Game Changer Grant Awarded to Praveen Raju and Oren Becher

A major barrier to the treatment of brain tumors has been the inability of most anti-cancer drugs to cross into the brain to reach sufficient levels at the tumor site. We have recently developed a novel nanoparticle drug delivery technology that efficiently crosses the blood-brain brain barrier (BBB) and delivers drugs to the site of brain tumors thus minimizing toxicity to normal brain regions and systemic organs. In this proposal, we will optimize the use of this technology to deliver drugs targeting the molecular drivers of DIPG. Our technically innovative and multidisciplinary collaborative studies seek to realize the promise of precision medicine to improve outcomes for children with these deadly cancers.

The primary objective of this proposal is to address issues with blood-brain barrier penetration of precision medicines for DIPG to improve their efficacy and minimize toxicities. Our primary endpoint is to identify tumor-selective treatment strategies that synergize with current standard of care therapies such as radiation therapy for DIPG.

Delivering therapeutics specifically to intracranial tumors to minimize systemic toxicities is possibly, the mainlimitation for the treatment of CNS tumors. Given our rational approach for coordinating tumor specific drug targeting technology with first line radiation therapy for DIPG, we are optimistic that a pharmacologically accessible therapeutic strategy poised for immediate clinical translation will emerge from our work. If successful, our studies allow for the delivery of combinations of several classes of anti-cancer drugs for treatment of DIPG.