Innovative Therapies for Bleeding Disorders
Over the last few years, new, innovative therapies have been approved to treat bleeding disorders. These treatments are sometimes referred to as “novel” therapies, because they work differently in the body to prevent bleeding than factor replacement therapy. Other ways these treatments differ from factor replacement therapies include fewer treatments (weekly, bi-weekly, monthly dosing) and how the medication is given.
As these new therapies are approved, they may have a limited target treatment audience. For example, they may only be for individuals who are 12 and over. Some may only be for people with inhibitors. As researchers learn more about these products they may become available to more people.
These treatments all work differently in the body and each have different risks. It is important that you speak with your healthcare team to learn in detail what the benefits and risks are so that you and your healthcare provider make the best shared decision on treatment options that are right for you.
Learn more about shared decision making.
What is Hemostatic Balance?
Your blood contains different elements that helps it clot (called pro-coagulants), and others that prevents 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.
Learn More About the Clotting Cascade.
Bispecific Antibody Therapy
Bispecific antibody therapy is a therapy used to treat hemophilia A, to prevent bleeding episodes in people both with and without inhibitors. It is known as a factor VIII(8) mimetic because it mimics, or imitates, the way factor VIII(8) works. It brings together factor IX(9) and factor X (10), which allows the blood to clot. Bispecific antibody therapy is given by an injection under the skin, called a subcutaneous injection (sub Q).
Read the MASAC for You on Bispecific Antibody Therapy.
Gene Therapy
Gene therapy is a way of treating a genetic disease or disorder by providing people with working copies of the gene to correct the disease or disorder. There are different approaches to gene therapy, including gene transfer and gene editing. Current approved gene therapies utilize gene transfer technology.
Currently, gene therapies for Hemophilia A and Hemophilia B work differently in the body and have different results. It is important that you work with your Hemophilia Treatment Center to learn more about gene therapy, to determine if you are eligible, to make certain you understand the risks and benefits, and to ensure you have the information you need to make the best decision for you.
Gene therapy for hemophilia A and gene therapy for hemophilia B have both been approved by the FDA.
Rebalancing Agents
Anti-tissue factor pathway inhibitor (TFPI) or anti-TFPI
Anti-TFPI is a novel treatment that seeks to 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, 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.
siRNA (small interfering RNA)
Similar to anti-TFPI, siRNA therapy targets an anti-coagulant called antithrombin. It is a novel therapy that seeks to restore hemostatic balance by decreasing antithrombin which allows the formation of enough thrombin, a coagulant, to 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.
Some rebalancing agents only treat people with inhibitors and others only people without inhibitors. Check with your provider to determine if this treatment will work for you.
How to Learn More
The best person to discuss new therapies to treat bleeding disorders is your healthcare provider at your hemophilia treatment center. NBDF regularly updates the bleeding disorders community on the progress of new therapies in our monthly newsletter, NBDF Notes, and in HemAware magazine.