Blocking EphA4 to protect motor neurons in ALS

Targeting the EphA4 in motor neuron disease: a structure-based approach

['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA RIVERSIDE · NIH-11331257

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

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorUNIVERSITY OF CALIFORNIA RIVERSIDE (nih funded)
Locations1 site (RIVERSIDE, UNITED STATES)
Trial IDNIH-11331257 on ClinicalTrials.gov

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.

Who could benefit from this research

Good fit: 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.

Not a fit: People without ALS, or those with very advanced ALS, are unlikely to receive direct benefit from this preclinical research.

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 performed: 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

RIVERSIDE, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Conditions: Amyotrophic Lateral Sclerosis Motor Neuron Disease, Animal Disease Models

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.