AI-driven drug repurposing for glioblastoma therapy: experimental validation of promising candidates

Authors

• KD McDaniel
• ZA Shyr
• S Sun
• D Tao
• Q Zhu
• CZ Chen
• W Zheng

Abstract

Glioblastoma (GBM) is a highly aggressive brain tumor with a poor prognosis and survival rates of less than two years. The current standard of care treatment is surgery, followed by radiation and chemotherapy with temozolomide (TMZ). However, the addition of TMZ to radiotherapy extends overall survival by only 2.5 months compared to radiotherapy alone. With an urgent need for better treatment, NCATS informaticists built an AI model based on gene expression profiles to identify drug repurposing candidates. Building upon these findings, this study investigates the evaluation of eight selected drug repurposing candidates and their effects on GBM cell lines. In vitro experiments validated Clofarabine and Ciclopirox as highly effective in selectively targeting GBM cancer cells among eight prioritized candidates. This study's success demonstrates a promising approach to expedite drug development by revealing gene expression interactions between drugs and diseases, applicable not only to rare diseases, but also to more common ones.

Scientific Focus Area: Molecular Pharmacology

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