Cerebrolysin

Picture a much-hyped foreign tonic that has actually been put through dozens of real trials. Every time independent referees add up the scorecard, they shrug and say we still cannot tell if it does anything, and it might cause a few more problems. It is a rare grey-market peptide with genuine human data. The trouble is the data does not convincingly say yes.

Proposed to act as a pleiotropic neurotrophic/neuroprotective agent. The peptide fragments are thought to partially mimic endogenous neurotrophic factors (BDNF, GDNF, CNTF, NGF-like signalling), supporting neuronal survival, reducing excitotoxic and apoptotic injury, modulating amyloid precursor protein processing, and promoting neuroplasticity, synaptogenesis and neurogenesis. Most mechanistic data are preclinical (cell and animal models); the precise active components and human pharmacodynamics of this incompletely-defined biological mixture are not fully characterised.

Research contexts. Not proven uses, and not recommendations.

Unusually for a grey-market peptide, real human RCTs exist. The CARS trial reported improved upper-limb motor recovery after stroke, and numerous manufacturer-supported studies report benefit. The largest stroke trial, CASTA (approximately 1070 patients), was neutral on its primary endpoint, with apparent benefit only in post-hoc severe-stroke subgroups. Independent Cochrane reviews provide the key assessment. The acute ischaemic stroke review (Ziganshina et al., updated 2023) found probably little or no effect on all-cause death and moderate-certainty evidence of increased non-fatal serious adverse events. The vascular dementia review (Cui et al., 2019) found a possible cognitive and global-function signal but rated the evidence as low/very-low quality and not definitive. Heavy industry sponsorship and risk of bias weaken confidence throughout.

• !Independent (Cochrane) reviews repeatedly conclude the evidence is insufficient or low/very-low quality to support routine use, despite many positive industry-sponsored trials
• !The 2023 Cochrane stroke review found moderate-certainty evidence of increased non-fatal serious adverse events
• !Derived from pig brain tissue: a biologically variable, incompletely-defined mixture with inherent batch-to-batch consistency and theoretical biological-contamination concerns
• !Not a licensed medicine in the UK, US or Australia; any UK supply would be unlicensed/imported and outside MHRA assurance
• !Injectable product: grey-market or self-administered injectables carry sterility and contamination risks
• !Marketing in some countries and on wellness sites overstates efficacy relative to the independent evidence

Not a licensed medicine in the UK. Cerebrolysin holds no UK marketing authorisation from the MHRA and is not approved for any indication. It is sold in many countries (much of Europe, Asia, the Middle East, Russia and CIS states, Latin America) but is also unapproved in the US and Australia. In the UK it exists only as an unlicensed, imported product or as a research chemical sold not for human consumption. There is no NHS use and no MHRA quality or safety assurance behind grey-market supply.

• NCT01070498· Phase 4· Completed

  Cerebrolysin and Recovery After Stroke (CARS)

  Motor recovery after acute ischaemic stroke; reported positive Action Research Arm Test outcome.

• · Phase 3/4· Completed

  Cerebrolysin in Acute Stroke Treatment in Asia (CASTA)

  ~1070 patients; neutral on primary endpoint, apparent benefit only in post-hoc severe-stroke subgroup.

1. 01
   Cerebrolysin for acute ischaemic stroke (Cochrane Database of Systematic Reviews) — Ziganshina LE, Abakumova T, Nurkhametova D, Ivanchenko K, Cochrane Database Syst Rev (2023)

   Independent review: probably little/no effect on all-cause death; moderate-certainty evidence of an increase in non-fatal serious adverse events.

2. 02
   Cerebrolysin for vascular dementia (Cochrane Database of Systematic Reviews) — Cui S, Chen N, Yang M, et al., Cochrane Database Syst Rev (2019)

   Possible cognitive/global-function signal but rated low/very-low-quality evidence; not definitive.

3. 03
   Cerebrolysin and Recovery After Stroke (CARS): A Randomized, Placebo-Controlled, Double-Blind, Multicenter Trial — Muresanu DF, Heiss WD, Hoemberg V, et al., Stroke (2016)

   Reported improved upper-limb motor recovery (ARAT); industry-supported.

4. 04
   Cerebrolysin in patients with acute ischemic stroke in Asia: results of a double-blind, placebo-controlled randomized trial (CASTA) — Heiss WD, Brainin M, Bornstein NM, Tuomilehto J, Hong Z (CASTA Investigators), Stroke (2012)

   Largest stroke RCT (~1070 patients); neutral on primary endpoint, benefit only in post-hoc severe-stroke subgroup.