Gene therapy in humans may still be in its infancy, but it’s already making some previously impossible stories a reality. From editing out the symptoms of sickle cell disease to curing two baby girls of an incurable form of leukemia, the future is already here for some.
Now, as reported by two remarkable studies in The New England Journal of Medicine, it appears that gene therapy’s making headways when it comes to hemophilia. After two pioneering experimental approaches, most of the participants in the study experienced normal levels of blood clotting, and many no longer required injections of coagulating elements that they previously needed on regular basis.
The journal, which is naturally cautious when it comes to declaring medical breakthroughs, ran an editorial entitled “A Cure for Hemophilia within Reach.”
Hemophilia doesn’t make the news that often, probably because it’s so rare. Those that have it are almost always male, and it’s usually inherited. Although patient’s symptoms vary in terms of their severity, they all have one thing in common: they aren’t able to clot their blood as usual, which means that they bleed for longer than usual. This can be anything from deeply problematic to fatal.
Currently, there is no cure. The best that can be done is the aforementioned injections, which add clotting factors to the blood. Although normal lives can be lived by those afflicted, they have to be far more cautious than others day-to-day.
The two main types of hemophilia are denoted A and B, although others do exist. The far more commonplace type A, thanks to the unusual nature of the genetics driving the condition, was thought to be far more difficult to treat using gene therapy. Type B, on the other hand, was suspected of being comparatively easier, but by no means no cakewalk.
These studies took on both, and won.