Institution:  University of California, San Francisco
Study Name:  Clinical Trial: Determinants of Response and Resistance in PLGG – Everolimus 
Principal Investigator:  Dr. Michael Prados/Dr. Daphne Haas-Kogan
Study date:  December 2013

This proposal fills a critical unmet need for a pediatric population with few other therapeutic alternatives and addresses an important problem in PLGG therapy, namely, how to best target dysregulated components within signaling pathways. This study (PNOC001) will elucidate mechanisms by which the most common genetic aberrations in PLGGs influence responses to novel, promising, translatable agents and will enable the next generation of clinical trials in which rational drug combinations are administered to appropriate patients in hypothesis-driven studies. This proposal steps beyond the usual approach of testing agents empirically in response-based trials. It utilizes a novel clinical trial statistical design to establish if specific molecular features are predictive markers of response to a targeted agent and establishes a new paradigm for PLGGs in which tumor tissue is acquired from each child in order to identify biomarkers of response. Funding support for the trial provides for the trial’s implementation and continued patient accrual across PNOC’s 15 member clinical trial consortium. Funding for the trial by A Kids’ Brain Tumor Cure Foundation thus supports the robust accrual into an important clinical trial and enables children from all areas of the country to have access to a novel, directed treatment for PLGGs.

For more information, please contact Dr. Daphne Haas-Kogan at dhaas-kogan@lroc.harvard.edu

For more information about other clinical trials through the Pacific Pediatric Neuro-Oncology Confortium, please see their website at www.pnoc.us.