Cerebrolysin Improved Cognition in Vascular Dementia: A Randomized Controlled Trial

This was a multicenter, double-blind, placebo-controlled, randomized trial involving 242 patients meeting diagnostic criteria for vascular dementia. Patients received either intravenous cerebrolysin 20 mL or placebo once daily, administered over two treatment cycles as add-on therapy to aspirin. The primary endpoint was a combined measure of cognition (ADAS-cog+) and overall clinical functioning (CIBIC+) at 24 weeks.

Vascular dementia is the second most common form of dementia after Alzheimer's disease, yet no drug has been approved by the FDA or EMA to treat it. This leaves millions of patients without effective pharmacotherapy. This trial provides some of the strongest evidence that cerebrolysin — a neurotrophic peptide preparation — can meaningfully improve cognition and daily functioning in these patients, potentially filling a critical treatment gap.

Cerebrolysin has been used clinically in parts of Europe and Asia for decades, primarily for stroke recovery and various forms of dementia, though it lacks FDA approval. This trial is one of the larger, better-designed studies testing it specifically in vascular dementia. The robust effect sizes reported here — combined with cerebrolysin's known neurotrophic peptide content — contribute to the broader research into whether peptide-based neurotrophic therapies can address neurodegenerative conditions that currently have few treatment options.

The trial enrolled 242 patients, which is moderate but not large by modern standards. The study was conducted in European centers and may not generalize to all populations. Cerebrolysin requires daily intravenous infusions, which is a significant practical barrier compared to oral medications. The 24-week follow-up does not address whether benefits persist long-term. The manufacturer (EBEWE Pharma) was involved in the study, which could introduce bias.