Hemophilia B Updates: According to British experts, a “transformational” medication has successfully treated patients with the bleeding condition haemophilia B.
The procedure corrects a genetic flaw that makes it difficult for people’s blood to clot and stop bleeding.Elliott Mason, a participant in the therapy experiment, claims that his life now feels “totally normal.”According to specialists, in the coming years, developments like this could completely transform the lives of adults with haemophilia.
From the moment Elliott was born, he was unable to produce enough of clotting factor IX, a necessary protein. One of the proteins that stops the bleeding when you cut yourself and it heals over is clotting factor IX.Elliott was told to “wrap myself in bubble wrap” by his teachers since he grew up “anxious about getting wounded.” He was not allowed to participate in the sport he wanted to play, rugby.I didn’t like being different and unable to perform some tasks, the man claims.Elliott once required factor IX injections every other day to stop a potentially catastrophic haemorrhage. But unlike many other haemophiliacs who experience substantial joint damage from the bleeding, he was able to keep his health. According to Prof. Pratima Chowdary of the Royal Free Hospital and University College London, there is nothing we can do to reverse the joint injury. She explains, “We have a lot of young people who are in terrible pain.”
A virus that had been specially created and loaded with instructions for producing the missing factor IX was given to Elliot.The liver receives the blueprints from the virus like a tiny mailman, which causes the liver to begin manufacturing the clotting protein.It was a onetime infusion that took Elliott’s body about an hour to absorb.When the amount of factor IX in his blood increased from just 1% of normal levels to normal, he recalled being “astonished” by the change.Nine out of ten patients receiving the therapy no longer required their clotting factor IX injections, according to the results, which were published in the New England Journal of Medicine.Elliott, who now resides in London, adds, “I’ve not had any treatment since I had my therapy. It’s all a miracle really, well, it’s science, but it feels rather wonderful to me.”My life is entirely normal; there is nothing that requires me to pause and consider how my haemophilia can effect this.”For Elliott, those activities include motorcycling and skiing.
The results have us quite enthusiastic, said Prof. Chowdary.About a year following the therapy, according to her, there was a“transformational impact,” when patients suddenly realised they didn’t need to worry about their haemophilia at all.This trial is merely the most recent in a string of developments in the management of haemophiliacs A and B.Prof. Chowdary informed me that she is currently “searching for my next employment,” as the majority of adults with haemophilia “will be cured in the next one to three years.”However, further inquiries need to be addressed:
• What will the price of gene therapy be? How much will the treatment cost? Currently, clotting factor injections can cost between £150,000 and £200,000 per patient per year. Studies predict it will take at least ten years, but nobody is certain.
• How soon can therapy be administered? The liver is still developing before the age of 12, but researchers hope it will be an option after that.The Haemophilia Society’s chairman, Clive Smith, stated: “This preliminary result is encouraging, but as with all novel therapies, we continue to watch gene therapy trials carefully.
“If they are shown to be safe and effective, NICE [National Institute for Health and Care Excellence] and the NHS must work together to make them available.” “NICE [National Institute for Health and Care Excellence] and the NHS must work together to make these novel medicines available.”