Stilbene Boronic Acids Form a Covalent Bond with Human Transthyretin and Inhibit Its Aggregation

J Med Chem. 2017 Sep 28;60(18):7820-7834. doi: 10.1021/acs.jmedchem.7b00952. Epub 2017 Sep 18.

Abstract

Transthyretin (TTR) is a homotetrameric protein. Its dissociation into monomers leads to the formation of fibrils that underlie human amyloidogenic diseases. The binding of small molecules to the thyroxin-binding sites in TTR stabilizes the homotetramer and attenuates TTR amyloidosis. Herein, we report on boronic acid-substituted stilbenes that limit TTR amyloidosis in vitro. Assays of affinity for TTR and inhibition of its tendency to form fibrils were coupled with X-ray crystallographic analysis of nine TTR·ligand complexes. The ensuing structure-function data led to a symmetrical diboronic acid that forms a boronic ester reversibly with serine 117. This diboronic acid inhibits fibril formation by both wild-type TTR and a common disease-related variant, V30M TTR, as effectively as does tafamidis, a small-molecule drug used to treat TTR-related amyloidosis in the clinic. These findings establish a new modality for covalent inhibition of fibril formation and illuminate a path for future optimization.

Publication types

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

MeSH terms

  • Amyloidosis / drug therapy
  • Amyloidosis / genetics
  • Amyloidosis / metabolism
  • Boronic Acids / chemistry*
  • Boronic Acids / pharmacology*
  • Crystallography, X-Ray
  • Humans
  • Ligands
  • Models, Molecular
  • Point Mutation
  • Prealbumin / chemistry
  • Prealbumin / genetics
  • Prealbumin / metabolism*
  • Protein Aggregates / drug effects*
  • Stilbenes / chemistry*
  • Stilbenes / pharmacology*

Substances

  • Boronic Acids
  • Ligands
  • Prealbumin
  • Protein Aggregates
  • Stilbenes