Targeting a specific protein to help treat motor neuron disease
Official title: Targeting the EphA4 in motor neuron disease: a structure-based approach
Funded at University of California Riverside · NIH-11086636 · Data updated 2026-11-12
This study is looking at a specific part of the body called the EphA4 receptor to find new treatments for ALS, a serious disease that affects movement, with the hope that these new therapies can help slow down or stop the disease for patients.
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 | 2025 |
| Award (this year) | $397,551 |
| NIH project number | 5R01NS107479-08 |
| Project ID | NIH-11086636 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with amyotrophic lateral sclerosis (ALS), particularly those with specific genetic mutations related to the disease.
✕Less likely to help
Patients with other forms of motor neuron diseases or those without a diagnosis of ALS may not benefit from this research.
What this research studies
This research focuses on understanding and targeting the EphA4 receptor, which is believed to play a significant role in the progression of amyotrophic lateral sclerosis (ALS), a severe motor neuron disease. The study aims to develop and optimize new therapeutic agents that can effectively interact with this receptor, potentially leading to better treatment options for ALS patients. By using advanced techniques to analyze how these agents work in cellular and animal models, the research seeks to uncover the mechanisms behind motor neuron death and identify effective pharmacological tools. Patients may benefit from this research as it could lead to novel therapies that slow down or halt the progression of ALS.
Why it matters
Potential benefit: If successful, this research could provide new therapeutic options that significantly improve the quality of life for ALS patients.
How similar studies have gone: While targeting the EphA4 receptor is a relatively novel approach, previous studies have shown promise in using similar strategies to develop therapies for neurodegenerative diseases.
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.
-
Blocking EphA4 to protect motor neurons in ALS
This project develops and refines drug-like molecules that target the EphA4 protein to try to protect motor neurons in people with ALS.
-
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.
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.