Putting the 'HAT' back on survival signalling: the promises and challenges of HDAC inhibition in the treatment of neurological conditions

Expert Opin Investig Drugs. 2009 May;18(5):573-84. doi: 10.1517/13543780902810345.

Abstract

Decreased histone acetyltransferase activity and transcriptional dysfunction have been implicated in almost all neurodegenerative conditions. Increasing net histone acetyltransferase activity through inhibition of the histone deacetylases (HDACs) has been shown to be an effective strategy to delay or halt progression of neurological disease in cellular and rodent models. These findings have provided firm rationale for Phase I and Phase II clinical trials of HDAC inhibitors in Huntington's disease, spinal muscular atrophy, and Freidreich's ataxia. In this review, we discuss the current findings and promise of HDAC inhibition as a strategy for treating neurological disorders. Despite the fact that HDAC inhibitors are in an advanced stage of development, we suggest other approaches to modulating HDAC function that may be less toxic and more efficacious than the canonical agents developed so far.

Publication types

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

MeSH terms

  • Animals
  • Histone Acetyltransferases / antagonists & inhibitors*
  • Histone Acetyltransferases / metabolism
  • Histone Deacetylase Inhibitors / therapeutic use*
  • Histone Deacetylases / metabolism*
  • Humans
  • Nervous System Diseases / drug therapy
  • Nervous System Diseases / enzymology*
  • Signal Transduction / drug effects
  • Signal Transduction / physiology*
  • Treatment Outcome

Substances

  • Histone Deacetylase Inhibitors
  • Histone Acetyltransferases
  • Histone Deacetylases