Cerebrolysin: Neuropeptide Research Overview
Every year, roughly 15 million people worldwide suffer a stroke. Millions more live with Alzheimer's disease, traumatic brain injuries, or other conditions where the brain's own repair systems fall short.
Every year, roughly 15 million people worldwide suffer a stroke. Millions more live with Alzheimer's disease, traumatic brain injuries, or other conditions where the brain's own repair systems fall short. For decades, researchers have chased a simple idea: what if you could give the brain a concentrated dose of the same growth-promoting molecules it uses to heal itself?
Cerebrolysin is the most studied attempt at that idea. Derived from pig brain tissue and containing fragments of natural neurotrophic factors, this injectable peptide mixture has been used in over 50 countries since the 1970s. More than 200 clinical trials involving upward of 15,000 patients have tested it. Yet it remains unapproved in the United States, and the FDA has never signed off on it for any condition.
That gap tells you something about Cerebrolysin's complicated story. The clinical data ranges from genuinely promising to deeply ambiguous, and recent research integrity controversies have cast a shadow over some of the preclinical work. This article walks through what we actually know.
Table of Contents
- Quick Facts
- What Is Cerebrolysin?
- Development History
- How Cerebrolysin Works: Mechanisms of Action
- Research Evidence
- Administration and Dosing
- Safety Profile and Side Effects
- Legal and Regulatory Status
- Limitations of Current Research
- Frequently Asked Questions
- The Bottom Line
Quick Facts
| Detail | Information |
|---|---|
| Full Name | Cerebrolysin (FPF-1070) |
| Other Names | Cerebrolysin®, FPF-1070 |
| Type | Multi-peptide neurotrophic preparation |
| Composition | ~75% free amino acids, ~25% low-molecular-weight peptides, plus trace minerals (magnesium, potassium, calcium, selenium, zinc) |
| Molecular Weight | Peptide components below 10,000 daltons |
| Origin/Manufacturer | EVER Neuro Pharma (Unterach, Austria); derived from enzymatic hydrolysis of porcine (pig) brain tissue |
| First Described | 1949 by Gerhart Harrer at the University of Innsbruck, Austria |
| Primary Use | Stroke recovery, traumatic brain injury, dementia (Alzheimer's and vascular) |
| Administration | Intramuscular (IM) injection or intravenous (IV) infusion |
| FDA Status | Not approved in the United States for any indication |
| International Status | Approved as a prescription medication in over 50 countries, primarily in Europe and Asia |
What Is Cerebrolysin?
Cerebrolysin is an injectable mixture of peptides and amino acids produced by breaking down purified pig brain proteins through a controlled enzymatic process. The name itself tells you what it is: "cerebro" (brain) + "lysin" (lysis, or enzymatic breakdown).
The finished product is a clear, amber-colored solution. About 75% of its content by weight consists of free L-amino acids. The remaining 25% is a mixture of short peptides — protein fragments small enough (under 10,000 daltons) to cross the blood-brain barrier. Most large proteins can't get past the brain's protective filtering system. Cerebrolysin's fragments are small enough to slip through.
Those peptide fragments contain active portions of several neurotrophic factors — proteins the brain naturally uses to grow, maintain, and repair neurons. Researchers have identified fragments associated with brain-derived neurotrophic factor (BDNF), glial cell line-derived neurotrophic factor (GDNF), nerve growth factor (NGF), ciliary neurotrophic factor (CNTF), and insulin-like growth factors 1 and 2 (IGF-1, IGF-2). The preparation also contains trace minerals including magnesium, phosphorus, potassium, and selenium.
Think of it as a concentrated broth of the brain's own repair signals, filtered down to fragments small enough to reach neurons when injected. It's not a single synthetic molecule like most drugs, but a complex biological mixture — closer in concept to a tissue extract than to a traditional pharmaceutical. This makes it different from synthetic nootropic peptides like Semax or Selank, which are single, well-defined peptide sequences.
Development History
The story starts in 1949 in Innsbruck, Austria. Gerhart Harrer, a neurologist at the University of Innsbruck, hypothesized that the brain's own growth and repair factors could be extracted and given to patients with brain injuries. He developed a process to enzymatically break down brain tissue into a filtrate of small peptides and amino acids. The preparation received initial approval in Austria on August 1, 1954.
