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Review organizes pharmacological strategies targeting gut-brain axis for neurological and psychiatric disorders

Review organizes pharmacological strategies targeting gut-brain axis for neurological and psychiatri…
Photo by Navy Medicine / Unsplash
Key Takeaway
Consider this review a theoretical framework, not evidence for specific gut-brain axis treatments.

This systematic review summarizes current understanding of microbiota–gut–brain axis dysfunction and organizes existing pharmacological strategies targeting this axis for neurological and psychiatric disorders. The theoretical application includes conditions such as Parkinson's disease, Alzheimer's disease, multiple sclerosis, autism spectrum disorder, depression, anxiety, and stroke. The review categorizes strategies including microbiota-directed interventions and immune–inflammatory modulators that aim to address shared pathophysiological mechanisms.

The review identifies the microbiota–gut–brain axis as a promising therapeutic target based on growing evidence of its involvement in these conditions. However, the article does not report specific clinical trial results, effect sizes, or safety data for any intervention. No primary or secondary outcomes, sample sizes, follow-up durations, or comparative data are provided.

Key limitations include the absence of new clinical study data and the review's nature as a summary of mechanisms and strategies rather than evidence of therapeutic effects. Safety and tolerability information for any pharmacological approach is not reported. The text appropriately notes that dysregulation of the axis has been 'implicated in' conditions, indicating association rather than causation.

For clinical practice, this review provides a conceptual framework for understanding potential therapeutic approaches but does not establish causal therapeutic effects of any pharmacological strategy. The evidence remains emerging, with descriptions of 'growing evidence' and 'increasingly recognized' mechanisms. Clinicians should view this as an organizing summary of a research area rather than guidance for specific treatment decisions.

Study Details

Study typeSystematic review
EvidenceLevel 1
PublishedApr 2026
View Original Abstract ↓
The microbiota–gut–brain axis represents a complex bidirectional communication network linking the gastrointestinal system and the central nervous system and has been increasingly recognized as a key contributor to neurological and psychiatric disorders. Growing evidence indicates that alterations in gut microbiota composition and function can influence brain development and function through neural, immune, endocrine, and metabolic pathways, thereby modulating neuroinflammation, neurotransmission, and blood–brain barrier integrity. Dysregulation of this axis has been implicated in a range of conditions, including Parkinson’s disease, Alzheimer’s disease, multiple sclerosis, autism spectrum disorder, depression, anxiety, and stroke. Recent pharmacological advances have identified the microbiota–gut–brain axis as a promising therapeutic target. Current strategies focus on modulating shared pathophysiological mechanisms rather than disease-specific endpoints and include microbiota-directed interventions, immune–inflammatory modulators, neurotransmitter-targeting agents, and approaches aimed at restoring intestinal and blood–brain barrier function. In this review, we summarize the core mechanisms underlying microbiota–gut–brain axis dysfunction and organize existing pharmacological strategies according to their primary targets. By integrating evidence across multiple disorders, we provide a mechanism-oriented framework to support future drug development and precision therapeutic approaches for brain disorders.
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