Phase 1/2a clinical trial in ALS with ropinirole, a drug candidate identified by iPSC drug discovery

The Pooled Resource Open-Access ALS Clinical Trials Consortium

研究成果: Article査読

10 被引用数 (Scopus)

抄録

iPSC-based drug discovery led to a phase 1/2a trial of ropinirole in ALS. 20 participants with sporadic ALS received ropinirole or placebo for 24 weeks in the double-blind period to evaluate safety, tolerability, and therapeutic effects. Adverse events were similar in both groups. During the double-blind period, muscle strength and daily activity were maintained, but a decline in the ALSFRS-R, which assesses the functional status of ALS patients, was not different from that in the placebo group. However, in the open-label extension period, the ropinirole group showed significant suppression of ALSFRS-R decline and an additional 27.9 weeks of disease-progression-free survival. iPSC-derived motor neurons from participants showed dopamine D2 receptor expression and a potential involvement of the SREBP2-cholesterol pathway in therapeutic effects. Lipid peroxide represents a clinical surrogate marker to assess disease progression and drug efficacy. Limitations include small sample sizes and high attrition rates in the open-label extension period, requiring further validation.

本文言語English
ページ(範囲)766-780.e9
ジャーナルCell stem cell
30
6
DOI
出版ステータスPublished - 2023 6月 1

ASJC Scopus subject areas

  • 分子医療
  • 遺伝学
  • 細胞生物学

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