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NEW! Gene Therapy for Hemophilia eLearning module

Have you heard the latest about gene therapy for hemophilia? Do you understand what genes are, how gene therapy works, and why it sparks such interest? Are you following the latest gene therapy clinical trial results, but have some questions about what it all means for individuals with hemophilia? The new WFH eLearning module Gene Therapy for Hemophilia explores all of these questions and more.

The Gene Therapy for Hemophilia module is an adaptation of the Global National Member Organization (NMO) Training 2018 plenary presentation by Glenn Pierce, WFH Vice President, Medical, and is part of the WFH Treatment Products eLearning Program designed for individual self-directed or small-group learning.

“Written for people with bleeding disorders and their carers and advocates, this comprehensive module is also an excellent foundational resource on gene therapy for any healthcare professional,” explains Glenn Pierce. As part of the Treatment Products eLearning Program, the module begins with a summary of some of the key concepts of modules 1 and 2: Introduction to Bleeding Disorders, and Evolution of Treatment Products. These two modules provide a thorough study of factor replacement therapies for bleeding disorders and reviewing them before embarking upon module 3 is highly recommended.

Gene therapy is an exciting therapeutic avenue that is currently being explored for a number of conditions, including hemophilia. Ongoing technical advances and developments in our understanding of how to deliver gene therapy and how the body responds to it are converging to make this the most promising moment in the history of gene therapy. Clinical trial results make headlines, but truly understanding what is happening in the field and what this may mean for individual people with hemophilia requires a deeper dive.

Gene Therapy for Hemophilia explains the complex concepts that underlie gene therapy in simple terms, with colourful medical illustrations and a handy glossary to help explain terminology. Interactive features, such as clickable slideshows, popup notes, and icons containing essential information make the learning experience fun and engaging. Linked resources allow learners to explore topics of particular interest to even greater depths. Self-assessment—through optional quizzes and progress checks—allows learners to test their knowledge, and guides them back to specific sections for re-study if necessary. Learners can work through a full module from start to finish, or jump straight to individual sections with the interactive table of contents.

The main tools currently used to deliver gene therapy are viral vectors. A tragic history of viral contaminants in blood products has left the bleeding disorders community extremely wary of viruses. This module makes clear how these viral vectors differ from viruses that cause disease and what is being done to fully understand the safety implications of all aspects of gene therapy.

Gene therapy offers great promise, and expectations are high, but there are also important unknowns, especially regarding long-term predictability, efficacy, and safety. Gene Therapy for Hemophilia explores these questions, and provides clear discussions of the state of our current understanding and the need for greater experience and knowledge. Some clinical trials report promising results, but participating in a gene therapy clinical trial is not right for every person with hemophilia. Gene Therapy for Hemophilia explains inclusion criteria for clinical trials and why lifelong follow up is required for those who do participate.

While gene therapy appears to have the potential to raise factor level in some people with hemophilia to normal or near normal levels, any person with hemophilia who receives gene therapy still has much to gain from, and much to contribute to, the bleeding disorders community. Continued association with the local hemophilia treatment centre and local and international patient organizations is important for long-term follow up, psychosocial support, genetic counselling, musculoskeletal management and other aspects of the person’s whole wellbeing. This module presents gene therapy within the broader context of the comprehensive management of hemophilia. Click here to access Gene Therapy for Hemophilia on the WFH eLearning platform.

The final module in the Treatment Products eLearning Program, Module 4 Non-Factor Replacement Hemophilia Therapies is coming soon! It will provide an engaging overview of another very exciting area of research and development in the treatment of bleeding disorders, discussing the driving force behind the development of non-factor replacement alternatives, and how these products differ from factor replacement products. Be on the lookout for this module, as it will also soon be available in the Treatment Products eLearning Program on the WFH eLearning Platform!