6 Pediatric Medicine Breakthroughs Made Possible by Federal Research Funding

Life-changing treatments for children are often driven by cutting-edge research at children's hospitals.

That essential research is funded by more than celebrity fundraisers and foundation galas.

It’s made possible by investments from federal sources like the National Institutes of Health (NIH) — funding that is now under threat.

Here are six scientific breakthroughs from children’s hospitals that show how public investment changes lives.

1. CAR T-cell therapy for leukemia

Pediatric leukemia that resisted every available drug got a powerful new treatment thanks to clinical trials at children’s hospitals supported by NIH and the National Cancer Institute. In CAR T-cell therapy, doctors collect a child’s immune T cells, engineer them to recognize cancer, and return them to the body to attack leukemia directly. This approach became the first gene therapy approved in the U.S.

2. Genome sequencing

Scientists at the NIH in the 1960s discovered how the genetic code worked, winning a Nobel prize. In 2003, scientists sequenced the full human genome, supported in part by NIH funding. This enabled a new era of medicine, allowing for earlier disease diagnosis, personalized treatments like targeted cancer therapies, and predictive genetic testing to assess individual disease risks. This foundational work also spurred the development of gene therapy, which can now cure children of life-threating and debilitating conditions. 

3. Fetal surgery for spina bifida

Spina bifida once meant a lifetime of paralysis for many children. The Management of Myelomeningocele Study (MOMS) trial, co-led by several children’s hospitals and funded by the National Institute of Child Health and Human Development, proved that repairing the spinal cord in utero improved mobility and reduced complications compared with surgery after birth. Today, families across the country have access to fetal surgery programs built on those findings, offering hope where none existed.

4. Cystic fibrosis precision drugs

For decades, cystic fibrosis was considered a fatal childhood disease. Early NIH-funded research uncovered the CFTR gene and its defective protein, paving the way for precision medicines like ivacaftor and newer triple-drug combinations. These therapies dramatically improve lung function and survival, turning cystic fibrosis into a condition many children now live with instead of die from.

5. Rotavirus vaccine development

Rotavirus used to send hundreds of thousands of infants to hospitals each year with dangerous dehydration. NIH-supported collaborations with pediatric researchers helped create RotaTeq, an oral vaccine that protects babies from severe diarrhea. Approved in 2006, it now prevents millions of hospitalizations worldwide.

6. Spinal muscular atrophy cure

Not long ago, spinal muscular atrophy was the leading genetic cause of infant death. In the 1990s, NIH-supported researchers discovered that mutations in the SMN1 gene caused the disease and tested gene replacement strategies. Steady federal support bridged the years when private investment was absent. In 2019, the FDA approved Zolgensma, a one-time gene therapy that now allows babies who once faced paralysis or death to sit, crawl, and even walk.

The bottom line

As global leaders in pediatric medical training and research, children’s hospitals are committed to advancing scientific innovation that focuses on pediatric patients and medical conditions that emerge in childhood.

To ensure continued progress, we urge Congress to make sustained and robust investments in pediatric medical research across the NIH and work with children’s hospitals to strengthen the biomedical research infrastructure and prioritize children’s health.

When we put children first, we build a stronger future for everyone.