Gene therapy for hemophilia results are recently published in the New England Journal of Medicine, trial led by NHS in London. Clinical researchers at Barts Health NHS Trust and Queen Mary University of London, members with hemophilia A (the most common type) have found that more than one year on from a single treatment with a gene therapy drug are indicating normal levels of the previously missing protein, and effectively curing them.
A single infusion of the gene therapy drug demonstrated improved levels of the essential blood coagulating protein Factor VIII, with 85% of patients achieving normal or near-normal Factor VIII levels even many months after treatment. As the first successful gene therapy trial for the hemophilia A, the transformational outcomes have specific importance.
There are around 2000 individuals with severe hemophilia A in the UK. A hereditary genetic condition dominantly affecting men, individuals with severe hemophilia A have virtually none of the protein factor VIII which is essential for blood to clot. It puts those affected at risk of excessive bleeding even from the slightest injury and additionally causing progressive internal bleeding, which can be life-threatening. Recurring bleeding into joints can likewise prompt dynamic joint damage and arthritis.
Currently, only treatment involves multiple weekly injections to control and prevent bleeding, however, there is no cure. The trial saw patients across England injected with a duplicate of the missing gene, which enables their cells to produce the missing clotting factor. Following patients for up to nineteen months, tests demonstrate that eleven out of thirteen patients in the trial now have normal or near-normal levels of the already missing factor and each of the thirteen patients has possessed the capacity to stop their previously regular treatment.
“We have seen mind-blowing outcomes which have far exceeded our expectations. When we began we thought it would be a huge achievement to demonstrate a 5% improvement, so to actually be seeing normal or near normal factor levels with a dramatic decrease in bleeding is quite simply amazing. We really now have the potential to transform care for individuals with hemophilia utilizing a single treatment for individuals who right now should inject themselves as frequently as each other day. It is so exciting “, explained by Professor John Pasi.
Haemophilia Centre Director at Barts Health NHS Trust and Professor of Haemostasis and Thrombosis at the Queen Mary University of London The group will now hold further tests widening participants globally to include individuals in the USA, Europe, Africa and South America.
“Incredibly exciting is the potential for a significant improvement in how hemophilia is treated globally. A single dose of medication that would so dramatically improve the lives of patients over the world is an amazing prospect”, continued by Professor Pasi.
“Pathology research is often responsible for ground-breaking developments in diagnoses and treatments that change the lives of patients, said by Professor Jo Martin, President, The Royal College of Pathologists.
“What is truly remarkable about this revolutionary new gene therapy are the significant life-changing impacts it offers patients with hemophilia. We might want to congratulate College fellow, Professor Pasi, and his group at Barts Health NHS Trust and the Queen Mary University of London for their work in making a simple but transformational treatment for patients.”