The revolutionary treatment will mean patients no longer require a weekly injection
Nigel was diagnose with haemophilia at eleven months old(Image: Cardiff and Vale University Health Board)
The first person in Wales has received a ground-breaking haemophilia treatment at a brand new facility at the University Hospital of Wales. The “life changing” gene therapy infusion means those who suffer from the disorder will no longer need weekly injections to stop bleeds.
Nigel Miller, 61, is the first person in Wales to receive the treatment having been diagnosed with the blood clotting disorder aged just 11 months old.
The Kidwelly man described it as being both an honour and a responsibility to be the first person to receive the treatment, and said he was proud that living with severe haemophilia would “finally begin to look very different”. For the biggest stories in Wales first, sign up to our daily newsletter here.
Nigel said: “Having the opportunity to receive gene therapy is so extraordinary. This is pioneering treatment and being first is an honour, but also a responsibility. I am deeply aware that my experience will help inform what comes next for others in Wales and beyond.
“I have had a weekly injection to stop my bleeds for as long as I can remember but being the first person in Wales to receive gene therapy for haemophilia B has meant an end to that.
“This is not a cure in the traditional sense, but it means I don’t have to inject myself weekly anymore. Instead of repeatedly replacing the missing clotting factor, gene therapy gives my body the instructions it has always lacked — enabling it to produce factor IX itself.
“Most of all, I feel proud — proud of how far treatment has come, proud of the clinicians and researchers who made this possible, and proud to stand at a moment where living with severe haemophilia B may finally begin to look very different.”
The Cardiff Haemophilia Centre will act as a “national hub” for the treatment(Image: Cardiff and Vale University Health Board)
Cardiff and Vale University Health Board have announced that the Cardiff Haemophilia Centre, based at the University Hospital of Wales, will be the first place in Wales administering a “revolutionary” gene therapy to those living with the blood clotting disorder. The facility will act as a national hub for the treatment in Wales, with patients from across Wales able to travel to Cardiff to receive their infusion.
Adult patients with severe and moderately severe Haemophilia B will receive Hemgenix (etranacogene dezaparvovec). The one-time intravenous infusion is designed to produce a functioning gene responsible for the clotting protein missing in those suffering with the disorder.
They said the infusion offers a major breakthrough for those living with the condition, offering new hope for sufferers.
Consultant Haematologist Dr Samya Gwen Obaji, Adult Lead and Co-Director of the Cardiff Haemophilia Centre said: “This is a significant step forward in our ability to manage haemophilia B which could be life changing for some of our patients. It is also a testament to the advancement of treatments available to haemophilia patients in the UK and these are exciting times.
“Administering the first dose of Hemgenix in Cardiff was made possible by the support of the NHS Wales Joint Commissioning Committee and the cross-centre collaboration between Cardiff where the patient was treated, and Singleton Hospital, Swansea their local haemophilia centre. We are extremely proud to be able to deliver this therapy in Wales.”
Suzanne Rankin, Chief Executive of Cardiff and Vale University Health Board and Senior Responsible Officer for the Advanced Therapies Wales Programme said: “We proudly acknowledge the outstanding dedication of Haemophilia Centre and Pharmacy team at the University Hospital of Wales, whose expertise and compassion have been central to delivering exceptional patient care.
“Their tireless commitment has ensured that individuals living with haemophilia receive the highest standards of treatment and support.
“We also extend heartfelt thanks to our valued partners, including Advanced Therapies Wales and NHS Wales Joint Commissioning Committee, whose collaboration and support have been vital in strengthening services. Together, these efforts reflect a shared mission to improve lives and advance care.”