Gene therapy is rapidly shaping up to be the next stage in the evolution of treatment for a wide array of conditions. And it makes sense: rather than treat the symptoms of the disease, gene therapy is all about going straight to the source and correcting the faulty genes themselves.
While the approach is still very much in its infancy, more forward-thinking companies are looking at how they can treat diseases on a genetic level. You can add to that list of innovators Shire Pharmaceuticals, who are looking at how to use gene therapy to treat hemophilia A and hemophilia B.
The venerable global company already has a portfolio of factor-replacement products, but in July they presented findings from some of their recent hemophilia studies at the 26th Biennial International Society on Thrombosis and Haemostasis Congress in Berlin.
The primary goal of these studies was to go beyond just replacing factor: It was to integrate a new gene that would encourage higher levels of Factor VIII and Factor IX production to boost the body’s natural protection against bleeds. The challenge with introducing anything that encourages cell growth is the risk of growing too much of a certain type of cell, which raises the risk for cancer. But in a Factor VIII study, researchers were able to boost the production of factor in mice without also growing the cells that could lead to liver cancer. Likewise, a Factor IX trial in mice boosted factor production in mice by as much as five times without triggering an adverse immune system response.
As is always the case in science, these results are not the end of the story, only the beginning. The results seen in mice need to be duplicated in humans, and more observation is needed to see how it holds up in the long-term. But in terms of changing the way patients are impacted by bleeds on a daily basis, it could be the start of a bold new age of living with hemophilia: An age where the emphasis is on “living.”
