Hemophilia B is cured by a ‘Transformational’ Therapy

British Doctor says people with bleeding disorders hemophilia were effectively cured by a ‘transformational’ therapy.

Transformational therapy corrects a genetic defect that leaves people's blood struggling to clot and stop bleeding. Elliott Mason was part of the trial which tested the treatment and says his life now feels "completely normal." The experts say, in the next few years, such advancement could revolutionize the lives of adults with hemophilia.

Elliott was given an engineered virus with instructions to manufacture the missing factor IX. The virus is a microscopic postman that delivers the blueprints to the liver, producing the clotting protein. The process was a one-off infusion and took about an hour to drip into the body of Elliott. He was astonished to see the amount of factor IX in his blood go from only 1% of normal levels to normal.

In the New England Journal of Medicine, the finding of Transformational therapy was published. It showed nine out of 10 patients who were given the therapy no longer needed their clotting factor IX injections. However, gene therapy costs between £150,000 and £200,000 per year. And the study shows that the treatment will take at least a decade. The chairman of the Haemophilia Society, Clive Smith, said: "This initial data is promising, but we continue to monitor gene therapy trials closely and cautiously, as with all new treatments.