December 3, 2018
Keio University School of Medicine
Keio University Hospital
Japan Agency for Medical Research and Development (AMED)
Professor Jin Nakahara, Head of the Department of Neurology at Keio University Hospital, and Associate Professor Shinichi Takahashi, Deputy Head of the Department, in collaboration with Professor Hideyuki Okano of the Department of Physiology, Keio University School of Medicine, have launched a Phase I/IIa investigator-initiated clinical trial. This trial aims to evaluate the safety and efficacy of ropinirole hydrochloride (the investigational drug), a new candidate drug for ALS treatment discovered by applying drug discovery technology using disease-specific iPS cells. The trial is scheduled to be conducted at Keio University Hospital for patients with ALS, a condition for which effective treatments are limited.
The investigational drug has been widely used worldwide as a treatment for Parkinson's disease since 1996. Extensive experience with its use has also been accumulated in Japan, and a version that provides a sustained effect with once-daily administration (a prolonged-release tablet) is available. This clinical trial will use these prolonged-release tablets, which are less burdensome for patients.
Professor Okano's group has been conducting research for many years, establishing iPS cells from tissues derived from patients with neurological diseases (disease-specific iPS cells). While research on ALS has been challenging due to the lack of appropriate disease models for animal experiments, Professor Okano's group has developed an innovative drug screening method using iPS cell models derived from patients, leading to the identification of a promising candidate drug for ALS treatment.
This clinical trial is expected to bring great hope to patients with ALS, a severe and intractable neurological disease for which effective treatments have been scarce.
For the full press release, please see below.