Increase of anti-metastatic efficacy by selectivity- but not affinity-optimization of synthetic serine protease inhibitors

Biol Chem. 2003 Oct-Nov;384(10-11):1515-25. doi: 10.1515/BC.2003.168.

Abstract

Although tumors frequently show elevated protease activities, the concept of anti-proteolytic cancer therapy has lost momentum after failure of clinical trials with broad-spectrum matrix metalloproteinase inhibitors. Thus we need to adapt our design strategies for protease inhibitors. Here, we employed a series of seven structurally fine-modulated and pharmacokinetically closely related synthetic 4-amidinobenzylamine-based inhibitors with distinct selectivity for prototypical serine proteases in a murine T cell lymphoma liver metastasis model. This in vivo screening revealed efficacy of urokinase inhibitors but no correlation between urokinase selectivity or affinity and anti-metastatic effect. In contrast, factor Xa-selective inhibitors were more potent, demonstrating factor Xa or a factor Xa-like serine protease likely to be more determinant in this model. Factor Xa selectivity, but not affinity, significantly improved anti-metastatic efficacy. For example, factor Xa inhibitors CJ-504 and CJ-510 exert similar affinity for factor Xa (K(i)=14 nM versus 8.8 nM) but CJ-504 was 70-fold more selective for factor Xa. This correlated with higher anti-metastatic efficacy (58.8% with CJ-504; 28.2% with CJ-510). Our results show that among the protease inhibitors employed that have affinities in the nanomolar range, the strategy of selectivity-optimization is superior to further improvement of affinity to significantly enhance anti-metastatic efficacy. This appreciation may be important for the future rational design of new anti-proteolytic agents for cancer therapy.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / chemical synthesis*
  • Antineoplastic Agents / pharmacology
  • Antithrombin III / chemistry
  • Antithrombin III / metabolism
  • Benzylamines / chemical synthesis
  • Benzylamines / pharmacology
  • Disease Models, Animal
  • Drug Design
  • Drug Evaluation, Preclinical
  • Liver Neoplasms / enzymology
  • Liver Neoplasms / prevention & control
  • Liver Neoplasms / secondary*
  • Lymphoma, T-Cell / drug therapy*
  • Lymphoma, T-Cell / enzymology
  • Lymphoma, T-Cell / pathology
  • Matrix Metalloproteinase Inhibitors
  • Membrane Proteins
  • Mice
  • Neoplasm Metastasis / pathology
  • Neoplasm Metastasis / prevention & control*
  • RNA, Messenger / analysis
  • RNA, Messenger / metabolism
  • Serine Endopeptidases / analysis
  • Serine Endopeptidases / chemistry
  • Serine Endopeptidases / genetics
  • Serine Endopeptidases / metabolism
  • Serine Proteinase Inhibitors / chemical synthesis*
  • Serine Proteinase Inhibitors / pharmacology
  • Structure-Activity Relationship
  • Urokinase-Type Plasminogen Activator / analysis*
  • Urokinase-Type Plasminogen Activator / antagonists & inhibitors

Substances

  • Antineoplastic Agents
  • Benzylamines
  • Matrix Metalloproteinase Inhibitors
  • Membrane Proteins
  • RNA, Messenger
  • Serine Proteinase Inhibitors
  • Antithrombin III
  • Serine Endopeptidases
  • matriptase
  • St14 protein, mouse
  • Urokinase-Type Plasminogen Activator