BRAIN and NERVE－神経研究の進歩71巻11号

2019年11月発行

増大特集 ALS2019

孤発性ALSに対するペランパネル

著者： 相澤仁志¹ 郭伸¹

所属機関： ¹東京医科大学神経学分野／神経分子病態学寄付講座

ページ範囲：P.1270 - P.1278

文献概要

孤発性筋萎縮性側索硬化症（ALS）の運動ニューロンではAMPA型グルタミン酸受容体を構成しているGluA2のQ/R部位の編集率が低下しており，AMPA受容体を介したCa^2＋流入が過剰となり，最終的に運動ニューロン死に至ると考えられる。実際，AMPA受容体拮抗薬ペランパネルの全身投与は，ALSの病態モデルマウスの病態進行を有意に阻止した。現在，孤発性ALSを対象としたペランパネルの無作為化二重盲検臨床試験を行い，2020年春にはその結果が得られる。

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