Today’s report, AAV5–Factor VIII Gene Transfer in Severe Hemophilia A, in the New England Journal of Medicine offers the prospect of people with severe Haemophilia A taking a single injection and achieving clotting factor levels of mild or moderate patients. This could mean huge reductions in use of clotting factor replacement products.
Dan Farthing-Sykes, CEO of Haemophilia Scotland responded saying,
These results are a huge achievement and offer someone born with Haemophilia today the prospect of a life without regular infusions. I am struck by the bravery of all of those who have volunteered to be part of this and similar gene therapy trials. Without their courage the amazing results achieved in studies like this would not be possible. Our thanks and congratulations go to everyone involved in this important work.
Many Haemophilia Scotland members will be excited by the prospect of a step-change in treatment. However, as with all new medicines, they will want to see these results repeated in larger studies and be assured that the treatment is as safe as possible, as well as being free from harmful side effects.
This breakthrough will require clinicians, patients, and Governments to work together to make sure patients are able to benefit fully. We will need to know if the therapy will continue working for the rest of a patient’s life. What levels of standard treatment might still be needed to keep all patients bleed-free or to respond to trauma? Gene therapy might also require new and innovative funding models to make sure that all the patients who might benefit are able to access it.
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