Novel Therapies eLearning Centre

After decades of a single approach—replacement factor infusion—the treatment landscape for the management of hemophilia is rapidly evolving and numerous exciting research avenues are being pursued. The WFH Novel Therapies eLearning Centre brings together introductory information and in-depth resources on these new developments in a convenient online hub that is updated often.

Hemostatic balance is a fine balance between too much clotting (thrombosis) and not enough clotting (bleeding). In hemophilia, an essential clotting factor is lacking, and the hemostatic balance is tipped toward too much bleeding. The current standard of care for hemophilia is life-long prophylaxis with clotting factor concentrates to replace the clotting factor that is missing and to prevent spontaneous bleeds. However, access to these products is restricted in many contexts. Furthermore, factor replacement products have several limitations, including the characteristic saw-tooth rise and fall in factor levels observed after each infusion. In the absence of stable factor levels, the body has reduced ability to form clots and to prevent and stop bleeds (hemostasis), which can lead to bleeding events and joint damage between infusions.

The “Information” tab of the Novel Therapies eLearning Centre provides a high-level overview of both the unmet need in hemophilia treatment with clotting factor concentrates, and the driving force behind the development of novel therapies. It then takes a deeper dive into innovative treatment approaches for hemophilia—non-factor replacement products and gene therapy—describing how these approaches prevent bleeds and considerations for their use. The goal with non-factor replacement products is to rebalance hemostasis without the need for replacing the clotting factor that is missing. In contrast, the goal in gene therapy is to replace an individual’s defective gene with a healthy copy. This means that an individual’s body may produce enough clotting factor on its own to prevent bleeds and reduce the need for factor infusion.

Drawn from two recently published WFH eLearning modules, Non-Factor Replacement Hemophilia Therapies and Gene Therapy for Hemophilia, the “Information” tab of the Novel Therapies eLearning Centre presents this complex content in an easy-to-read format supplemented by informative medical illustrations. With just a few clicks, the learner can gain an understanding of the essentials of these two novel therapy approaches. Written primarily for people with bleeding disorders and their families and advocates, the content also provides excellent foundational knowledge for healthcare professionals.

One type of non-factor replacement hemophilia product, factor VIII (FVIII) substitution therapy, takes the place of FVIII in the series of reactions that leads to clotting (coagulation cascade). FVIII substitution therapy carries out the natural function of activated FVIII (FVIIIa, missing or defective in hemophilia A) that is needed for effective clotting.

At the time of publication, only one such product has been approved in multiple countries for people with hemophilia A with and without inhibitors. Non-factor replacement hemophilia therapies are used strictly prophylactically to prevent bleeds, making them fundamentally different from clotting factor concentrates that can also be used to treat a suspected bleed.

Gene therapy is a promising experimental technique that uses genes rather than drugs or surgery to treat genetic diseases, including hemophilia. The most common form of gene therapy involves introducing a healthy copy of a mutated (altered) gene to restore normal function. Hemophilia A and B are simply due to a defect in a single gene (and therefore its protein product) and are ideally suited to gene therapy. The ultimate goal of gene therapy for hemophilia is a one-time administration that achieves a stable increase in factor levels over long periods of time so that no bleeds occur and regular factor infusions are no longer required.

Gene therapy techniques are relatively new and have some risks that we don’t yet fully understand and others that are as yet unknown. Researchers, institutions, and public authorities that regulate the quality and efficacy of medical products are working to ensure that gene therapy is as safe as possible. While a number are in phase 3 clinical trials, at the time of publication no gene therapy product has yet been approved for the market by a regulatory authority.

While the “Information” tab of the Novel Therapies eLearning Centre provides immediate access to the essential introductory facts, learners wishing to delve deeper into specific topics will find a wealth of materials to explore in the “Resources” tab. The resources on offer include collections of webcasts of expert presentations from the WFH 2016 World Congress and the WFH 2018 World Congress on gene therapy and non-replacement factor hemophilia therapies, detailed eLearning modules on each topic, and recently re-certified CME gene therapy modules, just to name a few. These resources have been developed for a variety of target audiences, including people with bleeding disorders, their families and advocates, hematologists and other members of the comprehensive care team. The handy social media bar associated with each resource makes sharing resources with colleagues extremely easy.

As the treatment landscape for hemophilia evolves, so will the WFH educational materials and the Novel Therapies eLearning Centre. Updated on an ongoing basis as new information or resources come available, the eLearning Centre constitutes a hub for reliable information for people with hemophilia, healthcare professionals, advocates, and the global bleeding disorders community as a whole. Check back often to see what’s new and keep abreast of developments!

As research continues, learners are also advised to consult the most recent clinical trial reports and local regulatory agency websites for the very latest developments. People with hemophilia are encouraged to initiate conversations with their healthcare provider or hemophilia specialist if they wish to explore whether novel therapies might impact the management of their bleeding disorder.