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Thread: Hemophilia and other blood disorders

  1. #1
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    waltky's Avatar Senior Member
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    Lightbulb Hemophilia and other blood disorders

    Breakthrough in blood platelet manufacturing...

    Huge leap to mass produced platelets
    Fri, 08 Apr 2016 - Scientists have made a significant leap towards mass producing platelets - the part of the blood that forms clots.
    The NHS and University of Cambridge team have discovered how to grow the body's platelet factories in the laboratory. It could provide a new source of platelets to stop heavy bleeding, for example after a car crash. But the researchers need to make the process more efficient before starting trials. If you donate blood, then it is separated out into red blood cells, plasma and platelets so patients are given only the component they need.

    Platelets are needed after trauma, surgery, leukaemia therapy and in some blood disorders like haemophilia. "We're totally dependent on blood donation to produce those platelets," said Dr Cedric Ghevaert, a consultant haematologist. His team has been trying to grow megakaryocytes - the platelet mother cells that live in your bone marrow and manufacture the clotting platelets. Their breakthrough, reported in the journal Nature Communications, was the discovery of a set of chemical switches needed to create megakaryocytes in the lab.


    Dr Ghevaert described their results as a "major step forward" and told the BBC News website that "the next big step is to get enough platelets out of each megakaryocyte". The lab-made cells produce around 10 platelets each. But each one functioning normally in the bone marrow would produce up to 2,000. It is hoped that recreating the same conditions as in the bone marrow could make the cells more effective. If the researchers are successful, then lab-grown platelets could be more useful than ones collected in a blood donation.

    Dr Ghevaert added: "We can modify the platelets so they can trigger the clotting even better which would have huge advantages indeed for patients who have had a crash or a bleed or even in soldiers who have been injured." It could also allow doctors to have stockpiles customised to different patients. Platelets come in different forms just as red blood cells come in A, B, O and AB. And some platelet types, particularly those common in black and Asian ethnic groups, are relatively rare.

    http://www.bbc.co.uk/news/health-35977200

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    Quote Originally Posted by waltky View Post
    Breakthrough in blood platelet manufacturing...

    Huge leap to mass produced platelets
    Fri, 08 Apr 2016 - Scientists have made a significant leap towards mass producing platelets - the part of the blood that forms clots.
    Thanks for posting that. I have a hemophiliac coworker who may one day benefit from that research.
    In quoting my post, you affirm and agree that you have not been goaded, provoked, emotionally manipulated or otherwise coerced into responding.



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    Lightbulb

    Mind-blowing Hemophilia breakthrough...

    Haemophilia A trial results 'mind-blowing'
    14 December 2017 - British doctors say they have achieved "mind-blowing" results in an attempt to rid people of haemophilia A.
    Patients are born with a genetic defect that means they do not produce a protein needed to stop bleeding. Thirteen patients given the gene therapy at Barts Health NHS Trust are now off treatment with 11 producing near-normal levels of the protein. Jake Omer, 29 from Billericay, Essex, was on the trial and says he feels like he has a new body. Like 2,000 other people in the UK, his body could not make clotting factor VIII. A minor injury used to cause severe bleeding. He remembers losing two front teeth as a child and bleeding for days afterwards. Even the impact of walking would lead to bleeding in his joints and eventually cause arthritis. Jake has needed at least three injections of factor VIII a week for most of his life.


    Jake Omer: 'Like a new body'

    But in February 2016, he had a single infusion of gene therapy. Jake told the BBC: "I feel like a new person now - I feel like a well-oiled robot. "I feel I can do a lot more. I feel my body allows me to do more. "I don't think I would have been able to walk 500m without my joints flaring up, whereas now I think sort of two, three, four-mile walk - I could quite easily achieve that." The first time he knew it had worked was four months after the therapy when he dropped a gym weight and bashed his elbow. He started to panic, but after icing the injury that evening, everything was normal the next day.

    'Transformational'

    The therapy is a genetically engineered virus. It contains the instructions for factor VIII that Jake was born without. The virus is used like a postman to deliver the genetic instructions to the liver, which then starts producing factor VIII. In the first trials, low doses of gene therapy had no effect. Of the 13 patients given higher doses, all are off their haemophilia medication a year on and 11 are producing near-normal levels of factor VIII. Prof John Pasi, who led the trials at Barts and Queen Mary University of London, said: "This is huge. "It's ground-breaking because the option to think about normalising levels in patients with severe haemophilia is absolutely mind-blowing. "To offer people the potential of a normal life when they've had to inject themselves with factor VIII every other day to prevent bleeding is transformational." An analysis of the first nine patients on the trial was published in the New England Journal of Medicine.

    Larger trials are now imminent to see if the therapy can truly transform the lives of patients. It is also uncertain how long the gene therapy will be effective. Liz Carroll, the chief executive of The Haemophilia Society, said: "Gene therapy is a potentially game-changing treatment. "Despite world-leading treatment standards in the UK many still suffer painful bleeds leading to chronic joint damage." However, she warned there was a wide variation in who responded to therapy, which still needed to be explained. Gene therapies are likely to be spectacularly expensive. However, the current cost of regular factor VIII injections is about £100,000 a patient per year for life. Jake says the therapy should help him live a full life with his family: "It's going to allow me as my boys grow up to be more active with them, to kick footballs about, to climb trees, to hopefully run around the park with them, not be someone who has to worry."

    http://www.bbc.com/news/health-42337396

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    Took the old lady to the Quest diagnostics center, for a blood and Urine specimen.

    They used vacutainers, took about 5, send the blood to a lab 100 miles away. How the hell does it not clot?
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