Patients with hemophilia B can breathe a little more now that there is a transformational therapy which is seriously changing their lives.
Hemophilia happens to be a mostly inherited genetic disorder or disease where some people’s body lack the ability to make blood clots, which is important to naturally slowing down bleeding, which can be fatal in some situations.
This new treatment is being done in the UK, with British doctors giving their patients a dead virus which has been re-engineered to produce a protein called factor IX, which is considered the vital missing component for those diagnosed with hemophilia B.
When people lack the protein factor IX, they have a tendency to bleed continuously from wounds, and it can be incredibly dangerous if the wound is internal, which means that it cannot be bandaged up. As a result, a large number of both adults and children are – as the Brits say – “wrapped up in cotton wool” for their entire lives.
One such person is Elliot Mason. A diagnosed hemophiliac, he was part of the trial group that underwent this new treatment where he was given a one-off through a slow, one hour IV drip. This brought his naturally-made levels of factor IX from around 1% and brought them up to those of a non-hemophiliac.
Mason told BBC News in an interview that as a child growing up, he was always “anxious of getting hurt,” and as a result, his teachers “wrapped me up in bubble wrap.” In fact, he said that it was his dream to play rugby as a child, but he was never allowed due to his disease.
Mason went on to say, “I didn’t like the fact that I was different and not able to do things.”
So when he was given the chance to be part of this trial, he jumped at the chance. Mason was one in ten patients that were given this treatment, while nine of them began producing their very own factor IX as if they had no exogenous factor IX injections, which is a common treatment, were necessary.
Mason also told BBC, “I’ve not had any treatment since I had my therapy, it’s all a miracle really, well it’s science, but it feels quite miraculous to me.”
Although there are a number of question about the possible future of this brand new treatment, like how much it’ll cost and at what age it can become available for everyone, they have yet to be answered.
According to chairman of the Hemophilia Society in the UK, Clive Smith, “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 these innovative treatments available.”
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