Engineering the proteolytic specificity of activated protein C improves its pharmacological properties

Proc Natl Acad Sci U S A. 2003 Apr 15;100(8):4423-8. doi: 10.1073/pnas.0736918100. Epub 2003 Apr 1.

Abstract

Human activated protein C (APC) is an antithrombotic, antiinflammatory serine protease that plays a central role in vascular homeostasis, and activated recombinant protein C, drotrecogin alfa (activated), has been shown to reduce mortality in patients with severe sepsis. Similar to other serine proteases, functional APC levels are regulated by the serine protease inhibitor family of proteins including alpha(1)-antitrypsin and protein C inhibitor. Using APC-substrate modeling, we designed and produced a number of derivatives with the goal of altering the proteolytic specificity of APC such that the variants exhibited resistance to inactivation by protein C inhibitor and alpha(1)-antitrypsin yet maintained their primary anticoagulant activity. Substitutions at Leu-194 were of particular interest, because they exhibited 4- to 6-fold reductions in the rate of inactivation in human plasma and substantially increased pharmacokinetic profiles compared with wild-type APC. This was achieved with minimal impairment of the anticoagulant/antithrombotic activity of APC. These data demonstrate the ability to selectively modulate substrate specificity and subsequently affect in vivo performance and suggest therapeutic opportunities for the use of protein C derivatives in disease states with elevated serine protease inhibitor levels.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Amino Acid Substitution
  • Animals
  • Anticoagulants / chemistry
  • Anticoagulants / metabolism
  • Anticoagulants / pharmacology
  • Genetic Variation
  • Humans
  • In Vitro Techniques
  • Kinetics
  • Macaca fascicularis
  • Models, Molecular
  • Protein C / chemistry*
  • Protein C / genetics
  • Protein C / metabolism*
  • Protein C / pharmacology
  • Protein C Inhibitor / pharmacology
  • Protein Engineering
  • Rabbits
  • Recombinant Proteins / chemistry
  • Recombinant Proteins / genetics
  • Recombinant Proteins / metabolism
  • Recombinant Proteins / pharmacology
  • Serpins / pharmacology
  • Substrate Specificity
  • alpha 1-Antitrypsin / pharmacology

Substances

  • Anticoagulants
  • Protein C
  • Protein C Inhibitor
  • Recombinant Proteins
  • Serpins
  • alpha 1-Antitrypsin