A new gene therapy, developed at UCL, could be the first step in finding a cure for haemophilia.
A single dose, given to people with severe haemophilia A, resulted in an increase of the clotting factor (exogenous factor VIII) and fewer bleeding events.
Haemophilia A is the most common type of haemophilia, affecting 2000 people in the UK. Patients with the disorder are born with a genetic defect that means they are unable to produce factor VIII that is needed to stop bleeding.
Of the 13 patients given higher doses, all are off their haemophilia medication a year later and eleven are producing near- normal levels of factor VIII.
Professor John Pasi, Haemophilia Centre Director at Barts Health NHS Trust, said: "We have seen mind-blowing results.”
He went on to explain “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."
UCL licensed the genetically engineered virus to Biomarin Pharmaceutical and the trial took place at the NIHR Imperial Clinical Research Facility and four other sites across the UK.
Dr H Marijke van den Berg, vice president of the World Federation of Haemophilia hails the trial as children born with the devastating disease could benefit from a life without bleeding.
Further studies for the gene therapy will now be carried out around the world. Including patients from USA, Europe, Africa and South America.
