HOMEPRODUCTSCOMPANYCONTACTFAQResearchDictionaryPharmaSign Up FREE or Login

Anticomplement C5 therapy with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.

Abstract
The complement inhibitor eculizumab is a humanized monoclonal antibody against C5. It was developed to specifically target cleavage of C5 thus preventing release of C5a and activation of the terminal pathway. Paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) are 2 diseases with distinctly different underlying molecular mechanisms. In PNH, progeny of hematopoietic stem cells that harbor somatic mutations lead to a population of peripheral blood cells that are deficient in complement regulators resulting in hemolysis and thrombosis. In aHUS, germline mutations in complement proteins or their regulators fail to protect the glomerular endothelium from complement activation resulting in thrombotic microangiopathy and renal failure. Critical to the development of either disease is activation of the terminal complement pathway. Understanding this step has led to the study of eculizumab as a treatment for these diseases. In clinical trials, eculizumab is proven to be effective and safe in PNH and aHUS.
AuthorsEdwin K S Wong, David Kavanagh
JournalTranslational research : the journal of laboratory and clinical medicine (Transl Res) Vol. 165 Issue 2 Pg. 306-20 (Feb 2015) ISSN: 1878-1810 [Electronic] United States
PMID25468487 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)
CopyrightCopyright © 2015 Elsevier Inc. All rights reserved.
Chemical References
  • Antibodies, Monoclonal, Humanized
  • Complement C5
  • Complement Inactivating Agents
  • eculizumab
Topics
  • Animals
  • Antibodies, Monoclonal, Humanized (adverse effects, therapeutic use)
  • Atypical Hemolytic Uremic Syndrome (genetics, immunology, therapy)
  • Clinical Trials as Topic
  • Complement C5 (antagonists & inhibitors)
  • Complement Inactivating Agents (adverse effects, therapeutic use)
  • Female
  • Hemoglobinuria, Paroxysmal (genetics, immunology, therapy)
  • Humans
  • Male
  • Meningococcal Infections (etiology)
  • Mice
  • Pregnancy
  • Translational Research, Biomedical

Join CureHunter, for free Research Interface BASIC access!

Take advantage of free CureHunter research engine access to explore the best drug and treatment options for any disease. Find out why thousands of doctors, pharma researchers and patient activists around the world use CureHunter every day.
Realize the full power of the drug-disease research graph!


Choose Username:
Email:
Password:
Verify Password:
Enter Code Shown: