There are several new medicines, or therapies, currently being explored for the treatment of bleeding disorders. These treatments are often referred to as “novel” therapies, because they use methods that are new.
Not all novel therapies being explored today focus on replacing the missing factor. Some seek to stop bleeding through other ways. Most of these products are in clinical trials, which are tests that researchers do to make sure medicines work the way they should. It is necessary for all new medicines to undergo testing in clinical trials and then be approved by the FDA before they can be prescribed by a doctor.
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 many different approaches to gene therapy, including gene transfer, cell therapy, and gene editing, also known as CRISPR. NHF has many resources that can help you understand gene therapy.
Who can undergo gene therapy?
At this time, gene therapy for hemophilia A and B is being studied in clinical trials to determine how well it works and how safe it is. This means only people who are part of a research study can receive it. In general, individuals able to participate in this type of study are:
- 18 and older
- Have severe hemophilia A or B (factor levels of less than 1%) without inhibitors
- Have no evidence of hepatitis and if they have HIV, it must be under good control
- Have no evidence of liver dysfunction (such as abnormal liver enzymes or abnormal liver biopsy)
- Have no detectable neutralizing antibodies against the vector. Antibodies are created by the body to protect us against potentially harmful things such as viruses and germs. Once our bodies make antibodies against something they neutralize or destroy that target as soon as it gets in contact with them.
- Otherwise healthy (no diabetes, history of cancer, heart disease, glaucoma, high blood pressure, etc.)
- Your healthcare provider can help you decide if participating in a study to receive gene therapy is right for you.
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. Anti-TFPI is currently in phase 3 clinical trials.
Encapsulated cell therapy
Specialized capsules containing cells which have been created to make clotting factors are placed in the body during a procedure. The clotting factors are delivered into the body at a constant rate. The capsules protect the cells from our immune system.
RNA interference (RNAi) therapy targeting antithrombin
Similar to anti-TFPI, RNA interference (RNAi) 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. RNAi is currently in phase 3 clinical trials.