Anti-complement Agents for Autoimmune Neurological Disease

Jennifer A. McCombe, Sean J. Pittock

Research output: Contribution to journalReview articlepeer-review


In recent years, there has been increasing recognition of the diversity of autoimmune neurological diseases affecting all levels of the nervous system. A growing understanding of disease pathogenesis has enabled us to better target specific elements of the immune system responsible for the cell dysfunction and cell destruction seen in these diseases. This is no better demonstrated than in the development of complement directed therapies for the treatment of complement mediated autoimmune neurological conditions. Herein, we describe the basic elements of the complement cascade, provide an overview of select autoimmune neurological diseases whose pathogenesis is mediated by complement, the effector system of autoantigen bound autoantibodies, and discuss the complement directed therapies trialed in the treatment of these diseases. Several complement-directed therapies have demonstrated benefit in the treatment of autoimmune neurological diseases; we also review the trials resulting in the approval of these therapies for the treatment of AChR Ab-positive myasthenia gravis (MG) and neuromyelitis spectrum disorder. Finally, on the heels of the recent successes described, we discuss possibilities for the future, including additional targeted therapies with greater ease of administration, improved risk profiles, and other possible uses for therapeutics targeting elements of the complement cascade.

Original languageEnglish (US)
Pages (from-to)711-728
Number of pages18
Issue number3
StatePublished - Apr 2022


  • Anti-complement agents
  • Autoimmune neurologic disease
  • Myasthenia gravis

ASJC Scopus subject areas

  • Pharmacology
  • Clinical Neurology
  • Pharmacology (medical)


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