St. Jude Children’s Research Hospital invites patients, families and clinicians to visit with St. Jude Hemophilia Team members at the National Hemophilia Foundation’s 70th Annual Meeting in Chicago. Stop by Booth 43 to learn more about our gene therapy research for hemophilia at the St. Jude Hemophilia Treatment Center.
Gene therapy for hemophilia
St. Jude has been a pioneer in gene therapy since 1997. Recently, our doctors helped develop a successful gene transfer treatment that improved the quality of life for some adult patients with severe hemophilia B. We will soon be recruiting participants for a new clinical trial to test this technique in adults with hemophilia A.
Learn how St. Jude is leading the way in gene therapy research for hemophilia.
Gene therapy provides life-changing relief from hemophilia
Gene therapy developed at St. Jude, University College London and the Royal Free Hospital has transformed life for 10 men with severe hemophilia. The therapy provides a safe, reliable source of the blood clotting protein factor IX that has allowed some patients to adopt a more active lifestyle. The results of the study were published in 2014 in the New England Journal of Medicine.
One of the beauties of the gene therapy approach is that it corrects the abnormality at a very basic level, at the gene level, but also has the potential after just a single intervention to correct the disease process long-term for the patients. Just a single administration of the viral vector can lead to a long-term cure. And that is a novel, groundbreaking approach.
All 10 participants with hemophilia B have had persistent benefit from the factor IX gene transfer with continuously measurable factor IX levels, decrease in bleeding events and less use of factor concentrates for several years now. It has been a significant change for their quality of life – an amazing experience for the whole team, participants and researchers included. We are excited that we now also have a gene therapy product for patients with severe hemophilia A and can start recruiting for a new trial evaluating a single infusion of a factor VIII gene containing vector.
Hemophilia Treatment Team at St. Jude
- Physician Assistant
- Nurse Practitioner
- Nurse Case Manager
- Senior Coordinator - Clinical Research Operations