News Release 

Gene therapy shown to offer long-term benefits for people with Haemophilia A

Queen Mary University of London

A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK.

Clinical researchers led by Professor John Pasi from Queen Mary University of London first announced in 2017 that 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.

In a new paper published in the New England Journal of Medicine, Professor Pasi, who is also Director of the Haemophilia Centre at Barts Health NHS Trust, confirmed that all the patients involved in the trial were still benefiting from a substantial fall in the rates of bleeding three years after receiving the treatment. None of the 13 patients had required regular factor VIII to prevent bleeding during that period.

The 13 patients included five patients from the Royal London Hospital, part of Barts Health NHS Trust.

A lack of factor VIII causes Haemophilia A, which accounts for around 80 per cent of all Haemophilia cases. With the blood unable to clot, patients are at risk of excessive bleeding from even the slightest injury - as well as potentially life-threatening spontaneous internal bleeding. Patients have to undergo three or more intravenous injections each week to control and prevent bleeding. Eliminating these regular injections greatly reduces the burden of treatment needed to keep bleeding at bay.

Professor Pasi said: "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."

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

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For more information, please contact:

Chris Mahony Public Relations Manager (School of Medicine and Dentistry)
Queen Mary University of London
c.mahony@qmul.ac.uk
Tel: +44 (0)20 7882 5315

Notes to the editor

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A Pasi, J et al New England Journal of Medicine

DOI: 10.1056/NEJMoa1908490

Available here: https://www.nejm.org/doi/full/10.1056/NEJMoa1908490?query=featured_home

About Queen Mary University of London

At Queen Mary University of London, we believe that a diversity of ideas helps us achieve the previously unthinkable.

In 1785, Sir William Blizard established England's first medical school, The London Hospital Medical College, to improve the health of east London's inhabitants. Together with St Bartholomew's Medical College, founded by John Abernethy in 1843 to help those living in the City of London, these two historic institutions are the bedrock of Barts and The London School of Medicine and Dentistry.

Today, Barts and The London continues to uphold this commitment to pioneering medical education and research. Being firmly embedded within our east London community, and with an approach that is driven by the specific health needs of our diverse population, is what makes Barts and The London truly distinctive.

Our local community offer to us a window to the world, ensuring that our ground-breaking research in cancer, cardiovascular and inflammatory diseases, and population health not only dramatically improves the outcomes for patients in London, but also has a far-reaching global impact.

This is just one of the many ways in which Queen Mary is continuing to push the boundaries of teaching, research and clinical practice, and helping us to achieve the previously unthinkable.

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