Professor Massimo Caputo and the team at the University of Bristol/Bristol Royal Hospital for Children, who have been awarded £1.4 million by the Sir Jules Thorn Charitable Trust to enable them to investigate the use of stem cell therapy for the repair of congenital heart abnormalities in very young children.
In the 1950s eight out of 10 babies with complex congenital heart disease (CHD) died before their first birthday. Today, thanks to advances in treatment and care, more than eight out of 10 CHD babies grow up to be adults. However, the long-term outcomes for most of them remain poor, significantly affecting their quality of life. The lack of growth potential in the materials used to repair CHD is the biggest problem; hence a patient’s heart outgrows the graft and requires several risky and distressing operations. At the same time a significant proportion of these children develop right heart dysfunction and failure, for which very limited treatment options exist.
This 5-year project aims to create “live” tissues using the patient’s own stem cells seeded on grafts used in every day surgery. These cellular grafts will be tested in a piglet model that closely resembles the “real world” scenario and tested for their capacity to grow and regenerate the damaged heart. The cellular grafts that show the best results in the animal model will be investigated in a first- in-human safety study in infants with the heart condition known as Tetralogy of Fallot.