Adriana Fonseca
The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility in of performing real-time drug screening on tissue taken during surgery in patients with relapsed ultra rare brain tumors (BCOR -altered, MN1-altered, CNS Sarcomas, other rare brain tumors) and of having a specialized tumor board assign a treatment plan based on the results of the drug screening and genomic sequencing. The aim of this trial is to allow every child and young adult with relapsed ultra rare brain tumors 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.
Following clinically indicated biopsy or resection, fresh tissue will be submitted for real-time high-throughput drug screening and tumor/paired-normal molecular profiling will be initiated. A specialized tumor board will issue a documented management recommendation within 28 days . Recommendations will integrate successful drug screening, all molecular/pathology data available at the time of review, prior therapy, and clinical context. Later molecular results may prompt an addendum. The treating physician retains responsibility for final treatment decisions.
Inclusion Criteria: 1. Patients must have a relapse or recurrence of one of the following rare brain tumors: * CNS sarcoma (e.g. EWS, CIC, DICER1 altered) * BCOR altered tumors (e.g. BCOR ITD; BCOR, BCORL1, EP300, fused) * Mesenchymal tumors (e.g. FET::CREB altered tumors) * MN1::PATZ1 fused tumors * PLAG- altered tumors * Astroblastomas/MN-1 altered (e.g. EWSR2BEND2 altered) * Unclassifiable tumors * Other rare brain tumors: (e.g. recently described, poorly characterized, and/or ambiguous entities) 2. Participant must be a candidate for surgical resection or biopsy.…
Specialized Treatment Plan of up to four FDA approved drugs based on participant's screening results will be assigned by Rare Brain Tumor specialized tumor board. Recommendations will be provided and to primary physician.