The hemophilia community has been waiting for gene therapy for years; many hoped it would be a cure. Quantitative and qualitative results from late stages of clinical trials for gene therapy in both hemophilia A and B have started to be compiled. With these results, we learned that the reality of gene therapy differs from original hopes and expectations: gene therapies that will be made available are promising new treatment options but are not full cures and are not for everyone. Gene therapy is a complex affair and people with hemophilia and their families must have all the information they need to make a fully informed decision about it and be able to manage their expectations.
With this in mind, the Canadian Hemophilia Society (CHS) has set in motion a series of tools to make information on gene therapy as accessible and comprehensive as possible to its community. A booklet, podcasts, webinars, surveys on expectations and knowledge, advocacy, multiple articles on its online news platform, a dedicated webpage … the CHS uses every tool in the box to make sure that gene therapy is understood by everyone and delivers all of its secrets.
The booklet, first published last March and updated this November, was inspired by, and adapted from Gene Therapy, A Practical Guide Book, by the European Haemophilia Consortium (EHC) [September 2022]. It was our first major branded tool produced and the foundation for the CHS gene therapy program. It answers multiple questions such as: How does hemophilia gene therapy work? What are the unmet needs that gene therapy answers? Who is eligible for hemophilia gene therapy? How effective is it? And, for how long? and more.
A series of podcasts were recorded in the fall of 2023 featuring well-known patient and physician experts in the field. We are pleased to announce that the first two episodes are now live.
Here are all the topics covered:
What is gene therapy for hemophilia? and What are the clinical trial results in hemophilia gene therapy? with Dr. David Lillicrap, Professor in the Department of Pathology and Molecular Medicine at Queen’s University in Kingston, Ontario
Who is eligible for gene therapy? and What is it like to receive gene therapy? with Brian O’Mahony, Chief Executive Officer of the Irish Haemophilia Society and a former president of the World Federation of Hemophilia and the European Haemophilia Consortium.
What don’t we know about gene therapy for hemophilia? and What other approaches to hemophilia gene therapies are down the road? with Dr. Glenn Pierce, a long-time researcher in coagulation products for hemophilia and currently the Medical Vice President of the World Federation of Hemophilia.
What is the shared decision-making process in hemophilia gene therapy? and How might gene therapy be delivered in Canada? with Dr. Jerry Teitel, Professor of Medicine at the University of Toronto, and Medical Director of the Hemophilia Treatment Program at St. Michael’s Hospital in Toronto, Ontario.
How might gene therapy be funded in Canada? with Dr. Durhane Wong-Reiger, President and CEO of the Canadian Organization for Rare Diseases and chair of Rare Diseases International.
On May 5, 2023, during Rendez-vous 2023 in Niagara Falls, Session 4 of the Canadian Bleeding Disorder Medical and Scientific Symposium was all about gene therapy. As for all conferences organized by the CHS since 2012, the presentations were recorded and are available on our YouTube channel.
Products in the pipeline are regularly featured on the CHS website and here on CHS CONTACT. We have also published many articles since 2020 on the subject. To read previous articles on gene therapy you simply need to do a search on top of this page using the keywords gene therapy.
Last but not least, we created a webpage entirely dedicated to gene therapy. Everything you want to know about gene therapy in one place: www.hemophilia.ca/gene-therapy.
Throughout its history, the CHS has made it its duty to provide high quality and evidence-based information to its community. Its extensive educational catalogue, numerous workshops and conferences, its former newsmagazine, Hemophilia Today, and current news platform, CHS CONTACT, are all testaments of this commitment. The gene therapy program stands as the latest comprehensive education program put together by the CHS to make sure that people with hemophilia and their families delve into gene therapy with as much relevant information as possible.
Remember: booklet, podcasts, webcasts, pipeline, articles, all in one place: www.hemophilia.ca/gene-therapy.
