Accelerating the Clinical Translation of Nanomedicines for Pediatric Brain Tumors (ECD)

Award: $300,000 over 3 years (awarded 2025)

Principal Investigator: Joelle Straehla, Seattle Children’s Hospital

Brain tumors especially aggressive embryonal tumors (Medulloblastoma and high-grade gliomas remain among the deadliest childhood cancer, in large part because therapies simply cannot reach where the cancer spreads. Once tumor cells infiltrate the cerebrospinal fluid (CSF) and leptomeninges, cure becomes nearly impossible. The medicines available today for delivery into this space are decades old, clear from the body quickly, and often cause significant side effects. At the same time, the brain’s natural protective barrier (Blood Brain Barrier – BBB) prevents many therapies from ever reaching the tumor. For children with aggressive or metastatic disease, this combination of poor access and poor tools leaves families with few meaningful options.

This project seeks to change that by developing a new way to deliver medicine directly and more effectively to pediatric brain tumors. This project entails designing tiny, highly specialized nanocarriers that can be injected into the fluid surrounding the brain and spine. These particles are engineered to bypass the brain’s protective barriers (BBB), travel deeper into tumor tissue, and release their medicine slowly over time. This approach has the potential to improve how long a drug stays active, reduce toxicity, and treat areas that conventional therapies cannot reach. Earlier versions of this strategy have shown success in adults, including a prior FDA-approved formulation, underscoring the promise of bringing a modernized, pediatric-focused version to children.

By studying how different nanocarrier designs behave in the brain and how well they treat aggressive tumors in rigorous laboratory models, this work will generate the knowledge needed to advance the safest, most effective candidates toward clinical translation.