Groundbreaking gene therapy could cure haemophilia

Groundbreaking gene therapy could cure haemophilia

A ‘cure’ for haemophilia is one step closer, following results published in the New England Journal of Medicine of a groundbreaking gene therapy trial led by the NHS in London, UK.

Clinical researchers at Barts Health NHS Trust and Queen Mary University of London have found that over one year on from a single treatment with a gene therapy drug, participants with haemophilia A (the most common type) are showing normal levels of the previously missing protein, and effectively curing them.

A single infusion of the gene therapy drug showed improved levels of the essential blood clotting protein Factor VIII, with 85% of patients achieving normal or near-normal levels even many months after treatment.

The ‘transformational’ results have particular significance as the first successful gene therapy trial for haemophilia A.

Professor John Pasi, Haemophilia Centre Director at Barts Health NHS Trust and Professor of Haemostasis and Thrombosis at Queen Mary University of London, said: “Incredibly exciting is the potential for a significant change in how haemophilia is treated globally. A single dose of medication that can so dramatically improve the lives of patients across the world is an amazing prospect.”

There are around 2,000 people with severe haemophilia A in the UK alone. A hereditary genetic condition dominantly affecting men, severe cases have virtually none of Factor VIII .

Haemophilia puts those affected at risk of excessive bleeding even from the slightest injury, as well as causing spontaneous internal bleeding, which can be life-threatening.

Recurring bleeding into joints can lead to progressive joint damage and arthritis.

The only current treatment involves multiple weekly injections to control and prevent bleeding, but there is no cure.

The team will now hold further tests widening participants globally to include people in the US, Europe, Africa and South America.


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