Pharmacogenomics in Brain Disorders

Brain disorders—including both neurological diseases (e.g., Alzheimer’s disease, Parkinson’s disease, epilepsy, stroke, multiple sclerosis, migraine) and psychiatric conditions (e.g., depression, schizophrenia, bipolar disorder, autism, anxiety, substance use disorders)—represent one of the most significant global health challenges. Affecting over one billion people worldwide, these conditions account for an enormous personal, societal, and economic burden, with estimated costs surpassing $2.5 trillion annually.

Despite progress in neurobiology and psychopharmacology, therapeutic response in brain disorders remains highly heterogeneous, with treatment failure rates exceeding 40% in major depression and up to 30% in epilepsy. Adverse drug reactions, polypharmacy, and trial-and-error prescribing further complicate clinical management.

Pharmacogenomics—the study of genetic variability influencing drug response—offers powerful tools to personalize treatment, minimize adverse events, and optimize efficacy. Polymorphisms in pathogenic, mechanistic, metabolic, transporter and pleiotropic genes affecting pharmacokinetics (e.g., CYP450, ABC transporters, UGTs) and pharmacodynamics (e.g., dopaminergic, serotonergic, glutamatergic receptors, ion channels, synaptic proteins) are now recognized as key modulators of therapeutic outcomes. Moreover, the integration of multi-omics, epigenetics, and AI-driven predictive models is accelerating translation into clinical practice.

This Discover Mental Health Collection seeks to provide a comprehensive platform for the latest advances in pharmacogenomics of brain disorders, bridging the gap between fundamental discoveries and clinical applications. By gathering high-quality contributions from international experts, our goal is to highlight novel biomarkers, mechanistic insights, translational strategies, and clinical implementation models that can shape the future of precision medicine in neurology and psychiatry.

We welcome submissions covering (but not limited to) the following areas:

- Pharmacogenomic biomarkers of drug efficacy and safety in brain disorders

- Clinical implementation of pharmacogenomic testing in neurology and psychiatry

- Multi-omics approaches (transcriptomics, proteomics, metabolomics) integrated with pharmacogenomics

- Epigenetic and pharmacoepigenomic regulation of drug response in brain diseases

- Population studies exploring genetic variability across ethnic groups

- Artificial intelligence and bioinformatics applied to pharmacogenomic prediction models

- Clinical trials and real-world evidence of pharmacogenomic testing in psychiatry and neurology

- Pharmacoeconomic evaluations: cost-effectiveness, health policy, and reimbursement models

- Ethical, regulatory, and implementation challenges in pharmacogenomics for brain disorders

We warmly invite researchers, clinicians, and scholars from all regions of the world to contribute and help advance the field of precision medicine in brain disorders.

Keywords: Brain disorders, Psychiatric disorders, Neurological disorders, Neurodevelopment disorders, Neurogenomics, Neuroepigenetics, Pharmacogenomics of brain drugs, Pharmacoepigenetics

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