Haemophiliacs cured with a single injection

Gene therapy frees some from need for weekly jabs

A SINGLE injection of gene therapy could transform the lives of haemophilia patients by effectively curing them.

The treatment dramatically cuts the risk of bleeding in those with the rare condition haemophilia B, experts said.

Researchers found that a single injection of the therapy, called FLT180a, removed the need for sufferers to inject themselves weekly with clotting factors.

Normally when someone cuts themselves, clotting factors mix with blood cells called platelets to make the bleeding stop.

But haemophilia affects the blood’s ability to clot so leaves patients at risk of heavy bleeding. It is usually inherited and mostly affects men.

Most sufferers, around 85 per cent, have haemophilia A, caused by a lack of the blood clotting factor VIII while haemophilia B is caused by a deficiency of clotting protein IX.

Those with haemophilia B need to inject themselves regularly to counter the lack of the protein but can suffer damage and pain caused by internal bleeding into their muscles and joints. In a 26-week trial, researchers found a single treatment with FLT180a corrected the genetic fault and had long-lasting gains.

It led to sustained production of the protein from the liver in nine out of ten of those with severe or moderately severe haemophilia.

Lead research author Professor Pratima Chowdary, of University College London, said: ‘Removing the need for patients to regularly inject themselves with the missing protein is an important step in improving their quality of life.’

A long-term follow-up study will monitor the participants, she added. Professor Amit Nathwani, of UCL and who co-authored the study, added: ‘Gene therapy is still a young field that pushes the boundaries of science for people with severe genetic diseases.’

He said the trial adds to ‘the growing body of evidence that gene therapy has the potential to free patients from the challenges of having to adhere to lifelong therapy or could provide treatment where none exists today’.

The study published in the New England Journal of Medicine was led by UCL, the Royal Free Hospital in London and biotechnology company Freeline Therapeutics, which was co-founded by Professor Nathwani.