Improving the health of children takes visionary support. Since 2014, the Nationwide Foundation Pediatric Innovation Fund has contributed $50 million to accelerate the most promising treatments and discoveries at Nationwide Children’s Hospital. The Nationwide Foundation's most recent donation brings their cumulative support to Nationwide Children's Hospital to $100 million.
The Innovation Fund has supported several key clinical and research efforts in heart health, neonatology, injury prevention and most notably genomics. New focus areas for genomic research and clinical testing include epilepsy and other seizure disorders, behavioral health disorders and infectious disease.
A Groundbreaking Gene Therapy: Transformational Hope for Children with SMA Type 1
A gene therapy for SMA1 developed at Nationwide Children’s Hospital is bringing hope to infants affected by this devastating disease.
SMA1 is a progressive neuromuscular disease caused by a mutation in a single gene, discovered through genome sequencing. Children with SMA1 typically die by two years of age. With the natural progression of the disease, patients require feeding and breathing support usually by 12 months of age, and are unable to swallow or speak effectively.
At the end of the first SMA1 gene therapy study conducted at Nationwide Children’s, all 15 infants in the study had a good safety outcome. Of the 12 patients treated with the highest dose, 92 percent achieved head control and could sit with assistance. Seventy-five percent of the infants are now sitting for 30 seconds or longer. Two can crawl, pull to a stand and walk independently.
In 2017, this therapy was recognized as a Science Breakthrough of the Year and in 2018, the Nationwide Children’s research team received the Distinguished Clinical Research Achievement Award from the prestigious Clinical Research Forum.
The Institute for Genomic Medicine
The Institute for Genomic Medicine is one of the first ventures into pediatric personalized genomic medicine at any children’s hospital. It focuses on genomics as the root cause of many childhood diseases.
The human genome is the blueprint for what makes up a human being and can help us determine the cause and best treatment for virtually all medical conditions. Other institutions need 72 hours to sequence and analyze the human genome. At Nationwide Children’s, we have developed technology to do the same in 90 minutes. This breakthrough will change how we treat childhood diseases because for our children, their future is now.
The Innovation Fund has enabled transformational impact by helping make possible the recruitment in 2016 of world-renowned genomics researchers Richard K. Wilson, Ph.D. and Elaine R. Mardis, Ph.D to lead the Institute for Genomic Medicine (IGM) in The Research Institute at Nationwide Children's. Established in 2016, the IGM remains one of the few programs in pediatric personalized genomic medicine in the U.S. and focuses on genomics as the root cause of many childhood diseases.
Thanks to the support provided by the Nationwide Foundation Pediatric Innovation Fund, over 250 families have received clinical genomics testing, providing diagnostic answers in several cases for children with complex and previously undiagnosed conditions. In addition, over 50 children with cancer have been evaluated by genome sequencing to identify tumor-specific vulnerabilities that represent potentially new treatment directions. In the coming year, again thanks to this support, IGM will be able to offer these tests – and hope – to many more patients and their families. IGM has already grown to 120 employees working in clinical testing, technology development/validation and research projects, and we continue to identify and recruit outstanding staff.
Meet first time parents who spent months on a diagnostic odyssey. Traditional testing couldn't give them a clear diagnosis for their daughter, Charlie. Dr. Kim McBride suggested genetic testing and that's when Charlie's life changed for the better.