Design of a superior cytokine antagonist for topical ophthalmic use

Proc Natl Acad Sci U S A. 2013 Mar 5;110(10):3913-8. doi: 10.1073/pnas.1217996110. Epub 2013 Feb 19.

Abstract

IL-1 is a key inflammatory and immune mediator in many diseases, including dry-eye disease, and its inhibition is clinically efficacious in rheumatoid arthritis and cryopyrin-associated periodic syndromes. To treat ocular surface disease with a topical biotherapeutic, the uniqueness of the site necessitates consideration of the agent's size, target location, binding kinetics, and thermal stability. Here we chimerized two IL-1 receptor ligands, IL-1β and IL-1Ra, to create an optimized receptor antagonist, EBI-005, for topical ocular administration. EBI-005 binds its target, IL-1R1, 85-fold more tightly than IL-1Ra, and this increase translates to an ∼100-fold increase in potency in vivo. EBI-005 preserves the affinity bias of IL-1Ra for IL-1R1 over the decoy receptor (IL-1R2), and, surprisingly, is also more thermally stable than either parental molecule. This rationally designed antagonist represents a unique approach to therapeutic design that can potentially be exploited for other β-trefoil family proteins in the IL-1 and FGF families.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Topical
  • Amino Acid Sequence
  • Animals
  • Crystallography, X-Ray
  • Cytokines / antagonists & inhibitors*
  • Cytokines / chemistry
  • Drug Design*
  • Drug Stability
  • Female
  • Humans
  • Interleukin 1 Receptor Antagonist Protein / antagonists & inhibitors
  • Interleukin 1 Receptor Antagonist Protein / chemistry
  • Interleukin 1 Receptor Antagonist Protein / genetics
  • Interleukin-1beta / antagonists & inhibitors
  • Interleukin-1beta / chemistry
  • Interleukin-1beta / genetics
  • Kinetics
  • Ligands
  • Mice
  • Mice, Inbred C57BL
  • Models, Molecular
  • Molecular Sequence Data
  • Ophthalmic Solutions
  • Protein Conformation
  • Receptors, Interleukin-1 Type I / antagonists & inhibitors
  • Receptors, Interleukin-1 Type I / chemistry
  • Receptors, Interleukin-1 Type I / genetics
  • Recombinant Fusion Proteins / antagonists & inhibitors
  • Recombinant Fusion Proteins / chemistry
  • Recombinant Fusion Proteins / genetics
  • Sequence Homology, Amino Acid
  • Static Electricity

Substances

  • Cytokines
  • Interleukin 1 Receptor Antagonist Protein
  • Interleukin-1beta
  • Ligands
  • Ophthalmic Solutions
  • Receptors, Interleukin-1 Type I
  • Recombinant Fusion Proteins

Associated data

  • PDB/4GAF
  • PDB/4GAI