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 are revolutionizing drug discovery for rare diseases through the use of induced pluripotent stem cells (iPS cells). These artificial cells can be made to differentiate into any cell type, providing an invaluable tool for modeling neurodegenerative diseases and testing potential treatments directly in human cells.
Hideyuki Okano, director of the Keio University Regenerative Medicine Research Center, has pioneered this approach, particularly for amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease that currently has no cure. Traditionally, ALS research relied on limited mouse models, which often yielded ineffective treatments in human clinical trials due to fundamental differences between mouse and human neurons.
Okano's team addressed this by generating iPS cells from ALS patients and differentiating them into motor neurons, resulting in a highly accurate model of the disease. Using this model, they successfully identified ropinirole, a Parkinson's medication, as a potential ALS treatment. In 2018, they discovered that ropinirole improved the phenotypes of ALS motor neurons, leading to an early clinical trial enrolling around 20 patients.
The rapid translation of lab-based discoveries into clinical trials is a significant advantage of using iPS cell-based research. Okano's team is also tackling patient-to-patient variability by generating iPS cells from hundreds of ALS patients and analyzing their characteristics. Their long-term goal is to use AI to develop personalized drug treatments for each patient, addressing the unique genetic makeup and disease progression of individual patients.
In addition to drug discovery, Okano's team is also conducting regenerative medicine trials using human iPS cell-derived neural precursor cells to treat spinal cord injuries. Initial results show promising improvements in mobility for patients with severe quadriplegia, and the team hopes to secure conditional approval for their therapy within the next few years.
Japan's progressive regulatory framework for regenerative medicine, established in 2014, allows for early provision of these products while additional evidence is gathered, enabling real-world use and commercialization. As the head of K Pharma, a biotech startup founded by Keio University, Okano is also working on manufacturing and marketing these innovative therapies, paving the way for their broader implementation in Japan.
Written by urgent.news from Nippon.com's reporting — not their text. Machine-written — may contain errors; check the original before relying on it.
