blood clotting - factor VIII for haemophilia A and factor IX for haemophilia B. Currently, the standard treatment is administration of the missing clotting factor. However, this requires regular intravenous injections, is not fully effective, and in about a third of patients results in the development of inhibitory antibodies. Nearly three-quarters of haemophilia sufferers have no access to treatment and have a life-expectancy of only 10 years. In a study published online today in Blood , the Journal of the American Society of Hematology, researchers report on a novel approach that gives the clotting process more time to produce thrombin, the enzyme that forms blood clots.  They suggest this treatment could one day help all patients with haemophilia, including those who develop antibodies against standard therapy.