Single jab could put a stop to haemophilia

HAEMOPHILIACS could receive a single injection to cure the faulty gene that causes the disease, a study suggests

There are about 6,000 people in the UK with haemophilia, a mostly inherited disorder that generally affects men and impairs the blood’s ability to clot.

People with haemophilia B take weekly injections of the Fix protein, which their body fails to make naturally, in order to prevent small cuts turning into serious bleeding.

But a team of researchers at University College London has created a form of gene therapy that allows a person’s body to generate the clotting protein.

In clinical trials, nine out of ten volunteers given one dose of the FLT180A therapy saw a sustained production of the Fix protein in the liver, meaning they no longer needed weekly jabs.

Five patients had normal levels of the protein, three had low but increased levels, and one patient treated at the highest dose developed a blood clot. The team is confident they can minimise adverse reactions with further research. The patients will be followed for 15 years to monitor their health.

Prof Amit Nathwani, co-founder of Freeline, the firm that makes the treatment, said: “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 findings are published in the New England Journal of Medicine.