Developing effective treatments for amyotrophic lateral sclerosis (ALS) remains a long-standing goal, especially for sporadic cases where no established therapeutic methodology currently exists.

Our research builds on a unique combination of resources: clinical and genomic data from a large-scale patient registry, patient-derived induced pluripotent stem (iPS) cells, and motor neurons generated from those cells. By integrating mathematical modeling and AI-based analysis, we aim to identify promising therapeutic targets—genes and molecules—and candidate drugs that can intervene in disease progression.

These candidates are then tested using motor neurons derived from ALS patients’ iPS cells, allowing us to evaluate their potential directly in disease-relevant human cells. Through this approach, we strive to establish a new paradigm for ALS drug development.