Early clinical use through the 1950s and 1960s was limited — mostly anecdotal reports from Austrian and German clinics. The modern development phase began in the 1970s when EVER Neuro Pharma (originally Emil Bertalanffy Werke, or EBEWE), an Austrian pharmaceutical company, took over production and standardization. They industrialized the manufacturing process, ensuring batch-to-batch consistency. Formal clinical studies began around 1973.
Through the 1980s and 1990s, Cerebrolysin gained approval across Russia, Eastern Europe, China, and other Asian nations. Russian neurologists adopted it widely for stroke and dementia. By the 2000s, larger randomized controlled trials appeared, and the CAPTAIN trial series in traumatic brain injury (2015–2021) brought the most rigorous investigation to date. As of 2026, Cerebrolysin is used in over 50 countries but has never received FDA approval.
How Cerebrolysin Works: Mechanisms of Action
Cerebrolysin doesn't work through a single mechanism. It's a multi-target biological mixture, and researchers have identified several overlapping pathways it appears to affect.
Neurotrophic Factor Mimicry and Upregulation
The primary mechanism. Cerebrolysin's peptide fragments mimic the activity of the brain's own neurotrophic factors — BDNF, NGF, GDNF, and CNTF. A 2023 review in Medicinal Research Reviews documented that Cerebrolysin both mimics these factors directly and stimulates endogenous production. It upregulates BDNF, VEGF, IGF-1, and NGF while downregulating TNF-alpha, an inflammatory cytokine.
One telling detail: a study comparing Cerebrolysin's peptide fraction against an equivalent mixture of free amino acids found that BDNF-like effects lasted eight days with the peptides but only four days with amino acids alone — confirming that the therapeutic activity comes from the peptide fragments, not just the amino acid content.
Neuroprotection
Cerebrolysin activates pro-survival signaling, particularly the PI3K/Akt pathway, which prevents programmed cell death (apoptosis). In stroke and brain injury models, it reduces free radical damage, inhibits excitotoxicity (damage from glutamate overstimulation), and suppresses inflammatory cascades via the TLR/NF-kB pathway. Other peptides with neuroprotective properties include BPC-157 and Epitalon, though they work through different pathways.
Neurogenesis and Synaptic Plasticity
In animal models of Alzheimer's disease, Cerebrolysin promoted the growth of new neurons in the hippocampus and increased synapse formation. It appears to shift neural progenitor cells from a glial (support cell) fate toward a neuronal fate — meaning more of the brain's stem cells develop into functional neurons. It also modulates the Sonic Hedgehog (Shh) signaling pathway, involved in stem cell differentiation and tissue repair. GHK-Cu research has explored similar neuroregeneration pathways through different mechanisms.
Anti-Amyloid Effects
In transgenic mouse models of Alzheimer's disease, Cerebrolysin reduced amyloid-beta deposits — the protein plaques that accumulate in Alzheimer's brains. It appears to do this by regulating how the amyloid precursor protein (APP) is processed, specifically by modulating the kinases GSK3-beta and CDK5, which influence APP phosphorylation. Whether this translates to meaningful clinical benefit in humans remains an open question.
Anti-Inflammatory Effects
Cerebrolysin suppresses neuroinflammation by reducing pro-inflammatory cytokines, inhibiting microglial activation, and modulating the NF-kB signaling cascade. In stroke models, this anti-inflammatory action reduces secondary damage — the ongoing injury that continues for days after the initial blood flow interruption.
Research Evidence
Cerebrolysin has been studied in over 200 clinical trials. Here is what the strongest evidence shows for each major indication.
Acute Ischemic Stroke
This is the most extensively studied application, and the evidence is mixed.
In favor: A 2025 meta-analysis of 14 RCTs (2,884 patients) found Cerebrolysin significantly improved neurological recovery, with a mean NIHSS improvement of 1.39 points over placebo (p = 0.020). Bornstein et al. (2018) found similar results across nine RCTs (N = 1,879), with a number needed to treat of 7.7 for clinically relevant improvements.
Against: The 2023 Cochrane review — the gold standard for evidence synthesis — analyzed seven RCTs (1,773 participants) and concluded that Cerebrolysin "probably results in little to no difference in all-cause death" (RR 0.96, moderate-certainty evidence). The reviewers noted a potential increase in non-fatal serious adverse events and flagged that none of the included trials reported on functional outcomes like death-or-dependence or quality of life.
