Cell-based therapy for bronchopulmonary dysplasia in preterm infants 1.

Bronchopulmonary dysplasia (BPD) is the most common complication of extreme prematurity. Currently, there is no specific treatment available. Preclinical studies support cell therapy as a promising therapy for BPD in preterm infants. A successful translation to a safe and effective clinical intervention depends on multiple factors including the perspective of neonatal health care providers. A 2-hour workshop with 40 Canadian neonatologists was held to enhance the design of a phase II trial of stem cells for babies at risk for BPD, with a focus on the population to target and the outcomes to measure in such a trial. The consensus was that infants recruited in an early trial of stem cells should be the ones with the highest risk of developing severe BPD. This risk should be established based on known antenatal, perinatal, and postnatal risk factors. The primary outcome in a phase II trial will be focussed on a non-clinical outcome (e.g., a dose-finding study or a safety study). With other aspects of a translational study discussed, this workshop contributed to accelerate the design of a first Canadian clinical cell-therapy study for BPD in preterm infants.