People with haemophilia A are born with a genetic defect that means they do not produce clotting factor VIII – a protein needed to stop bleeding. Thirteen patients at Barts Health NHS Trust were given the treatment, after which 11 have continued to produce near-normal levels of the protein.
The trial marks the first successful use of gene therapy to treat this type of haemophilia, and demonstrates the important role pathologists play at the forefront of medicine.
College President Professor Jo Martin said:
“Pathology research is often responsible for ground-breaking developments in diagnoses and treatments that transform the lives of patients.
“What is truly remarkable about this revolutionary new gene therapy are the profound life-changing effects it offers patients with haemophilia. We would like to congratulate College Fellow, Professor Pasi, and his team at Barts Health NHS Trust and Queen Mary University of London for their work in creating a simple but transformational treatment for patients.”