Mechanistic medicine: novel strategies for clinical trials

Autoimmunity. 2010 Nov;43(7):560-71. doi: 10.3109/08916931003674782. Epub 2010 Apr 30.

Abstract

Autoimmune diseases affect a significant proportion of the population and the development of therapeutics able to manipulate the immune response to deliver effective treatment in these diseases is an accepted approach for drug discovery. This article will focus on recent strategies for achieving selectivity through target choice, thus reducing overall clinical immunosuppression. We review the use of mechanistic pharmacodynamic assays preclinically and in the clinic to assess target engagement and to build the relationship between target coverage and efficacy, to guide dosing. Finally, we review the use of monogenic diseases to deliver proof of mechanism clinical studies and to identify patient populations in larger autoimmune diseases that may be sensitive to intervention with a specific therapeutic.

Publication types

  • Review

MeSH terms

  • Autoimmune Diseases / drug therapy*
  • Clinical Trials as Topic*
  • Drug Design*
  • Humans
  • Immunosuppressive Agents / therapeutic use*

Substances

  • Immunosuppressive Agents