Clinical Research Directory

Targeting MYC in High-Risk Medulloblastoma

Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Summary

Medulloblastoma is the most common malignant brain tumor in children. Group 3 medulloblastoma (G3 MB) represents the most aggressive molecular subtype and is associated with poor prognosis, particularly in cases characterized by high expression or amplification of the MYC oncogene. Current treatment strategies are not tailored to this subgroup and are associated with significant long-term toxicities, highlighting the need for more specific therapeutic approaches. This study aims to characterize biological processes and molecular pathways driven by high MYC expression in high-risk G3 medulloblastoma in order to identify potential therapeutic vulnerabilities. The study will investigate MYC-associated regulation of gene expression and RNA splicing in tumor cells and will define molecular dependencies that may be targeted using candidate or repurposed anticancer agents. To achieve this, publicly available genomic datasets will be analyzed, findings will be validated in patient tumor specimens, and patient-derived three-dimensional (3D) tumor models will be established from surgical samples. These models will be used for ex vivo assessment of selected therapeutic strategies in a system that preserves key features of the original tumor. This translational approach integrates computational analyses, molecular validation, and functional testing in patient-derived models to improve understanding of MYC-associated tumor biology in Group 3 medulloblastoma.

Official title: Exploiting High MYC Expression as a Potential Vulnerability for Treatment of High-Risk Medulloblastoma

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