When babies are born prematurely — especially at fewer than 28 weeks in the womb — their lungs are not fully formed and they need help to breathe. At neonatal units in hospitals around the world, doctors put these babies on ventilators to give them additional oxygen.
Unfortunately, the very device that keeps these tiny babies alive can create life-long consequences by triggering bronchopulmonary dysplasia (BPD).
“Approximately 40% of extreme pre-term babies will get BPD,” says Dr. Bernard Thébaud, a researcher and clinician with The Ottawa Hospital and the Children’s Hospital of Eastern Ontario. “They will have a higher risk of developing asthma and of being re-hospitalized after being sent home. They may be at risk of early onset aging of their lungs — something similar to smoker’s lung.”
There is no treatment for BPD. Steroids that decrease the inflammation it causes can also inhibit brain development. “So we’re very cautious in administering steroids,” says Dr. Thébaud, who is working on a stem cell solution to the ventilator/BPD conundrum.
In mesenchymal stem cells extracted from Wharton’s jelly, a gelatinous substance within the umbilical cord, Dr. Thébaud hopes to find a BPD treatment that will also help other organs thrive.
“These cells will do several things. They will attenuate the inflammation, but also promote lung growth. In addition, we believe their benefits will not be limited to the lung. They will also have some beneficial effects on the brain, the gut and other organs in preterm babies that still need to grow and need to be protected from injury. “
So far, Dr. Thébaud, who is also a professor of pediatrics at the University of Ottawa where his holds the uOttawa Partnership Research Chair in Regenerative Medicine, has only tested the mesenchymal stem cells on animals. But the results have been encouraging, with the stem cells protecting the lungs from oxygen induced injuries. “We still have to do further tests with the cells made in a Good Manufacturing Process facility for a clinical grade product.”
He looks forward to conducting a Phase 1 clinical trial in the near future to test the feasibility and safety in newborns. “If it’s safe, we’ll move to Phase 2 and enroll more babies.”
As a researcher who cares for these fragile babies every day, Dr. Thébaud is both frustrated and hopeful.
“When we see these babies in our unit and we discuss what we can offer, we can see how the disease evolves and we know that we will be stuck at one point. Sometimes a nurse will come up to me and say, ‘Oh, Bernard, this baby needs your stem cells. Can you bring some over from our lab?’ On one hand it’s frustrating. On the other, it’s very rewarding to see that everyone recognizes the potential of this therapy and is waiting for it.”
The beauty of these mesenchymal stem cells, also called stromal cells, is that they are easily harvested from umbilical cords that would otherwise be discarded as medical waste. They are also easy to expand in a lab, generating the millions of cells required for transplant. Because no genetic match is required between donor and recipient, they can be supplied off-the-shelf. And they are powerful.
“Because the cells are young, they might be more potent than bone-marrow-derived mesenchymal stem cells,” says Dr. Thébaud. “So it could be that the product we are developing for the babies could also be beneficial in treating adult diseases.” and superior to current bone marrow-derived stem cells.”