After several decades of very limited or no treatment at all, the haemophilia patients who have the added burden of inhibitors, are facing an era where new possibilities of treatment become available also for them.
In this brief article we would like to offer an insight into the gene therapy, a treatment option that is currently not available for people with inhibitors, but worth looking into nevertheless. For gene therapy prospects for people with inhibitors you can read in our Ask the Expert Section (available after 1st June) or listen during the live discussion with the EHC President Declan Noone on 25th May.
The idea of gene therapy for haemophilia is using a modified virus (so that it is unable to cause a disease) to introduce a copy of the gene into the patients body that then encodes the clotting factor missing. Following treatment with the virus, patients should begin producing their own clotting factor normally.
This series is the first major piece of patient-focused discussion and education about gene therapy and haemophilia.
What is gene therapy? with Prof. Flora Peyvandi
An overview in gene therapy for haemophilia A and B with Dr. Paul Giangrande
How does a clinical trial in gene therapy for haemophilia work? with Dr. Dan Hart
Safety and gene therapy with Prof. Michael Makris
Gene therapy: A patient’s perspective with Jack Grehan
Furthermore, in anticipation of broader use of gene therapies in bleeding disorders, the EHC in collaboration with European Association of Haemophilia and Allied Disorders (EAHAD) have developed a EAHAD-EHC Joint Statement on: Promoting hub-and-spoke model for the treatment of haemophilia and rare bleeding disorders using gene therapies.