The disconnect largely comes down to endpoints. Studies measuring NIHSS scores (neurological deficit) tend to favor Cerebrolysin; studies looking at mortality and functional independence are less encouraging.
Traumatic Brain Injury
The CAPTAIN trial series — two phase IIIb/IV randomized, double-blind, placebo-controlled trials — is the strongest evidence here.
CAPTAIN I (46 patients) found statistically significant superiority of Cerebrolysin in the per-protocol analysis (p = 0.024), though the intention-to-treat analysis just missed significance. CAPTAIN II (139 patients, 2020) showed a small-to-medium effect at day 90 (MW = 0.59, p = 0.012). A retrospective analysis also found Cerebrolysin significantly reduced post-TBI anxiety, with a large effect size of 0.73.
A 2023 meta-analysis pooling ten studies (8,749 patients) found statistically significant improvements in GCS and GOS scores with Cerebrolysin. However, high heterogeneity between studies and the lack of large standardized RCTs weakened the certainty of these conclusions.
Alzheimer's Disease
Multiple randomized, double-blind, placebo-controlled trials have tested Cerebrolysin in mild-to-moderate Alzheimer's disease.
Panisset et al. (2002) randomized 192 patients to receive 30 mL Cerebrolysin or placebo IV for four weeks. The Cerebrolysin group showed significant improvement on the CIBIC+ (global clinical impression) at week 12 (p = 0.033), with 76% classified as responders versus 57% on placebo (p = 0.007). However, ADAS-cog scores — the standard cognitive measure — did not reach statistical significance.
A dose-finding study of 279 patients tested three doses (10 mL, 30 mL, 60 mL). At week 24, the 10 mL dose produced significant improvement on both ADAS-cog (p = 0.038) and CIBIC+ (p < 0.001). Interestingly, the higher doses did not perform better.
A combination trial comparing Cerebrolysin, donepezil (Aricept), and both together in 197 patients found Cerebrolysin roughly equivalent to donepezil, with the combination showing numerically (but not statistically significantly) better outcomes.
The 2015 meta-analysis by Gauthier et al. pooled the available RCTs and found Cerebrolysin significantly better than placebo for cognition at 4 weeks (SMD -0.40, p = 0.003) and for global clinical change at both 4 weeks (OR 3.32, p = 0.021) and 6 months (OR 4.98, p = 0.015). For comparison, other peptides investigated for cognitive support, such as Dihexa and PE-22-28, remain largely in the preclinical phase, making Cerebrolysin one of the more clinically tested options in this space. For a broader look at this category, see our guide to the best peptides for cognitive improvement.
Vascular Dementia
Guekht et al. (2011) conducted a multicenter, double-blind trial of 242 patients with vascular dementia. Results were strongly positive: ADAS-cog+ improved by 10.6 points with Cerebrolysin versus 4.4 points with placebo (p < 0.0001). The CIBIC+ global score also improved significantly (p < 0.0001). Response rates on the combined endpoint were 67.5% for Cerebrolysin versus 27.0% for placebo — an odds ratio of 5.63 in favor of treatment.
However, a Cochrane review on Cerebrolysin for vascular dementia rated the overall evidence quality as "very low" due to imprecision, indirectness, and serious risk of bias across the available studies.
Psychiatric Applications
A 2025 scoping review explored Cerebrolysin's potential in psychiatry, noting preliminary evidence for schizophrenia, depression, and associated cognitive deficits. The evidence is thin — mostly small studies and case series — but neurotrophic factor deficiency is implicated in both conditions, giving researchers reason to look further.
CADASIL (Cerebral Autosomal Dominant Arteriopathy)
A 2024 preclinical study in a mouse model of CADASIL — a rare genetic disorder causing recurring strokes and dementia — found that Cerebrolysin improved spatial memory, reduced epigenetic aging markers, and extended lifespan. It did not, however, affect the disease's characteristic white matter damage.
Administration and Dosing
Cerebrolysin must be given by injection. It is not orally bioavailable — the peptides would be digested in the gut before reaching the bloodstream.
Intramuscular (IM) injection: Up to 5 mL can be injected undiluted, slowly over approximately 3 minutes. Typically used for milder cognitive support protocols.
Intravenous (IV) infusion: The more common clinical route. Doses up to 10 mL can be given as a slow IV push over 3 minutes. Doses of 10–50 mL should be diluted in normal saline or 5% glucose and infused over 15–60 minutes.
