2023/06/02
Keio University School of Medicine
Keio University Hospital
K Pharma, Inc.
A research group led by Professor Hideyuki Okano, Project Professor Shinichi Takahashi, and Project Lecturer Satoru Morimoto of the Department of Physiology, Keio University School of Medicine, and Professor Jin Nakahara, Head of the Department of Neurology at Keio University Hospital, conducted the ROPALS trial, an investigator-initiated clinical trial (translational research) administering ropinirole hydrochloride (ropinirole) to patients with amyotrophic lateral sclerosis (ALS), and has clarified its safety and efficacy. Furthermore, they succeeded in predicting the drug's efficacy by creating iPS cells from all trial participants and administering ropinirole to the patient-derived differentiated cells (reverse translational research). They also discovered that ropinirole exerts its anti-ALS effect by regulating cholesterol synthesis within nerve cells.
In 2016, the same group discovered that ropinirole, a drug for Parkinson's disease, is effective against the pathology of ALS, using iPS cells invented by Professor Shinya Yamanaka of Kyoto University. This clinical trial has now confirmed the drug's safety and efficacy in ALS patients. They have succeeded for the first time in the world in identifying a drug that can produce a greater clinical effect in suppressing disease progression than existing drugs through iPS cell drug discovery. The findings were published on June 2, 2023 (JST) in Cell Stem Cell (Cell Press), a prominent international scientific journal on research using iPS cells and other stem cells.
Specifically, taking a final dose of 16 mg of ropinirole may potentially delay the progression of the disease by 27.9 weeks (approximately 7 months) over a one-year trial period. This result was also supported by verification using data from an international patient registry for ALS clinical trials.
They also identified candidate surrogate markers for determining the efficacy of ropinirole. This is an important finding for conducting future clinical trials.
Furthermore, by using patient-derived iPS cell models, they have discovered the potential to evaluate the efficacy of a drug for each individual patient, marking a milestone in the realization of tailor-made medicine using iPS cells that contributes to appropriate medication.
The results of this research have demonstrated the utility of iPS cell drug discovery, suggesting the potential for a new treatment option for the intractable neurological disease ALS, for which effective therapies are scarce.
For the full press release, please see below.