Keio University

Investigator-Initiated Trial Based on iPS Cell Drug Discovery for Intractable Neurological Diseases Completed—Opening a New Door to the World of Amyotrophic Lateral Sclerosis (ALS) Treatment—

Publish: May 20, 2021
Public Relations Office

May 20, 2021

Keio University Hospital

Keio University School of Medicine

K Pharma, Inc.

A research group led by Professor Jin Nakahara, Chairman of the Department of Neurology at Keio University Hospital, and Professor Hideyuki Okano, Project Professor Shinichi Takahashi, and Project Lecturer Satoru Morimoto of the Keio University School of Medicine's Department of Physiology, conducted an investigator-initiated trial administering ropinirole hydrochloride to patients with amyotrophic lateral sclerosis (ALS), demonstrating its safety and efficacy.

In 2016, the same group discovered that ropinirole hydrochloride, a drug for Parkinson's disease, is effective against the pathology of ALS using iPS cells developed by Professor Shinya Yamanaka of Kyoto University. This clinical trial has now confirmed the drug's safety and efficacy in ALS patients, marking the world's first successful identification of a drug through iPS cell drug discovery that could potentially offer greater clinical disease progression-suppressing effects than existing medications.

Specifically, it was found that oral administration of a final dose of 16 mg of ropinirole hydrochloride could potentially delay the progression of the disease by 27.9 weeks (approximately 7 months) over a one-year trial period.

The results of this study suggest the potential for a new treatment option for ALS, a very severe disease for which effective therapies are scarce.

Please see below for the full press release.

Press Release (PDF)