[Molecular targeted therapy for neuroimmunological diseases]

Ichiro Nakashima1

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

Developments in using monoclonal antibodies for molecular targeted therapy have demonstrated promising efficacy for various neuroimmunological diseases, including multiple sclerosis, neuromyelitis optica, myasthenia gravis, chronic inflammatory demyelinating polyneuropathy, and polymyositis. Natalizumab was the first monoclonal antibody to be approved for the treatment of multiple sclerosis in countries outside of Japan. Rituximab is widely used for the treatment of neuromyelitis optica, even though it has not received FDA approval for this condition anywhere in the world. Monoclonal antibodies are recombinant biological compounds with a well-defined target and thus carry the promise of targeting pathogenic cells with high specificity while avoiding undesired side effects. Although they usually show high efficacy, the cost of the compounds may give rise to the question of whether continuous therapy is warranted.

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