Currently only 10% of the National Institutes of Health’s $37 billion annual research budget goes to support pediatric research, and only 5% of more than 7,000 rare pediatric diseases have a drug approved by the FDA.
“Pediatric research is underfunded nationally, which means kids are being left behind. This is a world-class research facility dedicated exclusively to advancing the health and well-being of children right here in Kansas City and beyond,” said Paul Kempinski, MS, FACHE, President and Chief Executive Officer, Children’s Mercy. “The research, innovation, and collaboration that happens inside this building will accelerate answers and give hope to families everywhere.”
What makes the CMRI unique is the translational approach to research. The CMRI fosters an integrated research environment where no boundaries exist between science and medicine – uniting physicians, scientists, academic colleagues and community partners working together to translate ideas and discoveries into new medicines, treatments and diagnostics.
“Every interaction at the child’s bedside or in the clinic is an opportunity for our researchers and staff to learn,” said Tom Curran, PhD, FRS, Senior Vice President, Executive Director and Chief Scientific Officer. “We’ve seamlessly integrated research with the clinical care to ensure our patients can take advantage of the latest technology and scientific advances.”
The research facility has a 50/50 ratio of dry and wet lab space, a biological safety level III facility to study organisms such as COVID-19, and a specialized Good Manufacturing Practice facility to support research involving cell and gene therapy technologies.
As CMRI rapidly emerges as a leader in pediatric translational research, it has established the following Areas of Emphasis:
- Genomic Medicine – The Genomic Medicine Center is one of the very few pediatric genome centers in the world carrying out clinical whole genome sequencing and single-cell genomics.
- Precision Therapeutics – Uses patient-specific information to optimize treatment for individual patients, which is supported by one of the largest pediatric clinical pharmacology programs in the nation.
- Population Health – Research focused on the communities we seek to help. Identifying the personal, social, economic, and environmental factors that influence the health of a child.
- Health Care Innovation – Identifies and advances products and processes that optimize the medical, surgical and therapeutic management of disease in children.
One of the initial flagship research projects of the CMRI is Genomic Answers for Kids (GA4K) – a first-of-its-kind pediatric data repository that is collecting genomic data and health information from 30,000 children and their families over the next seven years, to create a database of 100,000 genomes.
The data collected for GA4K will be openly shared with the wider international rare disease research community to hopefully lead to the discovery of unconventional genetic variants.
More than 2,230 families with rare disease and more than 2,600 patients have enrolled in the program to-date, which has resulted in more than 10,200 new genomic analyses, more than 250 genetic diagnoses and already contributed to the reporting of 10 new disease genes. In addition, the program has advanced research genomic analyses for children of 350 families with more common childhood disease: cerebral palsy and Down’s Syndrome.
The Sodens are just one of the families to receive answers through genetic testing. Ben Soden was a healthy teenager and high school wrestler when he started experiencing a rapid heart rate, even while resting. This was mysterious and worrisome since athletes normally have a low heart rate. A genetic test determined he has a rare genetic variant that causes his body to slowly metabolize a certain class of medications including his ADHD prescription.
“It was pretty amazing how quickly they were able to link my symptoms to my ADHD medication – what if I was one of those kids who suddenly had a heart attack in a middle of a wrestling meet and had no idea why?” Ben said. “The experience definitely made me think about all the other kids who could benefit in the future through genetic testing and hopefully lead to saving a lot more lives, like mine.”
Ben’s diagnosis, along with the DNA sequences based on three other Children’s Mercy patients, is displayed on the all-glass façade of the CMRI building. The windows represent the genetic anomalies and are illuminated at night, which was unveiled at the Grand Opening celebration. Ben was asked to flip the ceremonial switch.
“Seeing my DNA sequence lit up on the building for the first time was a surreal moment,” Ben said. “It gave me a sense of hope and is a way to show the entire community the important work that will be happening inside this new research institute.”
Dr. Curran added the exterior of the building was designed with intention, “The DNA of our patients is a reminder of the work that is being done inside this amazing facility every single day and the difficult cases our researchers are trying to solve.
The CMRI would not be possible without the generosity of the community. In 2018, the Hall Family Foundation and the Sunderland Foundation each donated $75 million to kickstart the construction of the new research building and the recruitment of top researchers. The combined $150 million donation is the largest one-time gift ever made to a children’s hospital for pediatric research. Since then, hundreds of members of the community have joined their generosity and made this research investment a reality.
“I’m continuously amazed at the generosity of the Kansas City community. Because of these donors, world-changing discoveries are happening right here in Kansas City, shaping our city’s future and the future of pediatric research,” said Dr. Curran. “By doing this, we will accelerate the development of medical advances and transform the lives of children around the world.”
Learn more about the Children’s Mercy Research Institute.
About Children’s Mercy
Founded in 1897, Children’s Mercy is a leading independent children’s health organization dedicated to holistic care, translational research, educating caregivers and breakthrough innovation to create a world of well-being for all children. With not-for-profit hospitals in Missouri and Kansas, and numerous specialty clinics in both states, Children’s Mercy provides the highest level of care for children from birth through the age of 21. U.S. News & World Report has repeatedly ranked Children’s Mercy as one of “America’s Best Children’s Hospitals.” For the fifth consecutive time in a row, Children’s Mercy has achieved Magnet nursing designation, awarded to only about 8% of all hospitals nationally, for excellence in quality care. More than 850 pediatric subspecialists, researchers and faculty across more than 40 subspecialties are actively involved in clinical care, pediatric research and education of the next generation of pediatric subspecialists. Thanks to generous philanthropic and volunteer support, Children’s Mercy provides hope, comfort and the prospect of brighter tomorrows to every child who passes through its doors. Visit Children’s Mercy and the Children’s Mercy Research Institute to learn more, and follow us on Facebook, LinkedIn, Twitter, Instagram and YouTube for the latest news and videos.
SOURCE Children’s Mercy