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

ロピニロール塩酸塩—iPS細胞創薬

著者： 髙橋愼一¹² 森本悟² 福島弘明² 中原仁³ 岡野栄之²

所属機関： ¹埼玉医科大学国際医療センター神経内科・脳卒中内科 ²慶應義塾大学医学部生理学教室 ³慶應義塾大学医学部神経内科

ページ範囲：P.1279 - P.1288

文献概要

2018年12月より，筋萎縮性側索硬化症（ALS）患者に対するロピニロール塩酸塩の安全性・忍容性および有効性を探索するプラセボ対照，二重盲検期および非盲検継続投与期から成る第Ⅰ/Ⅱa相試験を開始した。ロピニロール塩酸塩は，家族性および孤発性ALS患者の疾患特異的iPS細胞（iPSC）を用いて作成された脊髄運動ニューロンのin vitro疾患表現型を抑制し得る薬剤として，1,232種類の既存薬ライブラリーからスクリーニングされた薬剤であり，本試験の成否は今後のiPSC創薬研究の試金石となる。

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