The iPS Cell Breakthroughs Reshaping Drug Discovery for Rare Disease
By modeling patients’ diseases in the laboratory and evaluating candidate drugs directly in human-derived cells, researchers like Okano Hideyuki of Keiō University are using iPS cells to open a new era in drug discovery. With Japan’s February 2026 announcement of a manufacturing system for an iPS cell-based therapy for spinal cord injury, the field is now entering a new phase of real-world…
Researchers at Keio University, led by Dr. Okano Hideyuki, are using induced pluripotent stem cells (iPS cells) to revolutionize drug discovery for rare diseases. These cells, derived from reprogrammed skin or blood cells, can differentiate into nearly any cell type, providing a direct link between basic research and drug development.
Dr. Okano's groundbreaking work primarily focuses on amyotrophic lateral sclerosis (ALS), a progressive and fatal neurodegenerative disease affecting roughly 10,000 people in Japan with no curative treatment or effective drugs currently available. Traditional mouse models used in drug development have failed to accurately replicate the disease, leading to repeated success in mice but failure in human clinical trials.
To overcome this barrier, Dr. Okano's team generated iPS cells from ALS patients and differentiated them into motor neurons, accurately reproducing the disease's characteristics. This breakthrough allowed for the testing of existing drugs on patient-specific cells, significantly improving the ALS motor neurons' abnormal phenotypes.
In 2018, Dr. Okano's team discovered that ropinirole hydrochloride, a Parkinson's medication, improved ALS motor neurons' abnormal features. This drug repositioning strategy has led to the launch of a clinical trial enrolling around 20 ALS patients. The research also accounts for patient-to-patient variability, generating iPS cells from over 250 ALS patients to analyze each cell line's characteristics and build multidimensional datasets.
Dr. Okano's ultimate goal is to create an "AI prescription" tailored to each patient's biology, potentially revolutionizing individualized drug treatments. In addition to drug discovery, Dr. Okano's team is conducting regenerative medicine trials using human iPS cell-derived neural precursor cells in patients with spinal-cord injuries.
Two of four patients showed marked improvement, and one regained enough function to begin walking training, marking the world's first clinical study using this approach. Japan's 2014 revision of the Pharmaceuticals and Medical Devices Act allows for the early provision of regenerative medicine products while additional evidence is gathered, creating a progressive framework globally.
Keio University established K Pharma to translate basic research into practical applications, driving social implementation of these advancements.
Written by urgent.news from Nippon.com News's reporting — not their text. Machine-written — may contain errors; check the original before relying on it.
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