Human nerve–muscle junctions grown from patient stem cells to model ALS and related neuromuscular diseases
Official title: Human-iPSC derived neuromuscular junctions as a model for neuromuscular diseases.
Funded at Case Western Reserve University · NIH-11082176 · Data updated 2026-11-12
They are growing human nerve-to-muscle connections from patient stem cells to help develop better treatments for people with ALS and similar neuromuscular conditions.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Case Western Reserve University NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project start | September 8, 2023 |
| Project end | July 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $390,007 |
| NIH project number | 4R33NS133212-02 |
| Project ID | NIH-11082176 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults (21+) with ALS or related neuromuscular disorders who can provide a blood or skin sample for iPSC generation would be ideal candidates to contribute samples.
✕Less likely to help
People with conditions unrelated to neuromuscular junction dysfunction or those seeking an immediate clinical therapy may not benefit, because this is a lab-model development project rather than a treatment trial.
What this research studies
This project uses stem cells from adults to make motor neurons and muscle cells in the lab, then joins them to form functioning human neuromuscular junctions (NMJs). If you donate a blood or skin sample, researchers can turn your cells into these lab-grown NMJs to see how disease affects nerve–muscle communication. The team will use these human NMJ systems to test biological targets and screen candidate therapies in a setting that more closely mimics human tissue than animal models. The goal is to find approaches that are more likely to work in patients before moving to clinical trials.
Why it matters
Potential benefit: If successful, the work could lead to more reliable ways to find drugs that actually work in people with neuromuscular diseases and reduce treatments that fail in clinical trials.
How similar studies have gone: Other groups have built human iPSC-derived NMJ models and shown basic function, but translating those models into reliable patient treatments has not yet been widely proven.
Where this research is happening
-
Case Western Reserve University
Cleveland, United States Active
Researchers
- Principal investigator: Miranda, Helen C — Case Western Reserve University
- Study coordinator: Miranda, Helen C
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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.