BRAIN and NERVE－神経研究の進歩71巻9号

2019年09月発行

特集 神経疾患のドラッグ・リポジショニング—新時代へ

ALS治療薬候補としてのロピニロール塩酸塩

著者： 髙橋愼一¹² 森本悟² 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

ページ範囲：P.943 - P.952

文献概要

疾患特異的iPS細胞（iPSC）を用いて家族性および孤発性ALS患者から脊髄運動ニューロンを作製し，in vitroで疾患表現型を再現した。1,232種類の既存薬ライブラリーから有効性スクリーニングを行い，ドラッグ・リポジショニングとしてロピニロール塩酸塩を用いた医師主導治験を開始した。ALS患者に対する安全性・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本第Ⅰ/Ⅱa相試験は，本邦におけるALSに対するiPSC創薬の試金石となる。

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