By: Sara Li
Hemophilia is a rare bleeding disorder that is usually inherited, making being passed down through the family. Now, doctors in the UK say they have a therapy treatment to cure one of the two types. Medical teams even say that with it, most adults with Haemophilia could be cured in the next three years.
People with Haemophilia have trouble with blood clotting, meaning they find it hard to stop when bleeding. This is due to platelets, which help your blood form into a clot or scab. But people with Haemophilia don’t have many of these factors, resulting in them bleeding longer than usual. This condition is also more commonly found in males. Symptoms can range from mild to severe and mainly consist of nosebleeds that take a long time to stop, bleeding from wounds that last a long time, bleeding gums, skin that bruises easily, and pain and stiffness around joints, such as elbows, because of internal bleeding.
There are two main types of this disorder. They are called Haemophilia A, and Haemophilia B. Haemophilia A is when there is a lack or total absence of the clotting factor VIII (eight). On the other hand, Haemophilia B is when there is a lack or complete absence of clotting factor IX (nine). This treatment solves the problem of their blood not clotting for people with Haemophilia B. To use this, people must inject themselves regularly to make up for the clotting factor in their blood.
The research was carried out by the University College London (UCL), the Royal Free Hospital in London, and the biotechnology company Freeline Therapeutics.
They found that with just one injection of the gene therapy FLT180a, nine out of
ten patients no longer needed their clotting factor injections. Though scientists are still trying to find out how long this treatment lasts and whether it could also be offered to children with Haemophilia, this is already a great achievement that could benefit many people’s health in the near future.
Prof Chowdary, who took part in the study, said, “We’re very excited by the results,” and added that curing Haemophilia could “be a reality for the majority of the adults in the next one to three years.” Clive Smith, chairman of the Haemophilia Society charity, said, “This initial data is promising, but we continue to monitor gene therapy trials closely and cautiously, as with all new treatments.” He also added that this new treatment needs to be made available “if they are shown to be safe and effective.”
Source: Haemophilia: What is it? What is the new treatment? https://www.bbc.co.uk/newsround/62254020