TY - JOUR
T1 - Ropinirole, a New ALS Drug Candidate Developed Using iPSCs
AU - Okano, Hideyuki
AU - Yasuda, Daisuke
AU - Fujimori, Koki
AU - Morimoto, Satoru
AU - Takahashi, Shinichi
N1 - Funding Information:
We thank all members of the Okano Laboratory and Professors Masashi Aoki (Tohoku University), Gen Sobue (Nagoya University), and Jin Nakahara for valuable discussions. We appreciate Professors Kevin Eggan (Harvard University), Brian J. Wainger (Harvard University), Clifford J. Woolf (Harvard University), and Haruhisa Inoue (Kyoto University) for information on clinical trials. The basic study on the anti-ALS action of ROPI was performed under the support of the Japan Agency for Medical Research and Development (AMED) [the Acceleration Program for Intractable Disease Research Utilizing Disease-Specific iPS Cells to H.O. (Grant No. 19bm0804003h0003) and the Research on Practical Application of Innovative Pharmaceutical and Medical Devices for Rare and Intractable Diseases to H.O. (Grant No. JP 18ek0109395h0001, 19ek0109395h0002)]. This clinical trial will be conducted under the sponsorship of AMED [Research on Practical Application of Innovative Pharmaceutical and Medical Devices for Rare and Intractable Diseases to H.O. (Grant No. JP 18ek0109329h0001, 19ek0109329h0002)] and K Pharma, Inc. This research was also supported by grants for Keio University Global Initiative Research Projects. Regarding the study drug, all test drugs and part of the comparator will be supplied free of charge by GlaxoSmithKline K.K. H.O. is a Scientific Advisory Board member of K Pharma Inc. None of the authors have conflicts of interest with GlaxoSmithKline.
Funding Information:
We thank all members of the Okano Laboratory and Professors Masashi Aoki (Tohoku University), Gen Sobue (Nagoya University), and Jin Nakahara for valuable discussions. We appreciate Professors Kevin Eggan (Harvard University), Brian J. Wainger (Harvard University), Clifford J. Woolf (Harvard University), and Haruhisa Inoue (Kyoto University) for information on clinical trials. The basic study on the anti-ALS action of ROPI was performed under the support of the Japan Agency for Medical Research and Development (AMED) [the Acceleration Program for Intractable Disease Research Utilizing Disease-Specific iPS Cells to H.O. (Grant No. 19bm0804003h0003 ) and the Research on Practical Application of Innovative Pharmaceutical and Medical Devices for Rare and Intractable Diseases to H.O. (Grant No. JP 18ek0109395h0001 , 19ek0109395h0002 )]. This clinical trial will be conducted under the sponsorship of AMED [Research on Practical Application of Innovative Pharmaceutical and Medical Devices for Rare and Intractable Diseases to H.O. (Grant No. JP 18ek0109329h0001, 19ek0109329h0002)] and K Pharma, Inc. This research was also supported by grants for Keio University Global Initiative Research Projects. Regarding the study drug, all test drugs and part of the comparator will be supplied free of charge by GlaxoSmithKline K.K.
Publisher Copyright:
© 2019 The Authors
PY - 2020/2
Y1 - 2020/2
N2 - 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.
AB - 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.
KW - amyotrophic lateral sclerosis
KW - disease modeling
KW - drug repositioning
KW - induced pluripotent stem cells
KW - ropinirole
UR - http://www.scopus.com/inward/record.url?scp=85077653296&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85077653296&partnerID=8YFLogxK
U2 - 10.1016/j.tips.2019.12.002
DO - 10.1016/j.tips.2019.12.002
M3 - Review article
C2 - 31926602
AN - SCOPUS:85077653296
SN - 0165-6147
VL - 41
SP - 99
EP - 109
JO - Trends in Pharmacological Sciences
JF - Trends in Pharmacological Sciences
IS - 2
ER -