Thermal profiling reveals phenylalanine hydroxylase as an off-target of panobinostat

Nat Chem Biol. 2016 Nov;12(11):908-910. doi: 10.1038/nchembio.2185. Epub 2016 Sep 26.

Abstract

We describe a two-dimensional thermal proteome profiling strategy that can be combined with an orthogonal chemoproteomics approach to enable comprehensive target profiling of the marketed histone deacetylase inhibitor panobinostat. The N-hydroxycinnamide moiety is identified as critical for potent and tetrahydrobiopterin-competitive inhibition of phenylalanine hydroxylase leading to increases in phenylalanine and decreases in tyrosine levels. These findings provide a rationale for adverse clinical observations and suggest repurposing of the drug for treatment of tyrosinemia.

MeSH terms

  • Dose-Response Relationship, Drug
  • Hep G2 Cells
  • Histone Deacetylase Inhibitors / chemistry
  • Histone Deacetylase Inhibitors / pharmacology*
  • Humans
  • Hydroxamic Acids / chemistry
  • Hydroxamic Acids / pharmacology*
  • Indoles / chemistry
  • Indoles / pharmacology*
  • Molecular Structure
  • Panobinostat
  • Phenylalanine Hydroxylase / antagonists & inhibitors*
  • Phenylalanine Hydroxylase / chemistry
  • Phenylalanine Hydroxylase / metabolism
  • Structure-Activity Relationship
  • Temperature*

Substances

  • Histone Deacetylase Inhibitors
  • Hydroxamic Acids
  • Indoles
  • Panobinostat
  • Phenylalanine Hydroxylase

Associated data

  • PubChem-Substance/316972334
  • PubChem-Substance/316972335
  • PubChem-Substance/316972336
  • PubChem-Substance/316972337
  • PubChem-Substance/316972338
  • PubChem-Substance/316972339