Dissection, Optimization, and Structural Analysis of a Covalent Irreversible DDAH1 Inhibitor

Biochemistry. 2018 Jul 31;57(30):4574-4582. doi: 10.1021/acs.biochem.8b00554. Epub 2018 Jul 20.

Abstract

Inhibitors of the human enzyme dimethylarginine dimethylaminohydrolase-1 (DDAH1) can control endogenous nitric oxide production. A time-dependent covalent inactivator of DDAH1, N5-(1-imino-2-chloroethyl)-l-ornithine ( KI = 1.3 μM, kinact = 0.34 min-1), was conceptually dissected into two fragments and each characterized separately: l-norvaline ( Ki = 470 μM) and 2-chloroacetamidine ( KI = 310 μM, kinact = 4.0 min-1). This analysis suggested that the two fragments were not linked in a manner that allows either to reach full affinity or reactivity, prompting the synthesis and characterization of three analogues: two that mimic the dimethylation status of the substrate, N5-(1-imino-2-chloroisopropyl)-l-ornithine ( kinact /KI = 208 M-1 s-1) and N5-(1-imino-2-chlorisopropyl)-l-lysine ( kinact /KI = 440 M-1 s-1), and one that lengthens the linker beyond that found in the substrate, N5-(1-imino-2-chloroethyl)-l-lysine (Cl-NIL, KI = 0.19 μM, kinact = 0.22 min-1). Cl-NIL is one of the most potent inhibitors reported for DDAH1, inactivates with a second order rate constant (1.9 × 104 M-1 s-1) larger than the catalytic efficiency of DDAH1 for its endogenous substrate (1.6 × 102 M-1 s-1), and has a partition ratio of 1 with a >100 000-fold selectivity for DDAH1 over arginase. An activity-based protein-profiling probe is used to show inhibition of DDAH1 within cultured HEK293T cells (IC50 = 10 μM) with cytotoxicity appearing only at higher concentrations (ED50 = 118 μM). A 1.91 Å resolution X-ray crystal structure reveals specific interactions made with DDAH1 upon covalent inactivation by Cl-NIL. Dissecting a covalent inactivator and analysis of its constituent fragments proved useful for the design and optimization of this potent and effective DDAH1 inhibitor.

Publication types

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

MeSH terms

  • Amidines / chemistry
  • Amidines / pharmacology
  • Amidohydrolases / antagonists & inhibitors*
  • Amidohydrolases / metabolism
  • Crystallography, X-Ray
  • Enzyme Inhibitors / chemistry*
  • Enzyme Inhibitors / pharmacology*
  • HEK293 Cells
  • Humans
  • Models, Molecular
  • Nitric Oxide / metabolism
  • Ornithine / analogs & derivatives*
  • Ornithine / chemistry
  • Ornithine / pharmacology
  • Structure-Activity Relationship
  • Valine / analogs & derivatives
  • Valine / chemistry
  • Valine / pharmacology

Substances

  • 2-chloroacetamidine
  • Amidines
  • Enzyme Inhibitors
  • N(5)-(1-imino-2-chloroethyl)ornithine
  • Nitric Oxide
  • norvaline
  • Ornithine
  • Amidohydrolases
  • dimethylargininase
  • Valine