Ropinirole, a New ALS Drug Candidate Developed Using iPSCs

Hideyuki Okano, Daisuke Yasuda, Koki Fujimori, Satoru Morimoto, Shinichi Takahashi

Research output: Contribution to journalReview article

Abstract

Induced pluripotent stem cells (iPSCs) are increasingly used in the study of disease mechanisms and the development of effective disease-modifying therapies for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). Recently, three candidate anti-ALS drugs – ropinirole (ROPI), retigabine, and bosutinib – have been identified in iPSC-based drug screens and are now being evaluated in clinical trials for safety and effectiveness. We review the preclinical data, clinical research design, and rationale for ROPI as an anti-ALS drug candidate compared with those of the other two drugs. We also discuss the use of iPSCs for understanding and monitoring treatment response as well as for new insights into the development of new drugs and therapeutic interventions for major neurodegenerative diseases.

Original languageEnglish
JournalTrends in Pharmacological Sciences
DOIs
Publication statusAccepted/In press - 2020 Jan 1

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Keywords

  • amyotrophic lateral sclerosis
  • disease modeling
  • drug repositioning
  • induced pluripotent stem cells
  • ropinirole

ASJC Scopus subject areas

  • Toxicology
  • Pharmacology

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