Triptolide and its prodrug Minnelide target high-risk MYC-amplified medulloblastoma in preclinical models.

ABSTRACT: Most children with medulloblastoma (MB) achieve remission, but some face very aggressive metastatic tumors. Their dismal outcome highlights the critical need to advance therapeutic approaches that benefit such high-risk patients. Minnelide, a clinically relevant analog of the natural product triptolide, has oncostatic activity in both preclinical and early clinical settings. Despite its efficacy and tolerable toxicity, this compound has not been evaluated in MB. Utilizing a bioinformatic data set that integrates cellular drug response data with gene expression, we predicted that Group 3 (G3) MB, which has a poor 5-year survival, would be sensitive to triptolide/Minnelide. We subsequently showed that both triptolide and Minnelide attenuate the viability of G3 MB cells ex vivo. Transcriptomi

SUBMITTER: Rodriguez-Blanco J 

PROVIDER: S-EPMC11290968 | biostudies-literature | 2024 Jun

REPOSITORIES: biostudies-literature