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Series GSE209696 Query DataSets for GSE209696
Status Public on Jun 30, 2023
Title Ropinirole hydrochloride identified by iPSC drug discovery for amyotrophic lateral sclerosis: a single-centered, randomized, double-blind, placebo-controlled phase 1/2a trial
Organism Homo sapiens
Experiment type Expression profiling by high throughput sequencing
Summary We assessed the safety and efficacy of ropinirole, a candidate drug for amyotrophic lateral sclerosis (ALS) identified using induced pluripotent stem cells (iPSCs) technology, by a randomized controlled trial (UMIN000034954). Twenty participants were randomly assigned to ropinirole or placebo for 24 weeks in the double-blind period, followed by a 24-week open-label active extension period. Primary outcomes were safety and tolerability. Secondary outcomes evaluated the therapeutic effects of ropinirole. Adverse events were mostly those previously reported, with similar rates in both groups. Participants in the ropinirole group had lived an additional 27.9 weeks without disease progression compared with the placebo group at 12 months. We validated and confirmed the efficacy of ropinirole in patients with ALS using the largest publicly available real-world registry database of ALS (PRO-ACT). The iPSCs-derived motor neurons from the participants showed the D2R expression and the potential involvement of the SREBP2-cholesterol synthetic pathway in the therapeutic effects.
 
Overall design Motor neurons were differenciated from iPS cells derived from sporadic ALS patients and performed total RNA-sequencing under the conditions of cell culture with and without ropinirole hydrochloride.
 
Contributor(s) Morimoto S, Takahashi S, Ito D, Daté Y, Okada K, Kato C, Nakamura S, Ozawa F, Chyi CM, Nishiyama A, Suzuki N, Fujimori K, Takao M, Hirai M, Kabe Y, Suematsu M, Jinzaki M, Aoki M, Fujiki Y, Sato Y, Suzuki N, Nakahara J, Okano H
Citation(s) 37267913, 38997748
Submission date Jul 25, 2022
Last update date Aug 02, 2024
Contact name Satoru Morimoto
Organization name Keio University
Department Keio Regenerative Medicine Research Center (KRM)
Lab Okano lab
Street address 3-25-10, Tonomachi, Kawasaki-ku, Kawasaki-shi
City Kanagawa
ZIP/Postal code 2100821
Country Japan
 
Platforms (1)
GPL24676 Illumina NovaSeq 6000 (Homo sapiens)
Samples (276)
GSM6383758 10KALS, iPSC-derived motor neurons from patient with ALS, non, 0h
GSM6383759 11KALS, iPSC-derived motor neurons from patient with ALS, non, 0h
GSM6383760 12KALS, iPSC-derived motor neurons from patient with ALS, non, 0h
Relations
BioProject PRJNA862121

Download family Format
SOFT formatted family file(s) SOFTHelp
MINiML formatted family file(s) MINiMLHelp
Series Matrix File(s) TXTHelp

Supplementary file Size Download File type/resource
GSE209696_processed_count_data.csv.gz 145.7 Mb (ftp)(http) CSV
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Raw data are available in SRA
Processed data are available on Series record

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