Drug regulators have approved a new treatment for hemophilia, which could cure up to 2,000 Britons.
Haemophilia, a disorder that affects the blood’s ability to clot, is presently treated with regular injections that replace the patient’s blood clotting “factor.”
A clinical trial revealed, however, that a single infusion of the gene therapy Hemgenix significantly enhanced the blood clotting abilities of individuals with hemophilia B.
In addition, they have retained this ability years after treatment, indicating that the effects may be permanent.
The decision by the Medicines and Healthcare Products Regulatory Agency (MHRA) to approve its use signified a “revolution” in hemophilia treatment, according to a leading blood doctor.
Consultant hematologist Dr. Rashid Kazmi, who headed the Hemgenix trial in the United Kingdom, states that even with injections, hemophiliacs are susceptible to “trauma-related bleeding” at present.
These can be quite minor, such as a heavy blow or a sprained joint. But they can lead to severe complications, such as chronic pain and, in rare cases, paralysis.
University Hospital Southampton’s Dr. Kazmi stated, “We do not yet know how long the effects of the new treatment will last, but things are looking promising for hemophilia B.”
Hemgenix functions by smuggling a gene into blood cells, allowing them to produce the absent clotting factor. They accomplish this by entering cells using an innocuous virus as a Trojan horse.
Gene therapy reduced bleeding episodes by two-thirds in 54 haemophiliacs.
In addition, 96% no longer required their regular injections. Two years of monitoring have revealed no decline in coagulation ability.
The National Institute for Health and Care Excellence decides whether it will be made available on the NHS. (NICE). It will presumably make a decision later this year.
