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Person with haemophilia successfully treated with gene therapy in Ireland for first time

It’s hoped the breakthrough will have a major impact on the quality of life of people with the condition.

Image: Shutterstock/Billion Photos

GENE THERAPY HAS been used to treat a person with haemophilia for the first time in Ireland, the Irish Haemophilia Society has announced today.

The person received the therapy as part of a clinical trial. 

The society hailed it a major breakthrough and said it would have a major impact on the quality of life of people with the condition. 

Haemophilia is a genetic condition where a person’s blood doesn’t clot properly, meaning they can bleed much more profusely.

Since the 1970s, the standard treatment for haemophilia has been regular injections of the missing clotting factor – usually managed by the patient themselves. 

The new gene therapy helps the body independently produce the clotting factor, providing continuous bleed protection.  

“This is a momentous occasion for the haemophilia community in Ireland”, Irish Haemophilia Society Chief Executive Brian O’Mahony said.

“In the past, viruses such as HIV and Hepatitis C decimated the haemophilia population in Ireland through contaminated blood.”

Dr Niamh O’Connell, National Director of the National Coagulation Centre at St. James’s Hospital, said:

Having your own liver producing a continuous level of factor is a real game-changer versus the treatments we currently have.

O’Mahony added: “In the coming years, you might start to see people actually being changed from having severe haemophilia into mild, or even normal factor levels. So it’s quite exciting.”

More trials are needed before the gene therapy becomes more widely used. 

“There’s still lots to be learned. And I think the clinical trial setting is the way that we manage to get the benefits of new treatments in a safe environment, while gathering all of this information and data that helps us to improve the next round of studies,” Martina Hennessy, Director of the Health Research Board Clinical Research facility said. 

There are a number of people enrolled for the current trial, and there are further trials about to open for other forms of haemophilia. 

The trials were an academic collaboration between Trinity College and St James’s Hospital, and are funded by the Wellcome Trust in the UK and the Health Research Board in Ireland. 

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