Add-on treatment with Cerebrolysin improves clinical symptoms in patients with ALS: results from a prospective, single-center, placebo-controlled, randomized, double-blind, phase II study.

Firstenfeld AJ, Listorti J, Jalaff N, Loaiza Orozco CP, Navarrete Gosdenovich F, Schurr T

Journal of medicine and life1 Dec 2023Cited by 4 (Europe PMC)

PMID38585517PMCIDPMC10994623DOI10.25122/jml-2023-0459PepBankPUB-38585517

Summary

Amyotrophic lateral sclerosis (ALS) is a devastating and progressive neurodegenerative disease with limited treatment options available. Cerebrolysin is a drug candidate for the treatment of ALS because of its neuroprotective and neuroregenerative effects. We initiated a pilot clinical study of a combination of Cerebrolysin and riluzole to assess the therapeutic benefit of Cerebrolysin as an add-on treatment on clinical signs and symptoms in outpatients with ALS. Twenty patients with a clinically definitive diagnosis of ALS were enrolled and randomly assigned in a 1:1 ratio to receive Cerebrolysin or placebo. All patients received 50 mg of riluzole PO twice daily as a standard treatment. Patients in the Cerebrolysin group received intravenous injections of 10 mL of Cerebrolysin once daily, five days a week for the first month and three days a week for the next two months. Analysis of the ALS Functional Rating Scale - revised at Month 1 (primary outcome measure), showed a significant treatment effect in favor of Cerebrolysin with a 2.3-point improvement from baseline to Month 1 compared to a 0.9-point decrease in patients on placebo (P=0.005). The effect was maintained over the three-month study period, and the beneficial effect of Cerebrolysin over placebo was also evident in the secondary outcome measures. The safety analysis showed that the combination of riluzole and Cerebrolyisn was well tolerated. Our results demonstrate for the first time a significant clinical effect of Cerebrolysin in improving functional outcomes in patients with ALS and suggest that Cerebrolysin has potential as a novel therapeutic option for ALS.

CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study3

2 results from studies that gave the peptide. 2 results from people.

01HumanRandomized controlled trialEfficacy outcome
In ALS patients receiving riluzole, Cerebrolysin improved revised ALS functional scores at Month 1 compared with placebo.
In plain wordsIn ALS patients receiving riluzole, Cerebrolysin improved functional scores at Month 1 compared with placebo.
Peptide
Cerebrolysin
Outcome
IncreaseALS Functional Rating Scale revised at Month 1
Study
n = 20
Comparator
Placebo with background riluzole
Analysis
Primary
Outcome group
Exercise and physical function · Nervous system and cognition
Studied dose
10 mL once daily, five days a week for the first month and three days a week for the next two months · intravenous · 1 monthAs given in this study (human).
Measurements
ALSFRS-R change
2.3 points
Cerebrolysin improvement
ALSFRS-R change
0.9 points
Placebo decrease
P value for ALSFRS-R treatment effect
0.005
Cerebrolysin versus placebo, both with riluzole; change from baseline to Month 1
Source sentences
Summary
Analysis of the ALS Functional Rating Scale - revised at Month 1 (primary outcome measure), showed a significant treatment effect in favor of Cerebrolysin with a 2.3-point improvement from baseline to Month 1 compared to a 0.9-point decrease in patients on placebo (P=0.005).
3 context sentences(design, population, supporting numbers)
Summary · context
Twenty patients with a clinically definitive diagnosis of ALS were enrolled and randomly assigned in a 1:1 ratio to receive Cerebrolysin or placebo.
Summary · context
All patients received 50 mg of riluzole PO twice daily as a standard treatment.
Summary · context
Patients in the Cerebrolysin group received intravenous injections of 10 mL of Cerebrolysin once daily, five days a week for the first month and three days a week for the next two months.
Show in the study text Report a problemFND-3858551701
02HumanRandomized controlled trialEfficacy outcome
In ALS patients receiving riluzole, the Cerebrolysin functional benefit over placebo persisted throughout the three-month study.
In plain wordsIn ALS patients receiving riluzole, the functional benefit of Cerebrolysin over placebo persisted throughout the study.
Peptide
Cerebrolysin
Outcome
IncreaseSustained functional treatment effect
Study
n = 20
Comparator
Placebo with background riluzole
Outcome group
Exercise and physical function · Nervous system and cognition
Studied dose
10 mL once daily, five days a week for the first month and three days a week for the next two months · intravenous · three monthsAs given in this study (human).
Source sentences
Summary
The effect was maintained over the three-month study period, and the beneficial effect of Cerebrolysin over placebo was also evident in the secondary outcome measures.
4 context sentences(design, population, supporting numbers)
Summary · context
Analysis of the ALS Functional Rating Scale - revised at Month 1 (primary outcome measure), showed a significant treatment effect in favor of Cerebrolysin with a 2.3-point improvement from baseline to Month 1 compared to a 0.9-point decrease in patients on placebo (P=0.005).
Summary · context
Twenty patients with a clinically definitive diagnosis of ALS were enrolled and randomly assigned in a 1:1 ratio to receive Cerebrolysin or placebo.
Summary · context
All patients received 50 mg of riluzole PO twice daily as a standard treatment.
Summary · context
Patients in the Cerebrolysin group received intravenous injections of 10 mL of Cerebrolysin once daily, five days a week for the first month and three days a week for the next two months.
Show in the study text Report a problemFND-3858551702

Studies that did not test the peptide alone1

In this study, Cerebrolysin was a lab tool or a carrier, part of a mix, or the subject of a lab method. These results are not evidence about taking Cerebrolysin.

03HumanNot a test of Cerebrolysin aloneRandomized controlled trialSafety outcome
In ALS patients, the combination of riluzole and Cerebrolysin was well tolerated.
In plain wordsIn ALS patients, the combination of riluzole and Cerebrolysin was well tolerated.
Peptide
Cerebrolysin
Outcome
Not applicableTolerability
Study
n: not reported in source
Outcome group
Adverse events · Whole body
Source sentences
Summary
The safety analysis showed that the combination of riluzole and Cerebrolyisn was well tolerated.
3 context sentences(design, population, supporting numbers)
Summary · context
Twenty patients with a clinically definitive diagnosis of ALS were enrolled and randomly assigned in a 1:1 ratio to receive Cerebrolysin or placebo.
Summary · context
All patients received 50 mg of riluzole PO twice daily as a standard treatment.
Summary · context
Patients in the Cerebrolysin group received intravenous injections of 10 mL of Cerebrolysin once daily, five days a week for the first month and three days a week for the next two months.
In this study, Cerebrolysin was a lab tool or a carrier, part of a mix, or the subject of a lab method. This result is not evidence about taking Cerebrolysin.
Show in the study text Report a problemFND-3858551704

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
14 Oct 2026, 05:02 UTC
Study results updated
Version 1
14 Oct 2026, 01:46 UTC
First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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