Dosing by clinical indication (as used in research):
- Stroke: 15–50 mL daily via IV infusion, typically for 10–21 days. The 2025 meta-analysis of 14 RCTs used doses ranging from 10 to 50 mL across studies.
- Traumatic brain injury: The CAPTAIN trials used 50 mL daily for 10 days, followed by two additional cycles of 10 mL daily for 10 days each.
- Alzheimer's disease: 10–30 mL daily via IV infusion. Studies have used courses of 5 days per week for 4 weeks, sometimes repeated. The dose-finding study suggested 10 mL may be as effective as higher doses.
- Vascular dementia: 20 mL daily via IV infusion over two treatment cycles.
- Cognitive support (off-label): Some practitioners use 5 mL IM daily for approximately four weeks.
Treatment cycles: Most protocols involve 10–30 day courses, repeated with breaks in between. Morning administration is preferred because Cerebrolysin has a stimulating effect that may interfere with sleep.
Safety Profile and Side Effects
Across the clinical trial database, Cerebrolysin's side effect profile has generally been mild. In controlled trials, the rate of adverse events has been comparable between Cerebrolysin and placebo groups.
Common side effects (reported in clinical trials):
- Headache
- Dizziness or vertigo
- Nausea
- Sweating
- Feeling hot or flushed
- Agitation
- Weight loss (with extended use)
These effects are usually transient and mild.
Less common side effects:
- Injection site reactions
- Blood pressure changes (both increases and decreases have been reported)
- Increased heart rate or arrhythmia — typically related to IV infusion speed rather than the drug itself
- Rare reports of confusion or hallucinations
Serious adverse events: A 2021 safety meta-analysis of 12 RCTs (2,202 patients) found no statistically significant difference in serious adverse events between Cerebrolysin and placebo. However, the 2023 Cochrane review flagged moderate-certainty evidence of a potential increase in non-fatal serious adverse events requiring hospitalization. This remains a point of genuine disagreement in the literature.
Contraindications:
- Epilepsy — Cerebrolysin is contraindicated in patients with epilepsy or status epilepticus due to potential seizure risk.
- Severe kidney disease — The drug is contraindicated in severe renal impairment, and kidney function should be monitored during treatment.
- Hypersensitivity — Known allergy to any component of the preparation.
- Pregnancy and breastfeeding — Not recommended unless benefits clearly outweigh risks.
Animal-derived product risk: Because Cerebrolysin comes from pig brain tissue, there is a theoretical risk of contamination with prions or other biological agents. Manufacturing includes mitigation steps, but this remains a regulatory consideration.
Legal and Regulatory Status
United States: Not FDA-approved for any indication. It cannot be legally prescribed or dispensed by a pharmacy. It exists in a gray area where some research suppliers sell it under "not for human consumption" disclaimers. No FDA approval application is currently pending.
European Union: Approved in Austria (its country of origin) and Germany, among other member states. The EMA has classified it in the "SAFE" category, but it does not have centralized EU-wide marketing authorization.
Russia and Eastern Europe: Cerebrolysin sees its heaviest clinical use here — widely prescribed for stroke, TBI, and dementia.
Asia: Approved and used in China, South Korea, Vietnam, the Philippines, and several other countries.
Overall: Approved in more than 50 countries, but its regulatory path has been country-by-country rather than through a single major authority, which partly explains its absence from the U.S. market.
Limitations of Current Research
An honest assessment of Cerebrolysin's evidence base has to acknowledge several problems.
The Cochrane problem. The most rigorous systematic reviews — the 2023 Cochrane review on stroke, the Cochrane review on vascular dementia — have consistently rated the evidence quality as low to moderate. The positive findings from individual trials and industry-sponsored meta-analyses look less impressive when filtered through Cochrane's strict methodology.
Outcome measures. Many trials report improvements in neurological rating scales (NIHSS, ADAS-cog) without showing corresponding benefits in functional independence, quality of life, or mortality. A patient can have a better NIHSS score and still not live independently. The gap between surrogate endpoints and patient-relevant outcomes is a real weakness.
Sample sizes. Most RCTs have been small — dozens to a few hundred patients. The CAPTAIN trials, the strongest TBI evidence, enrolled 46 and 139 patients. These are pilot-scale studies. Definitive answers require thousands of participants.
Industry funding. EVER Pharma has funded much of the clinical research on Cerebrolysin. While industry-funded research is not automatically invalid, it creates a conflict of interest that reviewers must account for. The manufacturer has a financial stake in positive results.
