Novel Peptide Demonstrates Neuroprotective Potential in Traumatic Brain Injury

Researchers from the Spanish National Research Council (CSIC), part of the Ministry of Science, Innovation and Universities, have identified a promising new approach for treating traumatic brain injuries (TBI). Their study highlights the neuroprotective effects of a small peptide composed of four amino acids, called CAQK, which shows significant potential in reducing brain damage in animal models.

This peptide, when administered intravenously shortly after TBI, specifically targets injured areas of the brain by binding to proteins that are overexpressed following trauma. It accumulates in the damaged tissue and helps lessen inflammation, cell death, and overall tissue damage. The animal studies, conducted on both mice and pigs—whose brain structure closely resembles that of humans—demonstrated that CAQK reduces lesion size, diminishes neuroinflammation, and improves functional recovery without apparent toxicity.

The findings, published in EMBO Molecular Medicine, were led by the spin-off company Aivocode (linked to Sanford Burnham Prebys Institute) in collaboration with IQAC-CSIC and the University of California, Davis. The researchers plan to seek FDA approval to initiate Phase I clinical trials in humans soon. The short, easily synthesizable nature of CAQK gives it advantages for drug development, including good tissue penetration and low immunogenicity.

Traumatic brain injury, often caused by blows to the head during accidents or falls, affects an estimated 200 out of every 100,000 individuals annually. Currently, treatments mainly stabilize patients by controlling intracranial pressure, with no approved drugs that effectively halt the secondary damage such as inflammation and cell death. Existing therapies are invasive, requiring direct brain injections, which pose risks.

This breakthrough aligns with ongoing efforts to develop non-invasive treatments for brain injuries. The study builds on earlier research from 2016, where the same peptide was used as a delivery vehicle to target injured brain tissue. Importantly, the new research demonstrates that CAQK itself possesses therapeutic effects—reducing cell death and inflammation, and improving behavioral outcomes, all with a simple and scalable peptide structure.

The study underscores the potential for peptides like CAQK to become a new class of neuroprotective drugs, offering hope for more effective and less invasive treatments for TBI in the future.

source: https://medicalxpress.com/news/2025-10-peptide-neuroprotective-effects-traumatic-brain.html