Hemophiliac dogs get some good news!
Millions of people around the world suffer from hemophilia, a bleeding disorder that prevents the blood from clotting properly. Often, diseases that are found in humans are also found in animals, and in this study, researchers worked to find a treatment for dogs with naturally occurring hemophilia A.
Patients that suffer from hemophilia lack a coagulation factor (factor VIII) in their blood plasma. One treatment is to replace factor VIII via injection, but many hemophiliacs don’t respond to factor VIII therapy.
So researchers at the Medical College of Wisconsin figured out a way to ‘sneak’ factor VIII into the body. They took cells that would eventually turn into platelets and engineered them to express factor VIII. The cells were put into the dogs and began to make platelets. And when bleeding events started, these platelets did their jobs and dumped their contents at the bleeding site- sending factor VIII right where it was needed!
Before this gene therapy, these Great Pyrenees had approximately five serious bleeding events each year. But after introducing these engineered platelet precursor cells, that number was significantly reduced, and the bleeding events were easily treatable! And 2 1/2 years after the gene therapy, platelets are still expressing factor VIII.
Next step: human clinical trials. Is this an example of humans helping animals… or animals helping humans?