Discovery of agents that eradicate leukemia stem cells using an in silico screen of public gene expression data

Blood. 2008 Jun 15;111(12):5654-62. doi: 10.1182/blood-2007-11-126003. Epub 2008 Feb 27.

Abstract

Increasing evidence indicates that malignant stem cells are important for the pathogenesis of acute myelogenous leukemia (AML) and represent a reservoir of cells that drive the development of AML and relapse. Therefore, new treatment regimens are necessary to prevent relapse and improve therapeutic outcomes. Previous studies have shown that the sesquiterpene lactone, parthenolide (PTL), ablates bulk, progenitor, and stem AML cells while causing no appreciable toxicity to normal hematopoietic cells. Thus, PTL must evoke cellular responses capable of mediating AML selective cell death. Given recent advances in chemical genomics such as gene expression-based high-throughput screening (GE-HTS) and the Connectivity Map, we hypothesized that the gene expression signature resulting from treatment of primary AML with PTL could be used to search for similar signatures in publicly available gene expression profiles deposited into the Gene Expression Omnibus (GEO). We therefore devised a broad in silico screen of the GEO database using the PTL gene expression signature as a template and discovered 2 new agents, celastrol and 4-hydroxy-2-nonenal, that effectively eradicate AML at the bulk, progenitor, and stem cell level. These findings suggest the use of multicenter collections of high-throughput data to facilitate discovery of leukemia drugs and drug targets.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aldehydes / pharmacology
  • Animals
  • Anti-Inflammatory Agents, Non-Steroidal / chemistry
  • Anti-Inflammatory Agents, Non-Steroidal / pharmacology
  • Antineoplastic Agents / pharmacology*
  • Cell Death / drug effects
  • Cysteine Proteinase Inhibitors / pharmacology
  • Databases, Genetic
  • Gene Expression Profiling*
  • Gene Expression Regulation, Leukemic / drug effects
  • Humans
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / pathology
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Models, Genetic
  • Neoplastic Stem Cells / drug effects*
  • Neoplastic Stem Cells / pathology
  • Neoplastic Stem Cells / physiology
  • Oligonucleotide Array Sequence Analysis
  • Pentacyclic Triterpenes
  • Sesquiterpenes / chemistry
  • Sesquiterpenes / pharmacology
  • Terpenes / pharmacology
  • Triterpenes / pharmacology
  • Xenograft Model Antitumor Assays

Substances

  • Aldehydes
  • Anti-Inflammatory Agents, Non-Steroidal
  • Antineoplastic Agents
  • Cysteine Proteinase Inhibitors
  • Pentacyclic Triterpenes
  • Sesquiterpenes
  • Terpenes
  • Triterpenes
  • parthenolide
  • 4-hydroxy-2-nonenal
  • celastrol