INTRODUCTION: Neurodegenerative and demyelinating diseases represent major unmet medical needs, with existing therapeutics demonstrating limited efficacy and significant safety concerns. Drug repurposing offers accelerated development timelines and established safety profiles. GV1001, a 16-amino acid telomerase-derived peptide originally developed as a cancer vaccine, has emerged as a promising multi-mechanism neuroprotective agent. AREAS COVERED: This review examines preclinical evidence demonstrating GV1001 efficacy across Alzheimer's disease, stroke, experimental autoimmune encephalomyelitis, and depression models. Convergent mechanisms include gonadotropin-releasing hormone receptor-mediated neuroprotection, mitochondrial stabilization, modulation of glial functional states away from tissue-damaging inflammation, and promotion of remyelination. Clinical translation through Phase 2 trials in Alzheimer's disease demonstrated statistically significant cognitive improvements consistent with neurotrophic benefit, with excellent safety profiles lacking amyloid-related imaging abnormalities. EXPERT OPINION: The network pharmacology approach of GV1001-simultaneously addressing multiple pathological processes - positions it as a differentiated alternative to single-target therapeutics. The dual anti-inflammatory and pro-remyelination profile of the peptide addresses critical unmet needs in progressive multiple sclerosis. Phase 3 confirmation, biomarker-driven patient stratification, and combination therapy investigations represent critical development priorities. Successful development may help validate multi-mechanism approaches in neurodegeneration, potentially catalyzing paradigm shifts from reductionist single-target strategies.
Suk et al. (Thu,) studied this question.