First gene therapy against haemophilia, avoids infusions for several years

The first experimental gene therapy in Italy to treat a severe haemophilia A patient has been completed. At the Milan Polyclinic, "the first patient, a woman, was treated in early November, and today, almost 4 weeks after the infusion, she is in good condition and is conducting her life regularly." The therapy, in fact, will allow him to avoid for several years the frequent infusions to which he had been forced so far, and to have a blood coagulation equal to that of anyone else. Haemophilia A is a rare genetic disease that affects 5,000 people in Italy and consists of a defect in the blood that prevents it from clotting. Affected patients undergo bleeding episodes, spontaneous or caused by even trivial traumas, which until now were prevented by infusion even 3 times a week of the factors necessary for the coagulation which alone cannot produce (obtained from the plasma of a donor or synthesized in the laboratory). Gene therapy, using viruses rendered harmless as DNA transporters, can 'correct' a genetic defect. It has already been used to treat very few other pathologies (such as Ada-Scid or 'bubble babies' disease) and now for the first time in Italy on hemophilia. The first patient, explains Flora Peyvandi, head of the clinical trial, "was treated in early November, and today, almost 4 weeks after the infusion, she is in good condition and is conducting her life regularly, without any particular problem". The results "of our clinical studies - adds Silvano Bosari, scientific director of the Polyclinic - have shown that a single infusion of this therapy can allow a patient with severe hemophilia to reach almost normal factor VIII or factor IX levels for long periods of time, even for some years ".