Gene therapy used in clinical trial for person with haemophilia

Gene therapy has been used to treat a person with haemophilia for the first time in Ireland, a patient group has announced.

The Irish Haemophilia Society (IHS) confirmed on Thursday morning that the person received gene therapy as part of a clinical trial. IHS chief executive Brian O'Mahony said the treatment is "a momentous occasion for the haemophilia community in Ireland".

The general term haemophilia describes a group of inherited blood disorders in which there is a life-long defect in the clotting mechanism of the blood.

Since the 1970s, haemophilia has been treated by the administration of intravenous infusions of the missing clotting factor. However, work done by companies and academic institutions has given new hope that an effective treatment could be based on gene therapy, the IHS said.

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The clinical trial uses a “viral vector” to deliver gene therapy to the person’s liver intravenously. “In the past, viruses such as HIV and Hepatitis C decimated the haemophilia population in Ireland through contaminated blood. It is ironic that a virus could now be the delivery system which offers the best hope of a practical cure for severe haemophilia,” Mr O’Mahony said.

“It is hoped that the effect of the gene therapy infusion will last for many years and possibly for a lifetime.”

The principal investigator on the trial in Ireland is Dr Niamh O’Connell of the National Coagulation Centre in St. James’s Hospital. She said the gene therapy was “ground breaking”.

“The opportunity to participate in clinical trials is part of the commitment of the National Haemophilia Service to personalise treatment and to improve the quality of life and outcomes for people with haemophilia.”

The study, which is being run by drug manufacturer UniQure, involves three Irish patients among a total of 60 around the world. There will be an intensive period of monitoring of effectiveness at first, followed by a longer term evaluation over five years. Only one treatment is administered to trial patients.

The particular gene therapy is focused on patients who are missing clotting factor IX, the second most-common type of haemophilia. Earlier results show that the level of clotting factor increased from 1 per cent - generally seen as severe haemophilia - to between 33 and 51 per cent in a small number of individuals treated, levels seen in mild cases or even amongst the non-haemophiliac population.

Professor Martina Hennessy of the Wellcome HRB Clinicial Research facility in St James's, where the gene therapy was infused, said that "access to high quality research is an integral part of good healthcare because it raises standards and pushes the boundaries of what can be achieved.

“Delivering gene therapy requires specialised training and equipment, we have been preparing with Dr O’Connell and her team for over a year to undertake this exciting work, in partnership with the Irish Haemophilia Society. Other trials are planned, we hope this expertise leads other Irish patient groups also being able to access potentially life changing treatments in the future,” she said.