14 December 2017

The first successful gene therapy trial for haemophilia A has been reported by a team of clinical researchers from Barts Health NHS Trust and Queen Mary University of London. Now a potential cure for the monogenic disorder, the successful implementation of the procedure marks a milestone not only for the haemophilia community, but also for medicine as a whole.

The treatment, which involves gene therapy to encourage the production of missing protein factor VIII, was trialled in thirteen patients across England.

The team showed that a single infusion of the gene therapy, using a viral vector with a functional copy of the gene responsible for producing the essential blood clotting protein, led to improved levels of the protein. Out of the 13 patients treated, 11 achieved normal or near-normal Factor VIII levels over the following 19 months.

In addition, ten of the patients had no bleeding episodes requiring Factor VIII treatment from four weeks after infusion through the last follow-up visit.

Professor Pasi said: "The clinical data to date for this investigational gene therapy exceeded our expectations, in terms of increasing Factor VIII levels and reducing the annualised bleed rate.

“We are researching whether it may be possible for haemophilia A patients to reduce or eliminate Factor VIII treatment over an extended timeline."


Link: http://www.nejm.org/doi/full/10.1056/NEJMoa1708483#t=article

Source: Rangarajan et al. (2017). AAV5–Factor VIII Gene Transfer in Severe Hemophilia A. New England Journal of Medicine. doi:10.1056/nejmoa1708483


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