Identification of Novel Disruptor of Telomeric Silencing 1-like (DOT1L) Inhibitors through Structure-Based Virtual Screening and Biological Assays

J Chem Inf Model. 2016 Mar 28;56(3):527-34. doi: 10.1021/acs.jcim.5b00738. Epub 2016 Mar 4.

Abstract

Histone methyltransferases are involved in many important biological processes, and abnormalities in these enzymes are associated with tumorigenesis and progression. Disruptor of telomeric silencing 1-like (DOT1L), a key hub in histone lysine methyltransferases, has been reported to play an important role in the processes of mixed-lineage leukemia (MLL)-rearranged leukemias and validated to be a potential therapeutic target. In this study, we identified a novel DOT1L inhibitor, DC_L115 (CAS no. 1163729-79-0), by combining structure-based virtual screening with biochemical analyses. This potent inhibitor DC_L115 shows high inhibitory activity toward DOT1L (IC50 = 1.5 μM). Through a process of surface plasmon resonance-based binding assays, DC_L115 was founded to bind to DOT1L with a binding affinity of 0.6 μM in vitro. Moreover, this compound selectively inhibits MLL-rearranged cell proliferation with an IC50 value of 37.1 μM. We further predicted the binding modes of DC_L115 through molecular docking analysis and found that the inhibitor competitively occupies the binding site of S-adenosylmethionine. Overall, this study demonstrates the development of potent DOT1L inhibitors with novel scaffolds.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Gene Silencing*
  • Histone-Lysine N-Methyltransferase
  • Humans
  • Methyltransferases / antagonists & inhibitors*
  • Molecular Structure
  • Surface Plasmon Resonance
  • Telomere*

Substances

  • DOT1L protein, human
  • Methyltransferases
  • Histone-Lysine N-Methyltransferase