Complement C3-Targeted Therapy: Replacing Long-Held Assertions with Evidence-Based Discovery

Trends Immunol. 2017 Jun;38(6):383-394. doi: 10.1016/j.it.2017.03.003. Epub 2017 Apr 14.

Abstract

Complement dysregulation underlies several inflammatory disorders, and terminal complement inhibition has thus far afforded significant clinical gains. Nonetheless, emerging pathologies, fueled by complement imbalance and therapy-skewing genetic variance, underscore the need for more comprehensive, disease-tailored interventions. Modulation at the level of C3, a multifaceted orchestrator of the complement cascade, opens up prospects for broader therapeutic efficacy by targeting multiple pathogenic pathways modulated by C3-triggered proinflammatory crosstalk. Notably, C3 intervention is emerging as a viable therapeutic strategy for renal disorders with predominantly complement-driven etiology, such as C3 glomerulopathy (C3G). Using C3G as a paradigm, we argue that concerns about the feasibility of long-term C3 intervention need to be placed into perspective and weighed against actual therapeutic outcomes in prospective clinical trials.

Keywords: AMY-101; C3 glomerulopathy; C3 inhibitors; anti-C5 therapy; clinical efficacy; compstatin.

Publication types

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

MeSH terms

  • Animals
  • Antibodies, Blocking / therapeutic use
  • Clinical Trials as Topic
  • Complement Activation
  • Complement C3 / immunology
  • Complement C3 / metabolism*
  • Evidence-Based Medicine
  • Glomerulonephritis, Membranous / drug therapy*
  • Glomerulonephritis, Membranous / immunology
  • Humans
  • Immune Complex Diseases / drug therapy*
  • Immune Complex Diseases / immunology
  • Inflammation / drug therapy*
  • Inflammation / immunology
  • Models, Immunological
  • Molecular Targeted Therapy

Substances

  • Antibodies, Blocking
  • Complement C3