Major breakthrough for new haemophilia A treatment

Drug trials provide mind-blowing results.

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 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 Factor VIII levels even many months after treatment.

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

The trial saw patients across England injected with a copy of the missing gene, which allows their cells to produce the missing clotting factor. Following patients for up to 19 months, tests show that 11 out of 13 patients in the trial now have normal or near normal levels of the previously missing factor and all thirteen patients have been able to stop their previously regular treatment.

Professor John Pasi, Haemophilia Centre Director at Barts Health NHS Trust and Professor of Haemostasis and Thrombosis at Queen Mary University of London explained: "We have seen mind-blowing results which have far exceeded our expectations. When we started out we thought it would be a huge achievement to show a 5% improvement, so to actually be seeing normal or near normal factor levels with dramatic reduction in bleeding is quite simply amazing. We really now have the potential to transform care for people with haemophilia using a single treatment for people who at the moment must inject themselves as often as every other day. It is so exciting."

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

Professor Pasi continued: "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."