Pioneering trial could cure haemophilia

30 January 2018

The haematology team at University Hospitals Birmingham NHS Foundation Trust (UHB), in partnership with colleagues at the NIHR Wellcome Trust Clinical Research Facility (CRF), has recently worked on a national study that could potentially cure haemophilia.

The trial, which involved six Trusts nationally, saw 13 patients injected with a copy of the protein factor VIII, which is needed for blood to clot.

Haemophilia patients have virtually none of this missing gene, but the copy allowed cells to produce the missing factor, allowing all 13 patients to stop regular treatment.

Obayed Khan, 25, was recruited for the trial, and now has normal levels of the missing protein factor, 19 months on from the injection.

Obayed said: “This trial has been life-changing for me, and I’d really like to thank everyone involved.

“I’m really excited to be part of medical history, and it’s great to think gene therapy could help others with chronic illness.”

Dr Gillian Lowe, Consultant Haematologist, said: “The results of this trial have surpassed our wildest expectations.

“It’s the first treatment option that could potentially cure those with haemophilia, rather than just replace the factors that clot blood.

“The possibility of a single treatment to produce factor VIII could transform care for people with haemophilia, who currently have to inject themselves on a regular basis to prevent prolonged bleeding.

“We are currently setting up the next phase of this study, in partnership with other Trusts.”

It is estimated there are about 2,000 people with haemophilia in the UK.

Though there is currently no cure, injections and other are available for those with haemophilia.

Those with haemophilia can suffer excessive bleeding, even from slight injuries, as their blood is unable to clot effectively, because of the absence of protein factor VIII.

The condition is usually inherited, and most people with the condition are male.

The results have been published, co-authored by leads at the six Trusts, in the New England Journal of Medicine.