Engineered Protein Shows Promise as New Hemophilia Treatment

Researchers at Children’s Hospital of Philadelphia have genetically engineered coagulation factor Xa into a variant which shows promise as a novel treatment for hemophilia and other bleeding disorders. The investigators presented the promising results of their animal study in Nature Biotechnology.

Hemophilia is a group of blood diseases which impairs one’s ability to produce a blood-clotting protein. This leads to bleeding, which can be life-threatening. To treat patients with hemophilia, infusions of blood-clotting proteins are necessary. This can lead to an antibody production which negates the benefits of the treatment.

Factor Xa is a coagulation factor which in its normal shape is not very effective as a therapy. In an attempt to rediscover factor Xa as a treatment, Rodney Camire and his research team decided to custom-design the factor Xa protein to prolong its biological activity and limit its ability to trigger unwanted biochemical reactions. Infused into mice with hemophilia, their engineered factor Xa was successful in reducing blood loss and restoring  blood clotting ability. Now the team wants to move on and test it on other animal models to find out whether this might become a clinical treatment for hemophilia patients who produce antibodies against the first therapy of choice.

Announcement from the Children’s Hospital of Philadelphia: Bio-engineered Protein Shows Promise as New Hemophilia Therapy

Abstract in Nature Biotechnology: A zymogen-like factor Xa variant corrects the coagulation defect in hemophilia

Image credit:: Wellcome Images: Blood clot in a vein …