Creating a Hub for Pediatric Precision Medicine

Creating a Hub for Pediatric Precision Medicine
New center to advance research, training and patient care.

Vanderbilt University Medical Center is launching a new Center for Pediatric Precision Medicine to support tailored diagnostics and treatments for children.

The center aims to increase pediatric representation in the precision medicine space both at the university and nationwide. It is led by co-directors Sara Van Driest, M.D., an associate professor of pediatrics, and Prince Kannankeril, M.D., a professor of pediatric cardiology at Vanderbilt. Steve Webber, M.B.Ch.B., chair of pediatrics at Vanderbilt, is executive sponsor.

By focusing on pediatrics, the team hopes to facilitate translational research that improves long-term outcomes. “We have the opportunity to change trajectories of health by addressing diseases that affect children and have a lifelong impact,” said Van Driest.

Focus on Pediatrics

Pediatric precision medicine faces unique challenges, starting with consent and patient blood volumes that limit genetic or biomarker testing. Insurance coverage can also be an issue. Only recently was pediatric genetic testing included in Medicaid funding for precision medicine.

“We have the opportunity to change trajectories of health by addressing diseases that affect children and have a lifelong impact.”

As a result, biorepositories and genetic databases that support precision medicine often start with adults. Vanderbilt’s BioVU, for example, originally enrolled adult patients only. With advocacy from Van Driest and others, it later developed the infrastructure to accept pediatric samples.

“The new center is a formalized way to collaborate from the start and remove some of the barriers for investigators and trainees,” Kannankeril said.

Support for Translational Medicine

By crossing divisions and departments, the Center for Pediatric Precision Medicine will provide a network to help craft research questions, navigate IRB review, and leverage existing datasets. It will also serve as a precision medicine training ground for researchers and clinicians.

Said Van Driest, “In neonatology, for example, we’ve been working to expand access to the Vanderbilt Neonatal Clinical Repository that contains decades of data. We’ve imported those data into the synthetic derivative to increase visibility.”

The team is also expanding access to genomic data provided by pediatric patients with congenital heart disease to better support translational medicine.

The center will use its expertise to facilitate precision diagnostics and treatments at Monroe Carell Jr. Children’s Hospital at Vanderbilt. It will bring precision medicine into clinical practice, laying the groundwork to test implementation strategies and evaluate their impact. A major goal is to ensure existing pharmacogenomic initiatives reach pediatric patients.

Expanding Network

“We’re not going to be able to solve all the mysteries of pediatric precision medicine based on Vanderbilt data alone,” Van Driest said. “One of the long-term goals will be to build collaborations with other institutions to get the sample sizes we need to really understand differences in disease susceptibility and drug responses.”

Collaborations will also extend into the community. “A private practice pediatrician may see a pharmacogenetic result and not know exactly how to incorporate it into a treatment plan,” Kannankeril said. “They can get an expert consult through the center.”

“We can assist in interpreting results and making recommendations. It’s designed to be a backstop,” Van Driest added.

The new pediatric center will complement existing infrastructure at the university, including the Center for Personalized Medicine, Vanderbilt Genetics Institute and the Department of Biomedical Informatics’ Center for Precision Medicine, among others. Leaders from each are collaborating on a Scientific Advisory Board for the center.