Transcription:

Melanie Cole (Host): Welcome to Transformational Pediatrics with Children's Mercy, Kansas City. I'm Melanie Cole, and I invite you to listen as we examine transforming pediatric research. Joining me is Dr. Thomas Curran. He's the Senior Vice President, Chief Science Officer and Executive Director at the Children's Mercy Research Institute. Doctor, it's a pleasure to have you join us today as pediatric research is so exciting and important, and we've seen great improvements in children's health thanks to research; from cancer cure rates, to expanded life expectancies for kids with chronic disease, as well as prevention of disease through vaccines and awareness. Why is pediatric research even more important today?

Thomas Curran, PhD, FRS (Guest): Pleasure to chat with you, Melanie and really pediatric research is really important because kids are being left behind. Historically children were treated with medicines that were developed for adult disease and maybe the dose was stepped down a little bit and that simply doesn't work. What we're recognizing now, for example, in the rare disease arena, there may be as many as 8,000 rare diseases out there, but 7,000 of them manifest first in young kids. So, we need to study the diseases in the young kids themselves.

Host: So, as I mentioned in my intro when we're talking about prevention of disease, vaccines, awareness for parents and for kids, chronic disease, cancer, all of these things. What are the biggest challenges facing children's health and children's health research today?

Dr. Curran: Yeah, the challenges actually in essence based on a good thing, that diseases, severe diseases are quite rare in kids, but that creates a challenge because there are not enough of the kids suffering from a particular disease to give industry a significant return on the investment, on the research that we would need to put in to develop treatments for that disease. And that's why children's hospitals have to work on them. The second challenging aspect is that kids are not little adults and they keep changing. Every year, they're different. We have to keep up with those changes and what works for a three-year-old is not going to work for a teenager. So, we need to be very precise in our medications, depending on the age of the child.

Host: What great points you just made. All of those. So, tell us a little bit about Children's Mercy Research Institute and how you see it addressing these challenges that you just mentioned.

Dr. Curran: Well, Children's Mercy has been engaged in research almost from the very beginning, but it wasn't a big part of the operation. The primary focus was on delivering the highest quality clinical care to children. But over time, certain areas of research have grown. For example, our clinical pharmacology program grew to a very substantial program and requires an infrastructure for support. So, a decision was made in 2012, that Children's Mercy would embark on a process to define a full Children's Translational Research Institute embedded into the hospital. I came along in 2016 to meet that challenge and build an integrated environment where any encounter between a caregiver and a child can provide information that can then be used for research to benefit all children.

Host: What a lovely answer. So, tell us what's unique or different about the Children's Mercy Research Institute, and more specifically about your approach to pediatric research and your philosophy of care for children that have these conditions.

Dr. Curran: So, the unique aspects that we're really emphasizing, is the close integration with the clinic. All the research that goes on inside the new Research Institute is, is for a single purpose. It's to help children. So, that means that we are partners. So, it's not like there's a research scientist does something and then hands off to a nurse or a doctor in the clinic to continue. Everyone is around the table. So, that team spirit is something that's built into the fabric of the institution. We work on the diseases that the children bring to us. And we proclaim that on, on the front of the building. We put our kids DNA sequences in a pattern on the glass facade of the building, because we're telling people that what children bring to us in terms of problems, define what our research projects are going to be.

Host: That's so interesting. So, it does seem to me like so many institutions talk about translational research or taking research from bench to bedside. What is Children's Mercy doing to make that a reality and not just really a buzzword? How do you feel your research impacts patients as you get into this and other trials?

Dr. Curran: I'll give you an example of that. So, our Genome Center, which has been running for quite some time, we had one of the first pediatric genome centers anywhere in the world, has now been expanded. And the new head of that Genome Center, Dr. Tomi Pastinen coined phrase for what he does. He calls it bedside genomics. He wants to take genomics all the way to the patient bedside and make it as easy as possible for children to benefit from the genomics revolution.

So, we've launched a very large program called Genome Answers for Kids that specifically addresses children with rare diseases. We're able to consent those kids into research study that provides the support for genetic analysis because of the very generous donors in, in Kansas City. But that study is not like a de-identified clinical study. The results can be returned to the patients. So, it's kind of a win-win-win. And it means the research study comes right to the bedside.