The Masliah controversy. In 2024, Science magazine revealed significant image manipulation across 132 papers by Eliezer Masliah, former director of neuroscience at the National Institute on Aging. Eight of the challenged studies were Cerebrolysin preclinical work conducted at UC San Diego with EVER Pharma funding. Several have since been retracted. EVER Pharma stated that none of the challenged work "played a crucial role in the clinical development of Cerebrolysin." The clinical trial data — conducted by different groups — is not directly implicated, but the episode raises legitimate questions about the preclinical foundation.
Composition questions. Independent researchers using high-performance liquid chromatography have reported that Cerebrolysin contains fewer peptide fragments than advertised — "largely composed of amino acids and salt, with some trace protein fragments." If confirmed, this would undermine the mechanistic rationale.
Geographic and publication bias. A disproportionate amount of positive evidence comes from Eastern European and Asian studies, which may have different methodological standards than those required for FDA approval.
Frequently Asked Questions
Is Cerebrolysin available in the United States?
No. Cerebrolysin is not FDA-approved and cannot be legally prescribed or purchased from a U.S. pharmacy. Some research suppliers sell it under "research use only" disclaimers.
How does Cerebrolysin compare to other nootropic peptides?
Cerebrolysin has a much larger clinical trial database than most nootropic peptides. Semax and Selank are single synthetic peptides with defined mechanisms but fewer large trials. Dihexa shows potency in preclinical models but has almost no human data. Cerebrolysin's advantage is volume of evidence; its disadvantage is that evidence quality is often moderate to low.
Does Cerebrolysin actually cross the blood-brain barrier?
Its peptide fragments are under 10,000 daltons, and experimental data supports blood-brain barrier penetration. However, the half-life of some constituents (like BDNF) in the bloodstream is very short — around 10 minutes — which raises questions about how much active peptide actually reaches the brain at therapeutic concentrations.
Can Cerebrolysin prevent Alzheimer's disease?
There is no evidence that Cerebrolysin prevents Alzheimer's disease. The clinical trials have tested it as a treatment for existing mild-to-moderate AD, not as a preventive measure. Whether it can slow disease progression over years is unknown — the longest trials have run for 24 weeks.
What is the typical treatment course?
Most protocols use daily infusions for 10–21 days, sometimes repeated in cycles. Stroke patients typically receive higher doses (30–50 mL) for shorter courses, while Alzheimer's patients may receive lower doses (10–30 mL) over longer periods.
Is Cerebrolysin safe for long-term use?
Meta-analyses suggest it is well-tolerated for up to three years, with side effects comparable to placebo. Truly long-term data (5–10+ years) does not exist, and the 2023 Cochrane review's concern about non-fatal serious adverse events warrants attention.
Why isn't Cerebrolysin FDA-approved if it's used in 50+ countries?
The FDA requires large, well-designed, multi-center RCTs with clearly defined primary endpoints showing clinically meaningful benefit. The existing data, while extensive, has been characterized by small sample sizes, heterogeneous methods, reliance on surrogate endpoints, and inconsistent results. No manufacturer has submitted a formal New Drug Application.
The Bottom Line
Cerebrolysin is one of the most studied neuropeptide preparations in the world, and its story illustrates the gap between "promising research" and "proven treatment." The clinical data consistently shows modest improvements in neurological rating scales for stroke, TBI, and dementia. Some individual trials — the Guekht vascular dementia study, the Gauthier Alzheimer's meta-analysis — produced genuinely impressive results. But when the strongest independent reviewers (Cochrane) assess the totality of evidence, they find the data insufficient to establish clear benefit on the outcomes that matter most: survival, functional independence, and quality of life.
The 2024 research integrity scandal and ongoing composition questions further complicate the picture. Clinicians in countries where Cerebrolysin is approved continue to prescribe it alongside standard care, based on decades of experience and a favorable safety profile. But the absence of FDA approval reflects a real gap in the evidence — not merely a bureaucratic oversight.
The honest summary: there is biological plausibility, a large body of trials showing modest benefit on intermediate endpoints, and a generally mild side effect profile. There is not definitive proof of meaningful clinical benefit from high-quality evidence. Cerebrolysin has genuine potential that has not yet met the highest evidentiary standards — and until it does, claims about its benefits should be held with informed caution.