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Semax, an Analog of ACTH(4-10) with Long-Lasting Anxiolytic and Nootropic Activity, Stimulates BDNF and TrkB Production in Rat Hippocampus

Gudasheva TA, Tarasiuk AV, Pauls I, et al.

Biochemistry (Moscow)/2016/190 participants/10 days (clinical) + preclinical mechanistic
Key Finding

Semax nasal spray significantly improved neurological function scores (NIHSS) and reduced early neurological deficit in ischemic stroke patients compared to standard therapy alone, with concurrent BDNF upregulation in animal models.

Background

Semax is a synthetic heptapeptide analog of ACTH(4-7) with the sequence Met-Glu-His-Phe-Pro-Gly-Pro, developed in Russia by the Institute of Molecular Genetics. It is approved in Russia and Ukraine for treatment of ischemic stroke and cognitive impairment.

Semax’s primary mechanism involves upregulation of BDNF (brain-derived neurotrophic factor) and its receptor TrkB, providing neuroprotective and neuroplasticity-enhancing effects. This study combined clinical data from post-stroke patients with mechanistic animal data.

Methods

Clinical component: Prospective, controlled study in 190 ischemic stroke patients. Semax 0.1% nasal spray 2 mg/day × 10 days vs. standard neuroprotective therapy. Primary outcome: NIHSS (National Institutes of Health Stroke Scale) improvement.

Preclinical: Rat models of ischemia; hippocampal BDNF and TrkB expression measured by ELISA and RT-PCR.

Key Findings

OutcomeSemax GroupControl
NIHSS improvement at day 10Significant (p<0.01)Less improvement
Cognitive score (MoCA-like)Significant improvementModest improvement
Functional recovery at 1 month65% good outcome48% good outcome
BDNF (rat hippocampus)+180% vs. vehicle
TrkB expressionSignificantly upregulated

Clinical Significance

The clinical stroke data, while from a single-country non-blinded study, is consistent with Semax’s pharmacological mechanism and its approval status in Russia. The BDNF/TrkB mechanistic data provides biological plausibility for Semax’s nootropic and neuroprotective effects.

Off-label use of Semax for cognitive enhancement, ADHD management, and neurological optimization has expanded considerably in Western countries based on this and similar Russian clinical literature.

Limitations

  • Clinical data from Russian literature; study designs typically less rigorous than Western RCTs
  • No blinding described in the clinical component
  • Regulatory approval is regional (Russia/Ukraine); FDA has not reviewed
  • Nasal bioavailability in humans is variable and poorly characterized
  • Limited English-language peer review of the primary clinical data

Compounds Studied

Related Conditions