A new genetic therapy has been developed for the treatment of haemophilia, a blood clotting disorder which can lead to excessive bleeding. The new therapy could be life changing for patients with the illness, as the trial showed that 11 out of 13 patients tested saw their blood clotting levels return to normal after just 19 months.
Haemophilia sufferers have to take regular injections for the incurable condition; otherwise it can cause fatal symptoms including internal bleeding and even death. Missing injections can also lead to reduced quality of life due to the lack of protein factor VIII which is needed for the blood to clot properly.
The trial tested new genetic engineering techniques which could be used to replace defective genes that cause the condition. During the trial, 13 patients were injected with a copy of the missing genes. All the participants in the study were able to stop their normal regular treatment, and 11 saw their level of protein factor VIII reach “normal or nearly normal”
Jake Omer, who has the condition and took part in the trial said “The gene therapy has changed my life. I now have hope for my future. It is incredible to now hope that I can play with my kids, kick a ball around and climb trees well into my kids’ teenage years and beyond. It’s really strange to not have to worry about bleeding or swellings.”
“The first time I noticed a difference was about four months after the treatment when I dropped a weight in the gym, bashing my elbow really badly. I started to panic thinking this is going to be really bad, but after icing it that night I woke up and it looked normal. That was the moment I saw proof and knew that the gene therapy had worked.”
The researchers plan to carry out further tests in the UK, as well as throughout Europe, the US, Africa and South America.Professor John Pasi, Haemophilia Centre director at Barts, commented that “We have seen mind-blowing results which have far exceeded our expectations.
“When we started out we thought it would be a huge achievement to show a five per cent improvement, so to actually be seeing normal or near normal factor levels with dramatic reduction in bleeding is quite simply amazing. We really now have the potential to transform care for people with haemophilia using a single treatment for people who at the moment must inject themselves as often as every other day. It is so exciting.”