A pilot feasibility and efficacy (phase 2) trial of real time drug screening and genomic testing to determine an individualized treatment plan in children and young adults with relapsed medulloblastoma and ependymoma

The purpose of this single armed multi-center pilot trial is to allow every child and young adult with relapsed medulloblastoma to receive the most effective and least toxic therapies currently available and will pave the way for improved understanding and treatment of these tumors in the future. Moreover, if successful, it could serve as a paradigm for personalized medicine programs for other types of cancer. The study will test the feasibility of performing real-time drug screening on tissue taken during surgery, and of having a specialized tumor board assign an individualized treatment plan (up to four FDA- approved drugs) based on the results of real-time high-throughput drug screening, whole exome sequencing (WES), and RNA sequencing.

Are you Eligible? (Inclusion Criteria)

• Participants must have recurrent medulloblastoma and surgically accessible disease. Aging between ≥12 months to ≤ 39 years of age with Karnofsky ≥ 50 for participants > 16 years of age and Lansky ≥ 50 for participants ≤ 16 years of age. The participant must have recurrent medulloblastoma following at least one prior therapy for initial diagnosis or previous recurrence– surgery followed by high dose chemotherapy with stem cell rescue or multi-modality therapy of surgery, radiation and chemotherapy – prior to study registration. Participants must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.