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NIH Research Festival

September 23 – 25, 2024

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

This page was last updated on Tuesday, August 6, 2024

Download or preview the program here

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Current Research Festival

  • 2025
  • 2024
    • General Schedule of Events
    • 2024 NIH Distinguished Scholars Program
    • Poster Sessions
    • Concurrent Workshops
    • NIH Resource Information Fair
    • National Academy of Science Mini-Symposium
    • Special Wednesday Events
    • Vendor Exhibit Information
    • Green Labs Fair
    • Research Festival Committee
  • 2023
    • General Schedule of Events
    • NIH Early–Career Investigator Lectures
    • Poster Sessions
    • Concurrent Workshops
    • NIH Resource Information Fair
    • Vendor Exhibit Information
    • Research Festival Committee
  • 2021
  • 2019
    • General Schedule of Events
    • Plenary Sessions
    • Poster Session
    • Data Blitz
    • FARE Award Ceremony
    • Special Exhibits on Resources for Intramural Research
    • Technical Sales Association (TSA) Research Festival Exhibit Tent Show
    • Research Festival Committee
  • 2018
    • General Schedule of Events
    • Plenary Sessions
    • Concurrent Symposia Sessions
    • Poster Sessions
    • FARE Award Ceremony
    • Special Exhibits on Resources for Intramural Research
    • Technical Sales Association (TSA) Research Festival Exhibit Tent Show
    • Research Festival Committees
  • 2017
    • General Schedule of Events
    • Plenary Sessions
    • Concurrent Symposia Sessions
    • Poster Sessions
    • FARE Award Ceremony
    • Future Research Leaders
    • Special Exhibits on Resources for Intramural Research
    • Technical Sales Association (TSA) Research Festival Exhibit Tent Show
    • Research Festival Committees
  • 2016
    • General Schedule of Events
    • Plenary Sessions
    • Concurrent Symposia Sessions
    • Poster Sessions
    • Browse by Presenter Name
    • Browse by Presenter IC
    • FARE Award Ceremony
    • Future Research Leaders
    • Special Exhibits on Resources for Intramural Research
    • Technical Sales Association (TSA) Research Festival Exhibit Tent Show
    • Tours of the National Library of Medicine and Clinical Center
    • Research Festival Committees
  • 2015
    • General Schedule of Events
    • Plenary Sessions
    • Concurrent Workshop Sessions
    • Poster Sessions
    • Browse by Presenter Name
    • Browse by Presenter IC
    • FARE Award Ceremony
    • Future Research Leaders
    • Special Exhibits on Resources for Intramural Research
    • TSA Research Festival Exhibit Show
    • NIH Tours
    • Research Festival Committees
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