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Updated: Dec 6, 2025

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
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Published on: December 11, 2016

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Computational Drug Repositioning Identifies Potentially Active Therapies for Chordoma

Jeffrey I Traylor1, Hadley E Sheppard2, Visweswaran Ravikumar3

  • 1Department of Neurosurgery, The University of Texas MD Anderson Cancer Center, Houston, Texas.

Neurosurgery
|October 5, 2020
PubMed
Abstract

Insights

This study repurposed FDA-approved drugs for aggressive chordoma bone tumors. Cytarabine significantly reduced chordoma cell viability, while tretinoin showed cell-line-dependent effects, suggesting potential new therapies.

Area of Science:

  • Oncology
  • Pharmacology
  • Genomics

Background:

  • Chordomas are aggressive bone tumors with high recurrence rates.
  • Currently, no FDA-approved chemotherapy exists for chordoma treatment.
  • Computational drug repositioning offers a promising avenue for identifying novel chordoma pharmacotherapies.

Purpose of the Study:

  • To identify FDA-approved compounds for repurposing in chordoma treatment.

Main Methods:

  • Differential gene expression data from chordoma tissues were compared with pharmacogenomic interactions in the Comparative Toxicogenomics Database (CTD).
  • The ksRepo drug-repositioning platform was utilized to identify potential compounds.
  • Selected compounds underwent preclinical validation in human chordoma cell lines (CH22 and UM-Chor1).

Main Results:

  • In silico analysis identified 13 chemical compounds from the CTD.
  • Six compounds were selected for preclinical validation based on clinical relevance.
  • Cytarabine demonstrated significant concentration-dependent cytotoxicity in both CH22 and UM-Chor1 cells.
  • Tretinoin exhibited significant cytotoxicity in CH22 cells but less so in UM-Chor1 cells.

Conclusions:

  • Cytarabine effectively reduces human chordoma cell viability.
  • Tretinoin's efficacy is dependent on the specific chordoma cell line.
  • Further evaluation of cytarabine and tretinoin in expanded cell line and animal models is recommended.
Keywords:
ChemotherapyChordomaComputational drug repositioningCytarabineTretinoinksRepo

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