Using patient-derived cells to discover new treatments for ALS
Leveraging natural phenotypic variations of heterogenous ALS populations-in-a-dish to enable scalable drug discovery
This study is looking at the different genetic reasons behind ALS by using special nerve cells made from patients' own stem cells, and it hopes to find new treatments that work for everyone, especially for those with unknown causes of the disease.
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-10907756 on NIH RePORTER |
What this research studies
This research focuses on understanding the diverse genetic causes of Amyotrophic Lateral Sclerosis (ALS) by utilizing induced motor neurons derived from patient-specific stem cells. The team will employ a novel platform called GENEVA, which allows for the assessment of drug responses across multiple patient lines simultaneously. By identifying effective therapeutic targets, the research aims to develop scalable treatment strategies that can address the varying forms of ALS, particularly those with unknown genetic causes.
Who could benefit from this research
Good fit: Ideal candidates for this research include ALS patients with diverse genetic backgrounds, especially those without known mutations.
Not a fit: Patients with ALS who have specific known mutations that are already being targeted by existing therapies may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the development of new, personalized therapies for ALS patients, improving treatment options and outcomes.
How similar studies have performed: Previous research using similar patient-derived models has shown promise in identifying effective treatments for other diseases, suggesting potential success for this novel approach in ALS.
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.