As an adult with a blood or bleeding disorder, you may want to learn more about the future of your own treatment. Science and research are always evolving, and new treatments are becoming FDA approved on a regular basis. There are many ongoing clinical trials for future therapies.
This section of Future Therapies covers:
Anti-Tissue Factor Pathway Inhibitor (anti-TFPI)
RNA Interference (RNAi) Therapy
Hemostatic Balance
Your blood contains different elements that help it clot (called pro-coagulants), and others that prevent it from clotting (called anti-coagulants). Coagulation is another word for clotting. One type of imbalance can lead to too much clotting. This is called “thrombosis.” The other type of imbalance can result in too much bleeding or a bleeding disorder. The perfect balance between pro-coagulants and anti-coagulants is known as hemostatic balance. Several new therapies seek to reduce bleeding episodes by restoring this balance. Check out this 3D video on the clotting cascade to learn more about how the blood clots.
Gene Therapy
Gene therapy is a type of treatment where working genes are placed into cells (i.e., liver cells) so that the cells can make the proteins themselves. There are many different approaches to gene therapy, including gene transfer, cell therapy, and gene editing, also known as CRISPR. NBDF has many resources that can help you understand gene therapy. Gene therapy for hemophilia has not been indicated for children under the age of 18 or for women, girls, and people with the potential to menstruate at this time. Gene Therapy: The Basics - FAQs
Anti-Tissue Factor Pathway Inhibitor (anti-TFPI)
Anti-TFPI is a new treatment that will reduce bleeding by decreasing (or putting the brakes) on the system that prevents us from clotting too much. Anti-TFPI restores hemostatic balance by blocking one of the anti-coagulants (clotting) proteins, TFPI, and preventing it from working normally. Because anti-coagulants decrease clotting, interfering with the way they work allows clotting to occur. Anti-TFPI does not rely on replacing a specific clotting protein, like factor VIII (8) or factor IX (9). For that reason, it can be used to prevent bleeding episodes in both hemophilia A and hemophilia B. Anti-TFPI is currently in phase 3 clinical trials.
RNA Interference (RNAi) Therapy
Like anti-TFPI, RNA interference (RNAi) therapy targets an anti-coagulant called antithrombin. It is a new therapy that attempts to restore hemostatic balance by decreasing antithrombin which allows thrombin, a coagulant, to form and prevent bleeding. Because it does not rely on replacing a specific clotting protein, like factor VIII (8) or factor IX (9), it can be used to prevent bleeding episodes in both hemophilia A and hemophilia B. RNAi is currently in phase 3 clinical trials.