Fighting obesity with a sugar-based library: discovery of novel MCH-1R antagonists by a new computational-VAST approach for exploration of GPCR binding sites

J Chem Inf Model. 2013 May 24;53(5):1084-99. doi: 10.1021/ci4000882. Epub 2013 Apr 26.

Abstract

Obesity is an increasingly common disease. While antagonism of the melanin-concentrating hormone-1 receptor (MCH-1R) has been widely reported as a promising therapeutic avenue for obesity treatment, no MCH-1R antagonists have reached the market. Discovery and optimization of new chemical matter targeting MCH-1R is hindered by reduced HTS success rates and a lack of structural information about the MCH-1R binding site. X-ray crystallography and NMR, the major experimental sources of structural information, are very slow processes for membrane proteins and are not currently feasible for every GPCR or GPCR-ligand complex. This situation significantly limits the ability of these methods to impact the drug discovery process for GPCR targets in "real-time", and hence, there is an urgent need for other practical and cost-efficient alternatives. We present here a conceptually pioneering approach that integrates GPCR modeling with design, synthesis, and screening of a diverse library of sugar-based compounds from the VAST technology (versatile assembly on stable templates) to provide structural insights on the MCH-1R binding site. This approach creates a cost-efficient new avenue for structure-based drug discovery (SBDD) against GPCR targets. In our work, a primary VAST hit was used to construct a high-quality MCH-1R model. Following model validation, a structure-based virtual screen yielded a 14% hit rate and 10 novel chemotypes of potent MCH-1R antagonists, including EOAI3367472 (IC50 = 131 nM) and EOAI3367474 (IC50 = 213 nM).

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Anti-Obesity Agents / chemical synthesis
  • Anti-Obesity Agents / chemistry
  • Anti-Obesity Agents / pharmacology*
  • Anti-Obesity Agents / therapeutic use
  • Binding Sites
  • Carbohydrates / chemical synthesis
  • Carbohydrates / chemistry
  • Carbohydrates / pharmacology*
  • Carbohydrates / therapeutic use
  • Drug Design*
  • Drug Evaluation, Preclinical
  • Humans
  • Models, Molecular
  • Molecular Sequence Data
  • Obesity / drug therapy*
  • Protein Conformation
  • Receptors, Somatostatin / antagonists & inhibitors*
  • Receptors, Somatostatin / chemistry
  • Reproducibility of Results
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / pharmacology*
  • Small Molecule Libraries / therapeutic use
  • User-Computer Interface

Substances

  • Anti-Obesity Agents
  • Carbohydrates
  • MCHR1 protein, human
  • Receptors, Somatostatin
  • Small Molecule Libraries