Currently only 10% of the National Institutes of Health $ 37 billion The annual research budget is used to support pediatric research, and only 5% of more than 7,000 rare pediatric diseases have an FDA-cleared drug.
“Pediatric research is under-funded at the national level, which means children are left behind. This is a world-class research facility dedicated solely to promoting the health and wellbeing of children Kansas City and beyond, “said Paul Kempinski, MS, FACHE, President and Chief Executive Officer, Children’s Mercy. “The research, innovation and collaboration in this building will accelerate the answers and give hope to families everywhere.”
What makes the CMRI unique is its translational research approach. The CMRI promotes an integrated research environment in which there are no boundaries between science and medicine – doctors, scientists, academic colleagues and community partners work together to translate ideas and discoveries into new drugs, treatments and diagnostics.
“Every interaction at the child’s bedside or in the clinic is an opportunity for our researchers and staff to learn,” he said Tom Curran, PhD, FRS, Senior Vice President, Executive Director and Chief Scientific Officer. “We have seamlessly integrated research into clinical care to ensure our patients benefit from the latest technology and scientific advances.”
The research facility has a 50/50 ratio of dry to wet laboratory space, a Biosafety Level III facility for studying organisms such as COVID-19, and a specialized good manufacturing practice facility to support research with cell and gene therapy technologies.
As CMRI is rapidly becoming a leader in pediatric translational research, it has set the following priorities:
- Genomic Medicine – The Genomic Medicine Center is one of the few pediatric genome centers in the world that performs clinical sequencing of the whole genome and single cell genomics.
- Precision Therapeutics – Uses patient-specific information to optimize treatment for individual patients, supported by one of the largest pediatric clinical pharmacology programs in the country.
- Population Health – Research is focused on the communities we want to help. Identify the personal, social, economic and environmental factors that affect a child’s health.
- Healthcare Innovation – Identifies and develops products and processes that optimize medical, surgical, and therapeutic management of diseases in children.
One of the first CMRI research projects is Genomic Answers for Kids (GA4K) – a unique pediatric data archive that will collect genomic and health information from 30,000 children and their families over the next seven years. create a database of 100,000 genomes.
The data collected for GA4K will be openly shared with the broader international rare disease research community, hopefully leading to the discovery of unconventional genetic variants.
To date, more than 2,230 families with rare diseases and more than 2,600 patients have signed up for the program. This has led to more than 10,200 new genome analyzes and more than 250 genetic diagnoses, and has already contributed to the reporting of 10 new disease genes. In addition, the program has advanced genomic research analyzes for children from 350 families with more common childhood diseases: cerebral palsy and Down’s syndrome.
The Sodens are just one of the families who get answers through genetic testing. Ben Soden was a healthy teenager and high school wrestler when he started experiencing fast heart rates even when resting. This was mysterious and worrying as athletes typically have low heart rates. A genetic test revealed that he has a rare genetic variant that causes his body to slowly metabolize a certain class of drugs, 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 the middle of a wrestling meeting and had no idea why?” Said Ben. “The experience definitely made me think of all the other children who could benefit from genetic testing in the future and hopefully lead to many more lives being saved, like mine.”
Ben’s diagnosis is displayed on the all-glass facade of the CMRI building, along with the DNA sequences based on three other Children’s Mercy patients. The windows depict the genetic abnormalities and are illuminated at night, which was revealed at the opening ceremony. Ben was asked to flip the ceremonial switch.
“It was a surreal moment to see my DNA sequence lit up on the building for the first time,” said Ben. “It made me feel hopeful and is a way to show the entire community the important work that will be done in this new research institute.”
Dr. Curran added that the exterior of the building was designed with purpose: “Our patients’ DNA is a reminder of the work that is done every day in this amazing facility 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 made donations $ 75 million to stimulate the construction of the new research building and the recruitment of top researchers. The combined $ 150 million The donation is the largest one-time gift ever given to a children’s hospital for pediatric research. Since then, hundreds of community members have joined in their generosity and made this research investment a reality.
“I am always amazed at the generosity of the Kansas City Community. Because of these donors, world-changing discoveries are taking place here in Kansas City“Shaping the future of our city and the future of pediatric research,” said Dr. Curran. “In this way, we will accelerate the development of medical advances and change the lives of children around the world.”
Learn more about the Children’s Mercy Research Institute.
About the mercy of children
Founded in 1897, Children’s Mercy is a leading independent child health organization dedicated to holistic care, translational research, carer training, and breakthrough innovation to create a world of well-being for all children. With non-profit hospitals in Missouri and Kansasand numerous specialty clinics in both states offer Children’s Mercy the highest level of care for children from birth to 21 years of age. US News & World Report has repeatedly ranked Children’s Mercy as one of “America’s Best Children’s Hospitals”. For the fifth consecutive year, Children’s Mercy has received the Magnet Nursing Award, which only about 8% of all domestic hospitals receive for excellence in quality care. More than 850 pediatric subspecialists, researchers and faculties from more than 40 subspecialties are actively involved in clinical care, pediatric research, and training of the next generation of pediatric subspecialists. Thanks to generous philanthropic and volunteer support, Children’s Mercy provides hope, comfort and the prospect of a better tomorrow to every child who walks 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 Mercy of Children