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Oxford NHS researchers recruit global first patient

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Researchers at Oxford's Churchill Hospital have recruited the first patient in the world to a trial into using gene therapy to attempt to cure haemophilia B.

Haemophilia B is a rare genetic disorder where the protein Factor IX, which helps the blood to clot, is missing. In the most severe cases, patients are at risk of frequent episodes of bleeding into their joints, resulting in arthritis at an early age. There is currently no cure.

Gene therapy is a new treatment for haemophilia B, where faulty or missing genes are replaced with healthy ones. The FIX-GT trial - supported by the National Institute of Health Research (NIHR) - is testing the safety and effectiveness of giving haemophilia B patients the gene that produces the Factor IX protein.

Simon Fletcher, lead research nurse at the Churchill Hospital’s Oxford Haemophilia and Thrombosis Centre, said: "If this trial is successful and shows that the new treatment is capable of curing Haemophilia B then it is going to be transformative for patients.

"We've seen with recent press attention around a haemophilia A gene therapy study that a number of patients have contacted us asking if we are involved in the study and wanting to take part. It's good that it has raised the profile of research into haemophilia and seems to be encouraging an increase in the number of patients wanting to take part in clinical trials.

"We had a long lead-in time setting up the study. We were therefore able to speak to a number of our patients ahead of the start-up to see who would be interested."

FIX-GT is funded by Freeline Therapeutics Limited and sponsored by University College London, with local support from Oxford University Hospitals NHS Foundation Trust, which runs the Churchill Hospital.

Simon said: "The secret to recruiting the first global patient was close communication between the research staff and the patients so that we were able to coordinate and plan everything properly."

The patient on the trial said: "I have always felt my condition stopped me from going off and travelling the world.  

"I hope that as a result of the study I may at last be able to do this. I would love to just pick up a backpack and go off around Southeast Asia."

The study team aim to recruit six participants between four sites: the Churchill Hospital in Oxford, the Royal Free Hospital and the St Thomas' Hospital in London and the Royal Hallamshire Hospital in Sheffield before it closes on 10 August 2018.