‘Transformational’ Therapy Seems to Be a Cure For Hemophilia

A transformational therapy is changing the lives of patients with hemophilia B.

Hemophilia is a mostly inherited genetic disorder that impairs the body’s ability to make blood clots, which naturally slows bleeding.

British doctors are giving patients a dead virus that’s been re-engineered to produce a protein known as factor IX, which is the vital missing component in hemophiliacs.

Without it, they’re liable to bleed continuously from wounds—the most dangerous of which are internal as they can’t bandaged. This means many children and adults alike are “wrapped up in cotton wool” as the Brits say, for their whole lives.

Elliot Mason was recently part of a trial group for the treatment, which he received as a one-off through a slow, one hour IV drip. It took his naturally-made levels of factor IX from around 1%, up to those of a non-hemophiliac.

Mason was one of ten patients who received the treatment, nine of whom began producing their own factor IX such that no exogenous factor IX injections, a common treatment, were necessary.

“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,” Mason told the BBC.

Many questions about the future of the treatment, such as how much it will cost and at what age it can become available, remain unanswered.

“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,” stated Clive Smith, chairman of the Hemophilia Society, UK.