April 24, 2018
Keio University School of Medicine
Keio University Hospital
Japan Agency for Medical Research and Development (AMED)
Professor Ikuo Ogawa and Senior Lecturer Masato Fujioka of the Department of Otorhinolaryngology, Head and Neck Surgery, Keio University School of Medicine, will conduct an investigator-initiated clinical trial of low-dose sirolimus therapy for hearing loss and dizziness in Pendred syndrome. This trial is based on findings from research using iPS cells, conducted in a joint study with the Department of Physiology (Professor Hideyuki Okano).
Pendred syndrome is a hereditary disease that causes progressive hearing loss, dizziness, and goiter, and is one of the rare and intractable diseases with no established treatment. Because genetically modified mice that can accurately reproduce these symptoms cannot be created, the research group has been conducting drug discovery research in preclinical studies by using patient-derived iPS cells to reproduce the disease pathology in vitro. For the first time, they are now planning to conduct a clinical trial based on findings from iPS cell-based drug discovery without using a disease animal model.
The therapeutic drug was selected by screening existing commercially available drugs, choosing one with an established safety profile for other diseases. Its effect on the inner ear in vivo was confirmed using normal animals, and its therapeutic effect at low doses was investigated using an iPS cell model. This drug discovery approach has the potential to be applied to other diseases for which no animal models that reproduce the disease have been established, and it is expected to be useful for the future development of treatments for intractable diseases.
In this clinical trial, patients will be provided with testing equipment and tablet devices to monitor symptoms such as hearing loss and dizziness, as well as changes in their physical condition, at home on a daily basis. By sending the test results from home to the clinical trial data center via remote access, a vast amount of trial data can be managed and analyzed.
By leveraging iPS cell technology and the Internet of Things (IoT) in a clinical setting and advancing efficient drug discovery while ensuring safety, we aim to deliver new therapeutic drugs to patients with intractable diseases that currently have no cure as quickly as possible.
Please see below for the full press release.