Drug Discovery Related to iPS Cells

Ropinirole (Requip®) Ropinirole

Action: A drug discovered through research on ALS patient-derived iPS cells. Originally, it was widely administered as a treatment for Parkinson’s disease.

Clinical trial results: It reduced ALSFRS-R during phase I/II in the double-blind period; however, the difference was not statistically significant.


Action: The drug was discovered by high-throughput screening of ALS patient-derived iPS cells. Originally, this drug was a dopamine agonist and was widely administered to patients with Parkinson’s disease. It ameliorates mitochondrial dysfunction by inhibiting the accumulation of abnormal TDP-43 and FUS proteins. However, this is not effective for ALS-iPS with SOD1 mutations.

Previous results: Phase I/III: 13 patients on the active drug and 7 patients on the placebo drug.

ALSFRS-R showed a 5.9±4.1 points decline in the ropinirole group and a 15.6±8.8 decline in the placebo group during the double-blind period (6 months); however, this was not statistically significant. (Morimoto et al., 2022)

Dosage: Oral administration

Side effects: gastrointestinal symptoms, drowsiness, leg edema

Bosutinib (Bosulif®) Bosutinib

Action: A drug discovered in the study of ALS patient-derived iPS cells. Originally this drug was used for chronic myeloid leukemia.

Clinical trial results: In 5 among 9 patients, the decline in ALSFRS-R stopped after 12 weeks of phase I treatment.

Current status: Phase II trial started. No recruitment.


Action: Induces the removal of abnormally accumulated proteins via autophagy. This drug was discovered through research on ALS-patient-derived iPS cells. This drug is effective for ALS patients with SOD1 mutations.

Past clinical trial results: In phase I, three of nine patients with ALS discontinued treatment due to adverse events (Diarrhea, liver dysfunction).

After 12 weeks of treatment, in 5 among 9 patients, the decline (progressing) in the ALSFRS-R scores stopped. Patients with suppressed ALSFRS-R decline had low blood neurofilament L (a marker of neuropathy) at baseline.(Keiko Imamura et al., 2022)

Current status: Phase II open-label multicenter trial has started in Japan; the data will be compared with external data from previous ALS clinical trials and JaCALS*. Target number of patients: 25

No recruitment

Dosage: Oral administration

Side effects: diarrhea, liver dysfunction, etc. 




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