Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Clinical Trial
. 2023 Dec;16(12):1750-1755.
doi: 10.25122/jml-2023-0459.

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

Alfredo José Firstenfeld  1 , Jorge Listorti  1 , Nasser Jalaff  1 , Claudia Patricia Loaiza Orozco  1 , Francisco Navarrete Gosdenovich  1 , Timo Schurr  2
Affiliations
Clinical Trial

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

Alfredo José Firstenfeld et al. J Med Life. 2023 Dec.

Abstract

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.

Keywords: Cerebrolysin; amyotrophic lateral sclerosis; clinical study; functional outcome; riluzole.

PubMed Disclaimer

Conflict of interest statement

One of the authors, Alfredo José Firstenfeld, has received a grant from EVER Neuro Pharma GmbH. The authors have no other conflict of interests to declare.

Figures

Figure 1
Figure 1
Development of the ALSFRS-R score over time
See this image and copyright information in PMC

Similar articles

See all similar articles

Cited by

References

    1. Siddique N, Siddique T. GeneReviews®. Seattle (WA): University of Washington Seattle; 2001. Mar 23, Amyotrophic Lateral Sclerosis Overview; pp. 1993–2024. [updated 2023 Sep 28]
    1. Hardiman O, Al-Chalabi A, Chio A, Corr EM, et al. Amyotrophic lateral sclerosis. Nat Rev Dis Primers. 2017;3:17071. doi: 10.1038/nrdp.2017.71. - DOI - PubMed
    1. Abrahams S, Newton J, Niven E, Foley J, Bak TH. Screening for cognition and behaviour changes in ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2014;15(1-2):9–14. doi: 10.3109/21678421.2013.805784. - DOI - PubMed
    1. Longinetti E, Fang F. Epidemiology of amyotrophic lateral sclerosis: an update of recent literature. Curr Opin Neurol. 2019;32:771–776. doi: 10.1097/WCO.0000000000000730. - DOI - PMC - PubMed
    1. Brown CA, Lally C, Kupelian V, Flanders WD. Estimated prevalence and incidence of amyotrophic lateral sclerosis and SOD1 and C9orf72 genetic variants. Neuroepidemiology. 2021;55:342–353. doi: 10.1159/000516752. - DOI - PubMed

Publication types

MeSH terms