Synthesis and structure-activity relationship studies of N-benzyl-2-phenylpyrimidin-4-amine derivatives as potent USP1/UAF1 deubiquitinase inhibitors with anticancer activity against nonsmall cell lung cancer

J Med Chem. 2014 Oct 9;57(19):8099-110. doi: 10.1021/jm5010495. Epub 2014 Sep 17.

Abstract

Deregulation of ubiquitin conjugation or deconjugation has been implicated in the pathogenesis of many human diseases including cancer. The deubiquitinating enzyme USP1 (ubiquitin-specific protease 1), in association with UAF1 (USP1-associated factor 1), is a known regulator of DNA damage response and has been shown as a promising anticancer target. To further evaluate USP1/UAF1 as a therapeutic target, we conducted a quantitative high throughput screen of >400000 compounds and subsequent medicinal chemistry optimization of small molecules that inhibit the deubiquitinating activity of USP1/UAF1. Ultimately, these efforts led to the identification of ML323 (70) and related N-benzyl-2-phenylpyrimidin-4-amine derivatives, which possess nanomolar USP1/UAF1 inhibitory potency. Moreover, we demonstrate a strong correlation between compound IC50 values for USP1/UAF1 inhibition and activity in nonsmall cell lung cancer cells, specifically increased monoubiquitinated PCNA (Ub-PCNA) levels and decreased cell survival. Our results establish the druggability of the USP1/UAF1 deubiquitinase complex and its potential as a molecular target for anticancer therapies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, N.I.H., Intramural

MeSH terms

  • Antineoplastic Agents / chemical synthesis*
  • Antineoplastic Agents / pharmacology
  • Arabidopsis Proteins / antagonists & inhibitors*
  • Carcinoma, Non-Small-Cell Lung / drug therapy*
  • Cell Line, Tumor
  • Humans
  • Lung Neoplasms / drug therapy*
  • Nuclear Proteins / antagonists & inhibitors*
  • Proliferating Cell Nuclear Antigen / metabolism
  • Pyrimidines / chemical synthesis*
  • Pyrimidines / pharmacology
  • Structure-Activity Relationship
  • Ubiquitin-Specific Proteases / antagonists & inhibitors*
  • Ubiquitination

Substances

  • Antineoplastic Agents
  • Arabidopsis Proteins
  • Nuclear Proteins
  • Proliferating Cell Nuclear Antigen
  • Pyrimidines
  • USP1 associated factor 1, human
  • USP1 protein, human
  • Ubiquitin-Specific Proteases