Hemophilia A gene therapy trial results: ‘This is huge’

hemophilia

British doctors say they have achieved “mind-blowing” results in an attempt to rid people of haemophilia A. Patients are born with a genetic defect that means they do not produce a protein needed to stop bleeding. Thirteen patients given the gene therapy at Barts Health NHS Trust are now off treatment with 11 producing near-normal levels of the protein. Jake Omer, 29 from Billericay, Essex, was on the trial and says he feels like he has a new body.

The therapy is a genetically engineered virus. It contains the instructions for factor VIII that Jake was born without. The virus is used like a postman to deliver the genetic instructions to the liver, which then starts producing factor VIII. In the first trials, low doses of gene therapy had no effect. Of the 13 patients given higher doses, all are off their haemophilia medication a year on and 11 are producing near-normal levels of factor VIII.

Prof John Pasi, who led the trials at Barts and Queen Mary University of London, said: “This is huge. “It’s ground-breaking because the option to think about normalising levels in patients with severe haemophilia is absolutely mind-blowing.

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An analysis of the first nine patients on the trial was published in the New England Journal of Medicine.

Read full, original post: Haemophilia A trial results ‘mind-blowing’

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