Robert W. Vaughan Lecture in Chemical Engineering

Children comprise roughly 27% of the world's population; yet, pediatric trials make up 17% of the total number of clinical trials registered with the World Health Organization, and only 7% of clinical trials involve newborns. Therapies intended for use in children can take up to 7 years longer to go from the first clinical trial in adults to the first trial in children; often, many approved adult therapeutics are used off-label for children. These numbers highlight a significant gap in technology development for the neonatal and pediatric populations, particularly in technology that focuses on improving therapeutic outcomes for children and newborns with a range of conditions. Our research seeks to develop and evaluate therapeutic delivery systems for newborns and children, who have unique physiologies compared to adults. We focus specifically on engineering therapeutics that mitigate or attenuate ongoing injury in the brain, with the goal to improve neurological function and quality of life across the lifespan. As part of this work, we have developed living brain tissue models that are tunable to different stimuli and that capture the regional complexity and variability in response to injury and treatment. In this talk, I will discuss our use of whole hemisphere brain slices to evaluate cellular-extracellular changes in the brain microenvironment and screen therapeutics, including nanotherapeutics. I will show key design considerations that increase nanoparticle uptake and transport within the brain for improved neuroprotection in neonatal and pediatric brain diseases. I will close with a forward-thinking perspective on how we can improve translation of drug delivery technologies to the clinic for neonatal and pediatric populations.