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Histone deacetylase inhibitors: from target to clinical trials

William K Kelly1, Owen A O'Connor, Paul A Marks

  • 1Memorial Sloan-Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA.

Insights

Histone deacetylase (HDAC) inhibitors show promise in cancer treatment by arresting cancer cell growth. Hydroxamic acid-based HDAC inhibitors are particularly effective and well-tolerated in clinical trials.

Area of Science:

  • Oncology
  • Molecular Biology
  • Epigenetics

Background:

  • Cancer cells exhibit uncontrolled proliferation but can be induced to arrest growth.
  • DNA is packaged into chromatin by histones, and gene expression is regulated by histone post-translational modifications like acetylation.
  • Histone deacetylases (HDACs) and histone acetyltransferases control histone acetylation status.

Purpose of the Study:

  • To review compounds that inhibit histone deacetylase (HDAC) activity.
  • To highlight the role of HDAC inhibitors in cancer therapy.

Main Methods:

  • Review of literature on HDAC inhibitors.
  • Analysis of in vitro and in vivo studies of HDAC inhibitor efficacy.
  • Focus on hydroxamic acid-based HDAC inhibitors.

Main Results:

  • HDAC inhibitors induce cancer cell growth arrest, differentiation, and/or apoptosis.
  • Several HDAC inhibitors are in clinical trials for cancer treatment.
  • Hydroxamic acid-based HDAC inhibitors demonstrate efficacy in cancers with good tolerability.

Conclusions:

  • HDAC inhibitors represent a promising class of anticancer agents.
  • Targeting histone acetylation through HDAC inhibition offers a viable therapeutic strategy.
  • Hydroxamic acid derivatives are leading candidates for further clinical development.

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