A ground-breaking gene therapy has “functionally” cured three Irish people with haemophilia.
Haemophilia involves inherited blood disorders in which there is a defect in the clotting mechanism of the blood.
The Irish Times reports that traditional treatment with a clotting factor requires weekly injections, while the new gene therapy means there is no need for injections for at least 10 years.
The therapy provides patients with a new “working copy” of the genes for clotting factor protein.
The three Irish patients are among 54 patients around the world who participated in a trial for severe haemophilia and received the gene therapy in a single intravenous dose to the liver last spring at St James’s Hospital.
After living my entire life with severe haemophilia, at the moment I don’t have it
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The patients have been closely monitored throughout the Covid-19 pandemic, with the positive early results of the trial set to be presented at the annual meeting of the American Society of Haematology on Tuesday.
One of the patients and head of the Irish Haemophilia Society, Brian O’Mahony, describes himself as “functionally cured”.
“I’ve had no side-effects and my factor IX level has stayed normal. After living my entire life with severe haemophilia, at the moment I don’t have it,” he said.
About 30 people with haemophilia B will benefit from the new treatment, with new gene therapies also being developed for those with haemophilia A.
In the 1980s, hundreds of Irish people with haemophilia were infected with HIV and hepatitis C are being given contaminated blood products supplied by the State.
