Promising treatment for preemies’ lungs receives $550,000 grant
Dr. Bernard Thébaud has spent a career treating the tiniest preemies at CHEO for lung disease.
Treatment and care of babies with bronchopulmonary dysplasia — underdeveloped lungs that become damaged from ventilation and long-term oxygen use — has shaped his career as a neonatologist. It has also driven world-leading stem cell research by Thébaud and colleagues that could prove to be a game-changer for babies with what is the most common complication of preterm birth.
Thébaud and colleagues from The Ottawa Hospital, CHEO and the University of Ottawa, whose research involves umbilical cord stem cells, have been awarded a $550,000 grant from the Ontario Institute of Regenerative Medicine aimed at getting prepared for clinical trials of the treatment that Thébaud has called “a breakthrough in the making.”
In lab rodents, the stem cell treatment transformed lungs similar to those of premature infants, which can look almost like smokers’ lungs, to fully functioning, healthy ones.
The next step is to begin testing the stem cell treatments on infants. At a time when medical breakthroughs have allowed ever younger babies to survive, lung damage continues to be a serious worry in premature babies. There are treatments to help protect and mature babies’ lungs. Some of those treatments have serious side effects and none have the “magical” effect — in Thébaud’s words — of regenerating healthy lung tissue seen in his research and benefiting other organs at the same time.
Thébaud said the possibilities for the experimental treatment are exciting — the cells, he said, are a powerful anti-inflammatory agent — but getting the clinical trials process right is key.
“Being first is always nice, but getting it right, especially for something like this that is so promising, is very important.”
That will mean consulting with parents, as well as physicians, about using stem cells on newborns.
Asking parents of some of the most fragile infants to participate in the clinical trial could be tricky.
Trials sometimes fail, Thébaud noted, because of a lack of participants. Working with parents on the research and design of the study is aimed at preventing that.
The Phase 1 clinical trial is unlikely to begin until early 2019, he said. It would involve injecting stem cells into 10 to 15 preemies born before 28 weeks gestation and still on ventilation.
The clinical trial will assess safety and feasibility of the experimental treatment, but not its effectiveness — that would come with later clinical trials. But it could help the babies nonetheless.
“I would be very candid and honest (with parents), ” said Thébaud, “saying this is experimental and studies in the lab show promising results suggesting it protects the lungs of lab animals. And in order to get to test this in humans we need to do Phase 1, in which we would enrol patients we think would benefit.”
Researchers have already done some public forums on the treatment, he said, noting some parents were asking why they couldn’t get the treatment now.
Working with patients to develop clinical trials is a trend in research, a move away from what is now seen as a more paternalistic approach.
“It is important to engage the parents,” he said. “In the end, it is about their babies.”
It has been decades since the development of artificially produced surfactant, which keeps the air sacs in the lungs open and is not produced sufficiently in many premature infants. As recently as the 1980s, Thébaud said, preterm infants used to die because they hadn’t produced sufficient surfactant. Thébaud called that “an amazing breakthrough,” adding, “I hope we can do the same.”