Revolutionary gene therapy could cure thousands of hemophiliacs if a new treatment is given the green light
- One trial found that an infusion of Hemgenix therapy improved blood clotting ability
- Consultant hematologist Dr Rashid Kazmi said it was a ‘revolution’ in treatment
Up to 2,000 Britons could be cured of haemophilia if a new treatment gets the go-ahead from drug regulators.
Hemophilia – a condition that affects the blood’s ability to clot – is currently treated with regular injections, which replace the clotting factor in the patient’s blood.
But one trial found that a single infusion of a gene therapy called Hemgenix vastly improved the blood-clotting ability of people with a type of condition known as hemophilia B.
In addition, they have retained this ability for years after receiving treatment, indicating that the effects may be permanent.
Last night, a leading blood specialist said the Medicines and Healthcare products Regulatory Agency’s (MHRA) decision to approve it for use represented a “revolution” in the treatment of haemophilia.
Hemophilia – a condition that affects the blood’s ability to clot – is currently treated with regular injections, which replace the clotting factor in the patient’s blood
Consultant haematologist Dr Rashid Kazmi, who led the Hemgenix trial in the UK, says hemophiliacs are currently prone to ‘trauma-related bleeding’, even with injections.
These can be very minor — as insignificant as a bad bump or a joint sprain — but can lead to serious problems, including chronic pain and, rarely, paralysis.
Dr Kazmi, from Southampton University Hospital, said: ‘We don’t yet know how long the effects of the new treatment will last – but for haemophilia B it looks promising.’
Hemgenix works by smuggling a gene into blood cells, allowing them to produce the missing clotting factor. They do this by using a harmless virus like a Trojan horse to invade cells.
About 54 hemophiliacs participated in the study and their bleeding rate decreased by an average of two-thirds after gene therapy treatment.
About 96 percent also stopped their usual injections. After two years of follow-up, no reduction in coagulability has been observed.
Whether it becomes available on the NHS is up to the National Institute of Health and Care Excellence (NICE). The decision is likely to come later this year.