Hemophilia and other blood disorders

waltky · Apr 9, 2016

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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waltky · May 18, 2017

Big step forward...

Human blood stem cells grown in the lab for the first time
17 May 2017 - The stem cells that produce our blood have been created in the lab for the first time. These could one day be used to treat people who have blood diseases and leukaemia with their own cells, rather than bone marrow transplants from a donor. They could also be used to create blood for transfusions.

“This is a very big deal,” says Carolina Guibentif at the University of Cambridge, who was not involved in the research. “If you can develop [these cells] in the lab in a safe way and in high enough numbers, you wouldn’t be dependent on donors.” In a healthy adult, blood stem cells are found in bone marrow, where they replenish the supply of red and white blood cells and platelets. “They are sort of master cells,” says George Daley at Harvard Medical School.

When these cells don’t work properly, they fail to maintain an adequate supply of blood cells. As a result, not enough oxygen reaches the body’s tissues. This can cause serious disease if organs such as the heart are affected. Blood stem cells can also be wiped out by chemotherapy for leukaemia and other cancers. People with these disorders tend to be treated with bone marrow – complete with blood stem cells – from a healthy donor. The difficulty is finding a match. There is a one in four chance of achieving this from a healthy sibling, but the odds are slashed to one in a million if a stranger needs to be found, says Daley.

Making cells

In an attempt to create blood stem cells in the lab, Daley and his colleagues started with human pluripotent stem cells – which have the potential to form almost any other type of body cell. The team then searched for chemicals that might encourage these to become blood stem cells. After studying the genes involved in blood production, the researchers identified proteins that control these genes and applied them to their stem cells.

Potential for a new supply line

They tested many combinations of the proteins, and found five that worked together to encourage their stem cells to become blood stem cells. When they put these into mice, they went on to produce new red and white blood cells and platelets. “It’s very cool,” says Daley. “We’re very excited about the results.” A separate team has achieved the same feat with stem cells taken from adult mice. Raphael Lis at Weill Cornell Medical College in New York and his colleagues started with cells taken from the walls of the animals’ lungs, based on the idea that similar cells in an embryo eventually form the body’s first blood stem cells. The team identified a set of four factors that could encourage these lung stem cells to make them.

Big step forward

Both sets of results represent a “breakthrough”, says Guibentif. “This is something people have been trying to achieve for a long time,” she says. By working with adult mouse epithelial cells, Lis and his team show that the feat could potentially be achieved with cells taken from an adult person. Daley’s team used human stem cells that could in theory be made from skin cells, bolstering the prospect that lab-made human blood could be next. The lab-made stem cells are not quite ready to be used in people just yet, says Daley. Although all of his mice were healthy throughout the experiments, there is a risk that the cells could mutate and cause cancer. And the cells are not quite as efficient at making blood as those found in the body.

But once Daley and his team have honed their procedure, they might be able to make platelets and red blood cells for hospital use. These cell types don’t have a nucleus, so are unable to divide and potentially cause cancer. He hopes this procedure could be used within the next couple of years. Eventually, Daley hopes his cells could be used to create whole blood suitable for transfusions. Not only would such a supply be more reliable than that from donors, but it would also be free of disease. “When new pathogens like Zika pop up, you have to make sure that blood is safe,” says Daley. “We’d be able to have more quality control.”

https://www.newscientist.com/articl...em-cells-grown-in-the-lab-for-the-first-time/
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Just_a_Citizen · May 18, 2017

If I ever get off my arse & use my PC here, I'll get more involved w/ a convo, but this interests me a lot. Glad to find the thread.

waltky · Dec 15, 2017

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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Brett Nortje · Feb 18, 2018

To produce blood platelets we need to understand what they are. They make up part of the blood and when they get exposed to air they start to dry, yes? This means that, since they dry, they will form scabs. The plates are like little scabs blocks that manifest to be dry once they happen upon the air, which dries them.

The problem here is making the blood dry quickly, yes? This means we merely need to observe that hot dry air will dry them quicker, so maybe a hot hair iron would work, a hair dryer? This would apply dry air, without pain, mere discomfort, tot he harmed area, and dry the blood quickly.

Now, how do we apply that to a car accident? Ambulances should carry hair dryers to apply to really nasty gashes, as they will not interfere with wounds, and, sill help stop bleeding quickly. While not ideal, it will help a lot.

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Hemophilia and other blood disorders

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