Tyrone man at the heart of new medical breakthrough

A TYRONE-born pharmacist has spoken of his pride after helping deliver a potentially life-changing gene therapy to a haemophilia B patient – the first time the treatment has been administered on the NHS in the UK.

Anthony Hackett, originally from Glencull and now Principal Pharmacist for Advanced Therapies at Guy’s and St Thomas’ NHS Foundation Trust in London, described the moment as a ‘proud day for the Trust’ and a ‘breakthrough’ for patients.

Haemophilia B is an inherited bleeding disorder caused by a lack of Factor IX, an important blood-clotting protein. Patients often experience spontaneous bleeding leading to chronic joint pain.

Up to now, these patients face the burden of regular infusions of clotting factors up to twice a week.

The treatment, Hemgenix (etranacogene dezaparvovec), is a one-off gene therapy given as a single infusion lasting one to two hours that could eliminate the need for regular injections of factor IX and prevent patients from having to experience painful bleeds.

It works by introducing a working copy of the Factor IX gene into the body.

This allows the liver to start producing the protein naturally, reducing or even eliminating bleeding episodes.

“It’s a very new and personalised way of delivering medicine,” said Anthony.

“With a select screening criteria, a lot of preparation takes place to make sure the patient is an appropriate recipient. Ultimately, my role is to make sure medicines are safe for patients.”

The treatment has already been used in the United States, but this was the first time it was given to a patient through the NHS.

“There are currently talks of introducing the treatment in paediatric clinical trials,” he continued.

“However, Hemgenix is only licensed and available for adults with moderately severe or severe haemophilia B, so any potential paediatric use would require separate regulatory approval”

“If we can eventually treat haemophilia B in children rather than waiting until adulthood, we could intervene much earlier to reduce or prevent the adverse effects of the condition. This early intervention approach could significantly improve patients’ long-term quality of life.”

The first patient to receive Hemgenix on the NHS is from the North East of England and was diagnosed with the condition in childhood.

“For now, we will spend the next 15 to 20 years monitoring and observing the responses of those who have received the treatment to ensure its long-term safety and effectiveness,” Anthony said.

It is funded through NHS England’s Innovative Medicines Fund (IMF).

Around 2,000 people in the UK live with haemophilia B.

The National Institute for Health and Care Excellence (NICE) estimates that approximately 250 patients in England could be eligible for the new treatment.