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Hemophilia A, a rare medical condition that prevents blood from clotting properly, may soon have a new therapy to contend with, courtesy of a team led by hematologist Barbara Konkle of Bloodworks Northwest. A new fusion protein based on factor VIII has shown promising results in a small-scale clinical trial and has some researchers hopeful that this novel therapy could make life easier for hemophilia A patients.
People with hemophilia A are not able to produce the correct form of factor VIII, a protein that is essential to blood clotting, and as a result can experience life-threatening bleeding episodes, amongst other symptoms. Currently, hemophilia A patients must infuse themselves with synthetic factor VIII multiple times every week in order to prevent bleeding. Once in the body, factor VIII is protected from disintegration by another protein known as von Willebrand factor.
However, VWF also limits the biological half-life of factor VIII, which is why patients must do infusions so frequently. The novel fusion protein, BIVV001, combines factor VIII with a small section of VWF, resulting in a clotting factor that boasts both stability and a substantially longer half-life. If this new therapy is successful in large-scale clinical trials, it could greatly reduce how often hemophilia patients need to do infusions. You can read more about it at Science magazine.