Hemophilia is a rare bleeding disorder. Patients with the disease bleed more easily than those without it, and their blood may not clot. As you can imagine, this can be extremely serious if bleeding can’t be controlled. However, patients with hemophilia B may have something to look forward to, if new research is any indication.
Those with hemophilia B lack the clotting factor IX. Scientists have been able to use an inert virus to deliver new factor IX clotting genes into animals, and so far, the treatment has proven to be safe and effective. It seems that this delivery method could provide a long-term solution to hemophilia. Researchers worked in the laboratory with dogs that have naturally occurring hemophilia. They gave the dogs injections of an inert virus (read: harmless) that carried factor IX genes into the dogs’ systems. They saw a big difference! Before the treatment, each of the three dogs experienced an average of five bleeding events each year that required medical treatment. But for the three years following the experimental treatment, each dog experienced none or only one bleeding event each year requiring medical treatment.
If this proves to be safe in humans, this could be a big deal for patients dealing with hemophilia. Human patients often require multiple injections of factor IX, as often as several times a week. But this new delivery system might mean that doctors could deliver factor IX in a long-lasting way. Read more about this research here– hopefully it moves from animal studies into human trials!