Therapy cures haemophilia B

FE Team | Published: July 21, 2022 21:31:59

Elliott Mason

LONDON, July 21 (BBC): A "transformational" therapy has effectively cured people with the bleeding disorder haemophilia B, say British doctors.
The treatment corrects a genetic defect that leaves people's blood struggling to clot and stop bleeding.
s, who was part of the trial which tested the therapy, says his life now feels "completely normal".
The medical team says the majority of adults with haemophilia could be cured in the next three years.
From the day Elliott was born, he was unable to make enough of a crucial protein called clotting factor IX. When you cut yourself and it scabs over, clotting factor IX is one of the proteins that stops the bleeding.
It meant Elliott grew up "anxious of getting hurt", and teachers "wrapped me up in bubble wrap". The sport he wanted to play - rugby - was completely off limits.
"I didn't like the fact that I was different and not able to do things," he says.
At one point, Elliott was having injections of factor IX every other day in order to prevent a deadly bleed.
But he managed to stay healthy, unlike many others with haemophilia who face severe damage to their joints from the bleeding.
"We have a lot of young patients in excruciating agony and there's nothing we can do to reverse the joint damage," says Prof Pratima Chowdary, from the Royal Free Hospital and University College London.
Elliot was given an engineered virus that was filled with the instructions for manufacturing the missing factor IX.
The virus acts like a microscopic postman that delivers the blueprints to the liver, which then starts producing the clotting protein.
It was a one-off infusion that took about an hour to drip into Elliott's body.
He recalls being "astonished" to see the amount of factor IX in his blood go from only 1% of normal levels to normal.