Translational research is a phrase used frequently today. But what does it really mean for children and their families?
At the Children’s Research Institute, translational research means we are translating the biology of pediatric disease and scientific discoveries made at the bench into improvements in diagnosis and clinical care for our patients at the bedside.
Research is integrated into everything we do at Children’s Mercy. Every patient encounter, every interaction, every piece of data we collect is held to the highest research standard and is focused on finding answers for our patients and their families.
Though we care for more than 200,000 children each year, many of the conditions we encounter at Children’s Mercy are rare. This makes research even more critical for these children and their families as they search for a diagnosis and treatment. It also makes the need for philanthropic support all the more important.
Unique to translational research at the Children’s Research Institute and Children’s Mercy is our approach to precision medicine. When we treat a child, we look at that specific child, that family, the nature of the disease and the nature of that child’s interaction with the disease.
Featured Research Projects
Clinical Pharmacology and GOLDILOKS
The Children’s Mercy Clinical Pharmacology program and GOLDILOKS are an excellent example of precision medicine and translational research in action.
GOLDILOKS is a research program designed to ensure that children receive the precise dose of medicine they need, when they need it. This research is funded by a $3.5 million grant from the National Institutes of Health as well as a grant from the Sarli Family Foundation.
The CHAMP® App is an innovative PC tablet-based application that automates and simplifies at-home monitoring for children born with a single ventricle heart defect and their families. Developed by Children’s Mercy researchers, the CHAMP App is the first of its kind for interstage home monitoring. The app allows parents and the medical team to track everything from the child’s oxygen saturation levels to nutritional intake.
Children’s Mercy has funding for several hundred tablets and is partnering with other hospitals across the country to provide this resource. The national distribution of tablets will allow for monitoring of nearly 800 patients a year for the typical six-month periods between the two critical surgeries for babies with single ventricle heart defects. This is made possible through two lead grants from the Claire Giannini Fund.
Bronchopulmonary Dysplasia (BPD)
The Donald Thibeault Neonatal Laboratories and the Center for Infant Pulmonary Disorders at Children’s Mercy has funding from the National institutes of Health as well as the Cooper Connor Endowed Fund for Pulmonary Research to examine the mechanisms by which sepsis in babies contributes to BPD using animal models.
The ability to study BPD from a clinical, translational and basic science perspective is a strength of Neonatology at Children’s Mercy.
Chronic Kidney Disease in Children (CKiD)
Children’s Mercy is one of two coordinating centers for the National Institutes of Health-funded Chronic Kidney Disease in Children (CKiD) study, and has contributed to an improved understanding of CKD and its comorbidities, knowledge that has been embraced by both basic scientists and clinicians.
CKiD is the largest study of its kind ever conducted in North America, with 42 pediatric centers currently participating in the project and nearly 900 children enrolled in the first two patient cohorts.
Most recently, the NIH gave CKiD approval to enroll an additional 190 children over the next two years in a third cohort.
Milestones in Pediatric Research at Children's Mercy
- As one of the two clinical coordinating centers for the Chronic Kidney Disease in Children study (CKiD), Children's Mercy has been advancing kidney care through the leadership of this study for more than 14 years. CKiD is the largest study of its kind ever conducted in North America, with 49 pediatric centers currently participating in the project and more than 900 children enrolled to date. The NIH-funded CKiD study has generated nearly 100 peer-reviewed publications with a truly translational impact on care.
- When the Food and Drug Administration announced it first U.S.-approved cancer gene therapy in August 2017, Children’s Mercy was recognized as one of the first sites to get involved with the multisite clinical trial. The new CAR-T therapy is for children and young adults with acute lymphoblastic leukemia that is resistant to treatment or has relapsed.
- The University of Kansas Cancer Center announced in August that the National Cancer Institute (NCI) renewed its national cancer center designation for five years and that Children’s Mercy had been formally approved as a cancer center consortium partner.KU Cancer Center and Children’s Mercy initially joined forces in 2015 to identify collaborative ways to explore medical innovations and increase pediatric research efforts that would benefit children with cancer.
- CHAMP is a dedicated, multi-disciplinary team that has been built to meet the needs of some of our most complex patients: babies who are born with single ventricle heart disease and are in the critical inter-stage period between the first and second stages of surgery. In February 2017, Children's Mercy received a Health Innovation Award from Microsoft for this life-saving technology developed at our Ward Family Heart Center. The NIH recently awarded a five-year grant worth nearly $3.5 million to Children’s Mercy, making it one of four Specialized Centers for Research in Pediatric Developmental Pharmacology. The grant will support continued development of GOLDILOKs, a research program designed to ensure that children receive the precise dose of medicine they need, when they need it.
- The Center for Pediatric Genomic Medicine at Children’s Mercy – the first genome center of its kind in a children’s hospital – made national headlines in 2012 (and many times since) by sequencing the DNA of critically ill babies in just 50 hours, drastically reducing the time it takes to solve these medical mysteries.
- Working with the World Health Organization, Children’s Mercy researchers introduced the world to Mercy Tape in 2013, creating a remarkably accurate way to estimate kids’ weights when scales aren’t available or plausible.