Using patient stem cells to discover better ALS treatments
Leveraging Natural Phenotypic Variations of Heterogenous ALS Populations-in-a-Dish to Enable Scalable Drug Discovery
Researchers are looking for medicines that help different types of ALS by studying nerve cells made from patients' stem cells all together in one experiment.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Southern California NIH-funded |
| Lab location | 1 site (Los Angeles, UNITED STATES) |
| Project ID | NIH-11160503 on NIH RePORTER |
What this research studies
This project grows motor nerve cells from ALS patients' blood or skin samples and mixes dozens of patient lines in a single dish so a new platform called GENEVA can read how each line reacts to many drugs using single-cell gene readouts and genetic barcodes. By pooling lines, the team can test far more patient samples at once while keeping costs and lab work manageable. The approach aims to find treatments that help specific patient subgroups or that work broadly across many forms of ALS, including cases without a known genetic cause. Donating a sample could help researchers spot which drugs are promising for which kinds of ALS more quickly.
Who could benefit from this research
Good fit: People with ALS who are willing to provide a blood or skin sample (for making patient stem cells) would be the ideal candidates to contribute to this work.
Not a fit: People who cannot or do not want to give biological samples, or who need immediate clinical treatment, may not receive direct benefit from this lab-focused research.
Why it matters
Potential benefit: This work could speed up finding drugs that benefit particular groups of people with ALS or that work across many ALS types.
How similar studies have performed: Prior uses of patient-derived cell models and single-cell readouts have shown promise in cancer and early ALS experiments, but applying pooled single-cell screening to many ALS patient lines is a newer, less-tested approach.
Where this research is happening
Los Angeles, UNITED STATES
- University of Southern California — Los Angeles, United States (Active)
Researchers
- Principal investigator: Ichida, Justin Kawika — University of Southern California
- Study coordinator: Ichida, Justin Kawika
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.