I have £2.6m blood and it has ‘cured’ my haemophilia

by | Jun 26, 2024 | Health

3 hours agoBBCA transformational gene therapy that treats the bleeding disorder haemophilia B is being made available on the NHS.Elliott Collins was one of the first to get the treatment when he took part in the trials that proved it worked. He says he “feels cured” and the gene therapy has given him a “new lease of life”.The treatment is one of the world’s most expensive, with an official cost of £2.6m.When you cut yourself, the body senses the injury and activates a series of clotting proteins in the bloodstream. This makes the blood sticky at the site of the wound and then seals it with a clot. But from the day he was born, Elliott was unable to made enough clotting factor IX (nine).Without this crucial clotting component, bleeds are bigger and longer. Bleeds also happen spontaneously inside joints – such as the knee or elbow – causing long-term damage. Elliott, who is 34 and from Colchester, rebelled against his diagnosis as a child by sneakily playing rugby or skateboarding. But any injury risked a damaging bleed, like when he was tackled playing football.”I got studs right across the shin and that came up like a cartoon, like Tom and Jerry,” he said.Elliott needed injections of factor IX twice a week – and more if he was injured – for 29 years.”I would have to think about it all the time,” he said. Elliott CollinsBut he took part in trials of the gene therapy when it was still just an experimental idea in 2019. Since then, he has not needed any factor IX injections and says it has transformed his life.He told me: “I don’t think you can go through something like that for 29 years, for it to completely disappear and not change someone mentally and physically.”I feel cured.”He knew it was working when he clattered his knee into a cupboard. He whacked it hard enough to balloon up and need a factor IX injection, but instead he watched as only a small mark appeared. “That solidified [the realisation] this is real, this is working and I can relax a bit.”Tests showed that levels of factor IX in Elliott’s blood had gone from nothing to 60% of normal. How it worksGetty ImagesElliot was born with a mutation in his DNA – his genetic code – that meant his body’s instructions for making factor IX were faulty. So doctors gave him engineered viruses that contained copies of the fully functional factor IX instructions. The viruses act like a fleet of microscopic postmen, delivering those blueprints to the liver.The organ is then able to manufacture the clotting protein. It was a one-off infusion, lasting about an hour, that Elliott is benefiting from five years later. Not everybody will have as …

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[mwai_chat context=”Let’s have a discussion about this article:nn3 hours agoBBCA transformational gene therapy that treats the bleeding disorder haemophilia B is being made available on the NHS.Elliott Collins was one of the first to get the treatment when he took part in the trials that proved it worked. He says he “feels cured” and the gene therapy has given him a “new lease of life”.The treatment is one of the world’s most expensive, with an official cost of £2.6m.When you cut yourself, the body senses the injury and activates a series of clotting proteins in the bloodstream. This makes the blood sticky at the site of the wound and then seals it with a clot. But from the day he was born, Elliott was unable to made enough clotting factor IX (nine).Without this crucial clotting component, bleeds are bigger and longer. Bleeds also happen spontaneously inside joints – such as the knee or elbow – causing long-term damage. Elliott, who is 34 and from Colchester, rebelled against his diagnosis as a child by sneakily playing rugby or skateboarding. But any injury risked a damaging bleed, like when he was tackled playing football.”I got studs right across the shin and that came up like a cartoon, like Tom and Jerry,” he said.Elliott needed injections of factor IX twice a week – and more if he was injured – for 29 years.”I would have to think about it all the time,” he said. Elliott CollinsBut he took part in trials of the gene therapy when it was still just an experimental idea in 2019. Since then, he has not needed any factor IX injections and says it has transformed his life.He told me: “I don’t think you can go through something like that for 29 years, for it to completely disappear and not change someone mentally and physically.”I feel cured.”He knew it was working when he clattered his knee into a cupboard. He whacked it hard enough to balloon up and need a factor IX injection, but instead he watched as only a small mark appeared. “That solidified [the realisation] this is real, this is working and I can relax a bit.”Tests showed that levels of factor IX in Elliott’s blood had gone from nothing to 60% of normal. How it worksGetty ImagesElliot was born with a mutation in his DNA – his genetic code – that meant his body’s instructions for making factor IX were faulty. So doctors gave him engineered viruses that contained copies of the fully functional factor IX instructions. The viruses act like a fleet of microscopic postmen, delivering those blueprints to the liver.The organ is then able to manufacture the clotting protein. It was a one-off infusion, lasting about an hour, that Elliott is benefiting from five years later. Not everybody will have as …nnDiscussion:nn” ai_name=”RocketNews AI: ” start_sentence=”Can I tell you more about this article?” text_input_placeholder=”Type ‘Yes'”]
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