EXTH-96. BIOPROCESSING OF SURGICAL PEDIATRIC BRAIN TUMOR SPECIMENS FOR GENOME-GUIDED PERSONALIZED DRUG TESTING

Abstract Novel treatment approaches for pediatric central nervous system (CNS) tumors are urgently needed. A lack of patient-derived tumor cells impedes progress towards developing such new therapies. We intended to overcome this challenge by establishing methods create a biorepository viable single cell suspensions brain surgical specimens. Quantitative and qualitative comparisons tissue processing strategies were performed preserve viability heterogeneous immune cells. drug targets identified analyzing pathways affected RNA transcripts that highly expressed (outliers) in patients’ tumor; outliers each patient determined RNA-seq data from individual compared with compendium 12,747 adult samples harmonized the Treehouse Childhood Cancer Initiative at UCSC Genomics Institute. The predicted anti-tumor efficacy small molecule inhibitors outlier was tested assays against short-term cultured matched patients. Successful collection required obtaining informed consent, standard operating procedures, sample recording using Laboratory Inventory Management Software (LIMS). Since 2020 we have banked 67 CNS specimens Stanford. Amongst those, 51 cases yielded sufficient cryoprotection. most common histology low-grade glioma (LGG, 26 67), majority which pilocytic astrocytoma (18 26). second third types embryonal (6 medulloblastoma, 3 AT/RT) ependymoma (4), respectively. significant differences different preservation media. An pathway previously not implicated LGG sensitivity inhibitor demonstrated. Taken together, established feasibility validating therapeutic vulnerabilities genome-guided approach cultures This works facilitates rapid development personalized treatment.