Haemophilia breakthrough cure by Royal London Hospital to missing blood-clotting protein
PUBLISHED: 07:00 19 December 2017 | UPDATED: 11:41 19 December 2017
Barts NHS Trust
A father-of-two diagnosed with haemophilia when he was two-years-old has been given the best Christmas present ever by medical researchers in Whitechapel who appear to have found a breakthrough cure.
Haemophilia is one step closer to being cured following results of a groundbreaking gene therapy trial at the Royal London Hospital in Whitechapel.
One of the test patients was Jake Omer, 29, who has had to have injections three times a week nearly all his life to prevent unstoppable bleeding.
His condition had left him with arthritis before he was treated with the gene therapy at the Royal London.
“The therapy has changed my life,” Jake said. “I struggled at 23 to run for a bus and used to worry how far I could walk with the arthritis once I would turn 40.
“But at 29 I’m walking two miles a day which I just couldn’t manage before the gene therapy.”
Jake, who is married with a son aged three and a five-week-old baby, feared playing with his children in the park for fear of being injured. Now he is looking forward to football and climbing trees well into his children’s teen years.
The test patients given a single with a gene therapy treatment are showing normal levels of the previously missing Factor VIII protein and effectively being cured, Barts Health trust and Queen Mary University clinical researchers found.
They showed improved blood clotting protein levels, with 85 per cent achieving normal or near-normal levels even months after treatment.
Royal London’s Haemophilia director Prof John Pasi said: “These are mind-blowing results which have far exceeded our expectations.
“We thought it would be a huge achievement to show just five per cent improvement when we started out, not 85 per cent.
“The trials have the potential to transform healthcare with a single treatment for those who must inject every other day. It is incredibly exciting.”
The Royal London and Queen Mary team are now running further tests and going global to include haemophiliacs in Europe, the USA, Africa and South America.
The breakthrough brought high praise from the medical world. The Royal College of Pathologists president Prof Jo Martin said: “We congratulate Prof Pasi and his team for creating a simple but transformational treatment.”
The trials are the first-ever successful gene therapy for the severe Haemophilia A condition. Those with the hereditary condition have virtually none of the protein essential for blood to clot, risking excessive bleeding even from the slightest injury. Spontaneous internal bleeding it causes can be fatal.
Those taking part in the Royal London trials were tested up to 19 months later and showed that 11 out of 13 now had normal or near-normal levels of Factor VIII and all 13 have been able to stop regular weekly injections.