Gene therapy could save lives of premature babies

Although medical advances in perinatal care mean many more premature babies are saved each year, those who survive are often at risk of developing bronchopulmonary dysplasia, a chronic lung disease caused by having to place the tiny infants on ventilators and oxygen-rich therapy for acute respiratory failure.

Using animal models, a team of researchers at the University of Alberta have taken what they say is the first important step towards a treatment; growing new blood vessels and alveoli (the tiny air sacs where gas exchange occurs between the lungs and blood vessels) in rat lungs.

To produce these results the scientists developed a new gene therapy technique. Knowing that a particular protein, VEGF, a vascular endothelial growth factor, is crucial for the normal development of the lung, and that angiopoeitin-1, another angiogenic growth factor is crucial for blood vessel maturation, the team attached the proteins to an adenovirus and administered it through an aerosol directly into the lungs.

In effect, the virus carried the protein to the heart of millions of lung cells. Once inside the cells, the growth factor proteins went to work doing the job they were programmed to do.

“At this stage it’s simply proof of principle,” said Dr Bernard Thébaud, a clinician-scientist and neonatologist in the Department of Pediatrics. “The next step is to prove it’s possible to replicate safely in larger mammals. Once that’s done, it’s possible the concept, growing blood vessels to cure a disease that is traditionally thought of as an airway disease, could be tested clinically in people”.