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分子標的薬は,分子サイズによって生物学的製剤と低分子合成化合物に分けられる。TNFやIL-6などのサイトカインや細胞表面分子を標的とした生物学的製剤,および,JAKをはじめとするサイトカインなどの細胞シグナル伝達分子を標的とした低分子合成標的薬の開発により,これまで難治性と言われた自己免疫疾患を制御し,関節リウマチでは関節の構造的損傷を抑止することが可能となってきた。中枢神経系の自己免疫性・炎症性疾患にも治療応用が期待される。
Abstract
It is well known that targeted therapies have led to a paradigm shift in the treatment of autoimmune/inflammatory diseases such as rheumatoid arthritis. Biologics use intravenous or subcutaneous injection because of their size (90-150 kDa) and target multiple cytokines such as TNF and IL-6, as well as cell surface functioning molecules such as CD28 and CD20. Orally available low-molecular weight products target intracellular signaling molecules, including JAK, BTK, and S1P1. Biologics and JAK inhibitors are novel and have new modes of action that effectively close the unmet medical needs of multiple autoimmune/inflammatory diseases. Furthermore, such targeted therapies have the potential to bring about a paradigm shift in the treatment of immune/inflammatory diseases of the central nervous system.
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