‘Mind-blowing success’ in gene therapy that can halt haemophilia
A DRAMATIC breakthrough in gene therapy is offering hope of a cure for the life-threatening blood disease haemophilia.
Scientists have described the success of trials as ‘mind-blowing’ and far exceeding their expectations.
Those with the hereditary condition, which mainly affects men, have virtually none of the protein factor VIII which is needed for blood to clot.
In the trials led by Barts Health NHS Trust doctors injected 13 patients with a copy of a missing gene which allows cells to produce the missing factor.
All 13 patients were able to stop regular treatment for the condition.
Of these, 11 now have ‘normal or nearly normal’ levels of the missing protein factor after having their progress followed for 19 months. Professor John Pasi, Haemophilia Centre director at the trust, said: ‘This is huge. 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 5 per cent improvement. So to 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 those who at the moment must inject themselves as often as every other day.’
About 2,000 people in the UK have the disorder, which can cause internal bleeding and is usually inherited. Further problems include joint damage and arthritis,
Liz Carroll, chief executive of The Haemophilia Society, said: ‘Gene therapy is a potentially game-changing treatment.
‘Despite world-leading treat- ment standards in the UK many still suffer painful bleeds leading to chronic joint damage.’
Jake Omer, 29, from Billericay, Essex, was diagnosed with haemophilia when he was two years old and has had frequent injec- tions of factor VIII to prevent bleeds ever since.
Just the impact of walking could lead to bleeding in his joints. He needed at least three injections of factor VIII a week for most of his life. But now he feels like he has a new body.
The father-of-two noticed an improvement four months after treatment. He told the BBC: ‘I feel like a new person now, I feel like a well- oiled robot. I feel I can do a lot more. I feel my body allows me to do more.
‘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. I don’t think I would have been able to walk 500 meters without my joints flaring up, whereas now I think of doing a two, three, four-mile walk – I could quite easily achieve that.’
The therapy uses a genetically engineered virus which contains instructions for the Factor VIII that Jake was born without and delivers the genetic instructions to his liver.
These are then built into the DNA in the liver, which then starts producing factor VIII.
The team will hold more tests to include volunteers in the USA, Africa and South America.
‘Game-changing treatment’