Rifampicin and its neuroprotective properties in humans - A systematic review

Nina Vaezipour; Sandra Bigi; Rinn Song; Nicole Ritz

doi:10.1016/j.biopha.2025.117928

Rifampicin and its neuroprotective properties in humans - A systematic review

Biomed Pharmacother. 2025 Apr:185:117928. doi: 10.1016/j.biopha.2025.117928. Epub 2025 Mar 1.

Authors

Nina Vaezipour¹, Sandra Bigi², Rinn Song³, Nicole Ritz⁴

Affiliations

¹ Department of Paediatric Infectious diseases and Vaccinology, University Children's Hospital Basel UKBB, Basel, Switzerland; Mycobacterial and Migrant Health Research Group, University of Basel, Basel, Switzerland. Electronic address: nina.vaezipour@ukbb.ch.
² Department of Paediatric Neurology, Childrens Hospital of Central Switzerland, Lucerne, Switzerland; Institute for Social and Preventive Medicine, University of Bern, Bern, Switzerland.
³ Department of Paediatrics, Oxford Vaccine Group, University of Oxford, Oxford OX3 7LE, United Kingdom.
⁴ Department of Paediatric Infectious diseases and Vaccinology, University Children's Hospital Basel UKBB, Basel, Switzerland; Department of Paediatrics and Paediatric Infectious Diseases, Childrens Hospital of Central Switzerland and Faculty of Health Sciences and Medicine, University of Lucerne, Lucerne, Switzerland; Department of Paediatrics, The Royal Childrens Hospital Melbourne, University of Melbourne, Parkville, Victoria, Australia.

PMID: 40024057
DOI: 10.1016/j.biopha.2025.117928

Abstract

Background: Rifampicin is an antimicrobial drug used in the treatment of mycobacterial and gram-positive bacterial infections. In vitro studies suggest additional rifampicin-associated reduction of neurotoxicity. The aim of this study was to review the evidence for neuroprotective effects of rifampicin in infectious and non-infectious diseases in human studies.

Methods: A systematic literature search was done in MEDLINE and Embase including studies from 1 Jan 1946/47-20 Jan 2024, in accordance with the preferred reporting items for systematic reviews and meta-analysis (PRISMA) (PROSPERO ID: CRD42022349852). Original articles investigating the effect of rifampicin on infections of the central nervous system (CNS) or on neurodegenerating diseases, were included. Screening, data extraction and risk of bias assessment were done using the Covidence software. Two authors assessed and extracted articles independently and blinded to each other.

Results: 1438 articles were identified of which eight were included in the final analysis: Four studies included patients with infectious diseases and four studies with neurodegenerative diseases. A neuroprotective effect of rifampicin was shown in five studies. The studies found reduced inflammatory parameters in the cerebrospinal fluid, improved neurological outcome, less cognitive decline, less brain atrophy or less metabolic decline on imaging as an effect of rifampicin. One RCT showed worsening of cognitive assessment scales in neurodegenerative patients.

Conclusion: Current evidence suggests a neuroprotective effect of rifampicin in humans. As evaluation of neuroprotection is not standardized, a consensus definition for evaluation of clinical, radiological, and neuropsychological follow-up after pharmacological intervention would be helpful for future studies assessing neuroprotection.

Keywords: Central nervous system inflammation; Neurodegenerative disease; Neuroinflammation; Neuroprotection; Neurotoxicity; Tuberculous meningitis.

Publication types

Systematic Review
Review

MeSH terms

Humans
Neurodegenerative Diseases / drug therapy
Neuroprotective Agents* / pharmacology
Neuroprotective Agents* / therapeutic use
Rifampin* / pharmacology
Rifampin* / therapeutic use

Substances

Rifampin
Neuroprotective Agents