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Related Experiment Video

Updated: Apr 22, 2026

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[C5: eculizumab].

Katsuichi Miyamoto1

  • 1Department of Neurology, Kinki University School of Medicine.

Brain and Nerve = Shinkei Kenkyu No Shinpo
|October 10, 2014
PubMed
Summary

Eculizumab, a monoclonal antibody, blocks complement system activation by targeting C5. It is approved for paroxysmal nocturnal hemoglobinuria and shows promise for treating complement-mediated neurological disorders.

Area of Science:

  • Immunology
  • Pharmacology
  • Neurology

Context:

  • The complement system is a critical part of innate immunity.
  • Complement system dysregulation is implicated in various autoimmune and neurological diseases.
  • Eculizumab targets the terminal complement cascade.

Purpose:

  • To review the mechanism of action of eculizumab.
  • To discuss the clinical applications of eculizumab in paroxysmal nocturnal hemoglobinuria.
  • To explore the potential of eculizumab in treating neurological disorders mediated by the complement system.

Summary:

  • Eculizumab is a humanized monoclonal antibody that inhibits the terminal complement cascade by binding to C5.
  • It prevents the formation of C5a and C5b, thereby blocking complement-mediated cell lysis.

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  • Eculizumab is clinically approved for paroxysmal nocturnal hemoglobinuria and is under investigation for neuromyelitis optica and myasthenia gravis.
  • Impact:

    • Eculizumab offers a targeted therapeutic approach for complement-mediated diseases.
    • Its efficacy in paroxysmal nocturnal hemoglobinuria demonstrates the potential of complement inhibition.
    • Further research may establish eculizumab as a treatment for intractable neurological conditions.