Blocking EphA4 to protect motor neurons in ALS
Official title: Targeting the EphA4 in motor neuron disease: a structure-based approach
Funded at University of California Riverside · NIH-11331257 · Data updated 2026-11-12
This project develops and refines drug-like molecules that target the EphA4 protein to try to protect motor neurons in people with ALS.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Riverside NIH-funded |
| Lab location | 1 site (Riverside, United States) |
| Project start | July 1, 2018 |
| Project end | April 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $397,551 |
| NIH project number | 5R01NS107479-09 |
| Project ID | NIH-11331257 on NIH RePORTER |
Who could benefit from this research
✓Could help
People diagnosed with ALS, particularly those with SOD1 or chromosome 9–related mutations or in earlier stages of the disease, would be the most likely candidates for future trials based on this work.
✕Less likely to help
People without ALS, or those with very advanced ALS, are unlikely to receive direct benefit from this preclinical research.
What this research studies
You would be hearing about work that tries to turn lab findings into possible medicines for ALS. Researchers are optimizing small molecules that act on the EphA4 receptor and can enter the brain. They will test these compounds in cell cultures and animal models of ALS to see whether they protect motor neurons and clarify how they work. The data will guide whether these drug candidates should move toward future human testing.
Why it matters
Potential benefit: If successful, this work could lead to new medicines that slow nerve damage and the progression of ALS.
How similar studies have gone: Earlier laboratory and animal studies of EphA4-targeting agents showed protection in cells and animal models, but benefit in people has not yet been shown.
Where this research is happening
-
University of California Riverside
Riverside, United States Active
Researchers
- Principal investigator: Pellecchia, Maurizio — University of California Riverside
- Study coordinator: Pellecchia, Maurizio
Related research
Other NIH-funded projects on the same topics.
-
T cell roles in a rare juvenile ALS form (SETX-related)
A study to understand how immune cells, especially CD8 T cells, influence a rare juvenile form of ALS caused by SETX mutations.
-
Investigating how motor neurons contribute to premature death in spinocerebellar ataxia type 1
A study at Baylor College of Medicine is exploring why people with spinocerebellar ataxia type 1 also develop muscle weakness and breathing problems, and how motor neurons are involved.
-
Understanding how sphingolipid changes cause immune-nerve damage in ALS
A Johns Hopkins team tests how abnormal lipid production may drive motor neuron disease in people with ALS, using patient-derived cells and mouse models.
-
Early causes of frontotemporal dementia and motor neuron disease
Researchers are using donated brain tissue from people with genetic and non-genetic frontotemporal dementia and motor neuron disease to find early changes that lead to TDP-43 protein problems.
-
Motor neuron causes of weakness and breathing problems in spinocerebellar ataxia type 1
Seeing if damage to motor neurons explains muscle weakness, trouble breathing and early death in people with spinocerebellar ataxia type 1.
-
T cells in a rare juvenile form of ALS (ALS4)
This research looks at whether certain CD8 T immune cells are linked to ALS4, a rare juvenile form of ALS, by studying mouse models and samples from people with ALS.
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.