Host: Which is such an important aspect of what you do. So, we've heard about the promise of genomics and personalized medicine. How is that promise being translated into better care, both globally and at Children's Mercy? You just briefly touched on that. So, expand just a little for us.

Dr. Curran: So, let me tell you about another program that exists in the Research Institute. It's got the greatest name of any program, and once I give you the name, you'll understand what it's all about. This is the Precision Medicine Program called GOLDILOKs®, you know, not too little, not too much, just the right amount.

Host: How clever.

Dr. Curran: And that's led by Dr. Steve Leeder who's really good at making up names as well as doing science. But the idea is that the design of, of the research project from the beginning is aligned with the needs of the patients and the families. The other area of strength that we have in Children's Mercy Research is so-called population health or outcomes research, where you go a step further. It's not enough just to come up with a newer treatment, a new process, and then give it to the doctor and say, hey, it's good. You're good to go. We actually need to find out how well does that play out in the community with patients and families in their everyday lives. So, outcome research, outcomes research is really built into everything we do.

We want to know when we make changes, that those changes are working. And importantly that everybody has access. So, we share the information on our website in consortium. Many of our investigators are part of national or international consortium, and we've taken the position that all our data needs to be shared with everyone across the world. And so that children everywhere can benefit from discoveries made here in Kansas City.

Host: Well, doctor, I would like you now to look into your crystal ball. And what do you see as some of the next big areas of emphasis in pediatric research? This can be globally or locally. What will everyone be talking about a year or three years from now? And what will Children's Mercy Research Institute be known for among your peers at other hospitals?

Dr. Curran: Those are great and very challenging questions, Melanie. And let me do my best to answer them. Some of what we're seeing is being affected by what we're just going through right now, the pandemic. The pandemic has actually made it very clear how important is that science and medicine must be tightly integrated and aligned. And we've seen complicated scenarios in some children. And as of now, we don't know exactly why one child gets a very mild response to the virus. And the other has a very severe response. But we are seeing a very significant impact of stress in the pediatric population. Kids have been out of school for awhile and, you know, school is stressful enough when you're in it. It's even more stressful when you're out of it and trying to manage from a remote learning perspective.

This generation of children are pointing us to the fact that there are a lot of issues that we need to take care of. Behavioral health, mental health issues in the pediatric population are really significant and require a whole community response. Children's Mercy will be a partner with all the other players in trying to do the very best in terms of behavioral health for our population. The Genome Study that I mentioned has the ambition to create the world's largest data repository of pediatric genomics. We plan to consent a total of 30,000 children and a total of a hundred thousand individuals when we include parents and siblings, where available, in this genetic data repository. That will be available for scientists everywhere to work on.

So, those will be two very big aspects, but the other challenge that we're seeing, which has also been made evident by the pandemic is the plight of underserved communities. Underserved communities that don't have the access to healthcare that they need. What happens when that's a child. So, we now need to look at every, every project that we're doing and ask, how are we serving the diversity and the equity and the inclusion of those populations to make sure that our work is as strong as it can be. Because the more diverse the population that we study, the stronger our data are. So, those are three areas that I think I would want to be at the top of the list of priorities for Children's Mercy in the next three years.

Host: That's absolutely fascinating and truly on point Doctor. So as we wrap up, for other providers that are looking to refer to the Children's Mercy Research Institute, what would you like them to know as a summary about the Institute and when, or how you can have the most impact on the future of children's health?

Dr. Curran: The biggest message I want to give to the providers is that we are your partners. We want to work with you and help you. We want to make it easy for you to get access to our services. We want to even give you opportunities to participate in research, when you want to. There is a lot of activity going on in engaging the community and understanding the perspective of, of the children themselves and their families and how they face the challenges of, of healthcare. And the providers are on the frontline. So, we need the providers, we need their insights, we need their collaboration and cooperation. And we hope to offer them in return, the benefits of, of the research studies that are ongoing and access to research studies, which sometimes mean a therapeutic option that a child might not have had because it's not approved standard of care. So, there can be an advantage to participating in research studies.

Host: Well there's certainly can be. What an informative episode this was Doctor. Thank you so much for joining us today. And for more information on transforming pediatric research at Children's Mercy Research Institute, please visit children'smercy.org/research to get connected with one of our providers. This has been Transformational Pediatrics with Children's Mercy, Kansas City. Please remember to subscribe, rate and review this podcast and all the other Children's Mercy podcasts. Until next time, I'm Melanie Cole.