Suberoylanilide hydroxamic acid (vorinostat) up-regulates progranulin transcription: rational therapeutic approach to frontotemporal dementia

J Biol Chem. 2011 May 6;286(18):16101-8. doi: 10.1074/jbc.M110.193433. Epub 2011 Mar 23.

Abstract

Progranulin (GRN) haploinsufficiency is a frequent cause of familial frontotemporal dementia, a currently untreatable progressive neurodegenerative disease. By chemical library screening, we identified suberoylanilide hydroxamic acid (SAHA), a Food and Drug Administration-approved histone deacetylase inhibitor, as an enhancer of GRN expression. SAHA dose-dependently increased GRN mRNA and protein levels in cultured cells and restored near-normal GRN expression in haploinsufficient cells from human subjects. Although elevation of secreted progranulin levels through a post-transcriptional mechanism has recently been reported, this is, to the best of our knowledge, the first report of a small molecule enhancer of progranulin transcription. SAHA has demonstrated therapeutic potential in other neurodegenerative diseases and thus holds promise as a first generation drug for the prevention and treatment of frontotemporal dementia.

Publication types

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

MeSH terms

  • Dose-Response Relationship, Drug
  • Frontotemporal Dementia / drug therapy*
  • Frontotemporal Dementia / metabolism
  • HEK293 Cells
  • Histone Deacetylase Inhibitors / pharmacology*
  • Humans
  • Hydroxamic Acids / pharmacology*
  • Intercellular Signaling Peptides and Proteins / biosynthesis*
  • Progranulins
  • Transcription, Genetic / drug effects*
  • Up-Regulation / drug effects*
  • Vorinostat

Substances

  • GRN protein, human
  • Histone Deacetylase Inhibitors
  • Hydroxamic Acids
  • Intercellular Signaling Peptides and Proteins
  • Progranulins
  • Vorinostat