Pulmonary arterial hypertension (PAH) is a rare and deadly disease that involves progressive damage to the tiny blood vessels that feed the lungs. In most cases, the cause is unknown and treatments are limited.
Dr. Duncan Stewart pioneered a novel biotherapeutic approach to treat PAH using a patient’s own genetically-enhanced cells. The approach involves collecting cells from the patient’s blood, growing them in the lab to produce blood vessel (endothelial) progenitor cells and then engineering the cells to produce greater amounts of nitric oxide, a natural substance that enhances their regenerative activity and enlarges blood vessels.
A Phase 2 clinical trial of this approach was recently published in The Journal of Heart and Lung Transplantation. Although the trial had to be stopped early due to the pandemic, 12 patients were enrolled and the treatment was well-tolerated. Patients who received the therapy also showed some signs of benefit, in terms of exercise ability and right heart function after six months.
“The field of biotherapeutics is rapidly advancing, and we will continue to investigate every avenue to improve the lives of people with this deadly disease,” said Dr. Stewart, senior scientist at The Ottawa Hospital, cardiologist at the University of Ottawa Heart Institute and professor at the University of Ottawa.
Authors: Stewart DJ, Mehta S, Chandy G, Hirani N, Granton J, Hambly N, Swiston J, Danovitch K, Sabri E, Giulivi A, Courtman DW, Fergusson DA.
Cores: Biotherapeutics Manufacturing Centre, Ottawa Methods Centre
Funding: Northern Therapeutics
The Ottawa Hospital is a leading academic health, research and learning hospital proudly affiliated with the University of Ottawa and supported by The Ottawa Hospital Foundation. All researchers at The Ottawa Hospital follow a Responsible Innovation Framework for developing and commercializing innovations in a responsible way. As an expert in pulmonary hypertension, Dr. Stewart provided advice to Northern Therapeutics on the administration of the trial. Dr. Stewart did not receive a salary from Northern and had no role in enrolling patients in the trial.
