The lives of 13 men with Haemophilia A in the UK have been transformed with a new gene therapy, which has shown to offer long-term benefits. The treatment, developed by Professor John Pasi and colleagues at Queen Mary University of London, was first announced in 2017. According to the reports 85% of men treated with a single infusion of a missing gene were showing normal or near-normal levels of the blood-clotting protein, factor VIII, one year on.

Now, the authors published a new paper in the New England Journal of Medicine confirming that none of the patients who participated in the trial had required regular factor VIII to prevent bleeding during that period. All of them are still benefiting from a substantial fall in the rates of bleeding three years after receiving the treatment.

In patients with Haemophilia A (which accounts for around 80 per cent of all Haemophilia cases) the blood unable to clot due to lack of factor VIII. This causes risk of excessive bleeding from even the slightest injury — as well as potentially life-threatening spontaneous internal bleeding. Eliminating intravenous injections that Haemophilia A patients have to receive each week to control and prevent bleeding greatly reduces the burden of treatment.

Our 2017 paper showed that gene therapy could significantly boost factor VIII levels in men with Haemophilia A. Our new data are critical in helping the scientific and medical communities understand this pioneering technology. 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,” said Professor Pasi, who is also Director of the Haemophilia Centre at Barts Health NHS Trust.

The treatment may be particularly important in the developing world where access to clotting products is difficult.


Source: Science Daily