I’m Aware That I’m Rare: Dr. Bernard Thébaud (409)

the phaware® interview

Dr. Bernard Thébaud is a clinician-scientist with a focus on the clinical translation of stem cell-based therapies for lung diseases. Dr. Thébaud is a senior scientist with the Ottawa Hospital Research Institute (OHRI) and Children’s Hospital of Eastern Ontario Research Institute (CHEO RI), and a neonatologist with the Children’s Hospital of Eastern Ontario (CHEO), where he provides care to critically ill newborns.

In this episode, Dr. Thébaud discusses the importance of innovative cell-based or gene therapies to lessen pulmonary hypertension.

My name is Bernard Thébaud. I’m a professor of pediatrics at the University of Ottawa in Canada. I’m also a clinician scientist. I’m a neonatologist taking care of critically ill babies in the NICU. I have a research lab that is focused on innovative therapies for neonatal lung diseases, including pulmonary hypertension.

The innovative therapies that we’re focusing on is on cell-based therapies and gene therapies. In pulmonary hypertension, so far, the therapies that are available are vasodilators that can do one thing, open up the blood vessels and vasodilate. But there’s more to the pathophysiology of pulmonary hypertension, especially in the causes of pulmonary hypertension due to poor lung growth such as bronchopulmonary dysplasia, congenital diaphragmatic hernia. In these particular cases, we now know that pulmonary vascular disease is prevalent. The idea here is that if we can sustain pulmonary vascular growth, maybe we can promote lung growth overall, but also attenuate pulmonary hypertension. With various type of cell therapies, we may be able to achieve that.

Now, it is clear, for example, in COPD in adults, there’s also a vascular component there, talking about the vascular phenotype. The same is happening with BPD, bronchopulmonary dysplasia there’s a vascular phenotype. These patients are more prone to display pulmonary hypertension. Also, those patients that survive BPD eventually get better, but we now know they never reach the full potential of their lung growth. It could be that at age 20, 30, 40, their underlying pulmonary vascular disease becomes more prominent, and these patients develop pulmonary hypertension in their prime of their life.

This is where I think cell therapies and early intervention with those cell therapies can maybe promote lung vascular growth and prevent pulmonary hypertension occurring later on. Unfortunately, the translation of discoveries from the Petri dish or the lab into patients is long. It takes 10 to 20 years. But if we want to get it right, it unfortunately takes 10 to 20 years to discovery. It takes a lot of money, as well. The federal granting agencies allow you to start the discovery process, but then when it gets to the translation, which is often called the Valley of Death, that’s where we need to find money from other sources to be able to finance and overcome that Valley of Death where we get to the confirmatory studies, into the clinical trials.

So I think that also takes, I would say, five to 10 years. It depends how promising the Phase 1 trials are. Or sometimes groups do some new innovative therapies on a compassionate basis. If this is very promising, it can become like wildfire, right? But we still have to go through the rigorous steps of testing in Phase 1 trials, Phase 2 trials, Phase 3 trials. Otherwise, we’ll never know really whether it works or not. But the Phase 1 trial or the isolated case reports can give you an indication or send a signal to the community, hey, there’s something growing there. So, yeah, I would say five to 10 years.

The PPHNet (www.pphnet.org) I think is a great concept and it is absolutely required to make great strides, accelerate the awareness, and the care that is provided to these patients. For me, specifically, PPHNet will be very critical when we get to translating those innovative therapies into the clinic. Because this is a network that allows you to very efficiently, quickly run clinical trials with dedicated patients, but also with dedicated investigators that want us to succeed.

For me, it’s important to see how the field evolves outside my specific niche interest, and allows me to put things in the bigger context of pediatric pulmonary hypertension. This is particularly of interest to me because we’re also developing gene therapies for various neonatal lung diseases. I think there could be some indications for our approach in pulmonary hypertension, as well.

So in Canada, we have access to the same medications that are here in the US. Our population obviously is smaller than in the US. So this is why collaborations with the US or PPHNet, for example, is so critical. Because again, we need the critical mass of patients to run clinical trials, and that’s why PPHNet is so important.

I’m Dr. Bernard Thébaud, and I’m aware that my patients are rare.

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