Why Life-Saving Pediatric Treatments Need NIH Funding

Trace the steps of any pediatric diagnosis, treatment, or cure and you’ll likely find footprints leading to the doors of a children’s hospital laboratory or the halls of a university associated with them.

The only FDA-approved gene therapy for spinal muscular atrophy — the top genetic cause of infant mortality — was tested in clinical trials led by Nationwide Children’s Hospital.

The development of essential pediatric vaccines — including those for rotavirus, COVID-19, and Ebola — relied on research and testing from children’s hospitals like Children’s Hospital of Philadelphia.

The latest advancement in treating childhood cancer — chimeric antigen receptor (CAR) T-cell therapy — was empowered by research at children’s hospitals like St. Jude Children’s Research Hospital. (Thanks to advances in effective therapies like CAR-T, the five-year survival rate for children with cancer has doubled over the last 50 years, reaching 85%).

Follow those steps further and the path will take you to the corridors of Congress, which historically helped fund scientific advancements through a bipartisan commitment to biomedical research and strengthening the National Institutes of Health.  

The necessity of federal research funding

Substantial time and resources are required to get from an observation at the microscope to treatment in the clinic: you need laboratories, registries, clinical trial teams, equipment, administration, infrastructure, preclinical studies, yearslong trials — and a wide range of pediatric research staff to do it all.

Compared to adult research, pediatric studies receive little private investment because the markets are small and pediatric clinical research is complex, requiring special considerations for developing bodies.

That’s why federal research funding is essential to improving the lives of children with disease, even though children’s hospitals make their own substantial investments in biomedical research. When federal funding drops or halts, labs shutter, personnel lose grants, and projects with years of momentum evaporate.

That means fewer trials available to families, reduced access to cutting-edge therapies, and slowed progress on cancer, debilitating diseases, and chronic conditions.

In short, it means less hope for kids and their families.

How federal funds save lives

Two decades ago, scientists discovered that a single missing gene caused spinal muscular atrophy (SMA), a degenerative disease that inevitably leads to paralysis and early death.

With NIH and foundation grants, researchers built animal models, tested replacement genes, and helped prove safety in early trials.

Federal funding covered the long, expensive middle years when private companies wouldn’t invest in a rare pediatric disease. That support let academic teams at Nationwide Children’s run the first clinical trials.

The result: Zolgensma, the first one-time gene therapy for SMA, giving children a chance to walk, breathe, and thrive for a full lifetime.

Return on investment

While 22% of the U.S. population is under 18 years old, less than 4% of total NIH funding was awarded to pediatric-related research.

Arbitrary cuts to research erode the very capacity the nation needs to treat childhood illness. Thoughtful policy will protect medical research investments so pediatric studies can endure long enough to prove a treatment’s safety and efficacy in improving long-term health outcomes.

The next breakthrough for pediatric cancer, congenital heart disease, or rare genetic disorders may be under study now in a children’s hospital.

How much is that worth?