Emerging Therapeutics for Brain Disorders: From Molecular Targets to Translational Interventions

IntroductionNeurological and neuropsychiatric disorders represent some of the most profound scientific and clinical challenges of the modern era, imposing a severe burden on patients, families, and global healthcare systems. While decades of laboratory-based research have illuminated the complex genetic, molecular, and cellular pathways that drive conditions such as Alzheimer’s, Parkinson’s, amyotrophic lateral sclerosis, traumatic brain injury, ischemic stroke, severe depression, and neurodevelopmental conditions, a critical bottleneck remains. Translating these laboratory-bench breakthroughs into robust, clinically viable therapies is a notoriously difficult and high-risk process. Traditional medical treatments frequently focus on managing clinical symptoms without arresting, halting, or reversing the underlying pathological progression of the disease. Consequently, there is an urgent and critical need to bridge the gap between basic mechanistic discovery and practical translational medicine. By focusing on highly selective, target-driven pharmacological approaches, innovative delivery systems, and sophisticated computational and multi-omics frameworks, contemporary neuroscience can accelerate the pipeline of next-generation disease-modifying interventions.AimThe primary aim of this Research Topic is to gather a comprehensive collection of pioneering research and authoritative reviews that accelerate the transition of therapeutic strategies from basic molecular targets to clinical application. Specifically, we seek to highlight innovative therapeutic modalities, objective diagnostic biomarkers, and predictive preclinical models that directly address the core pathophysiological drivers of brain disorders. By fostering multidisciplinary collaboration among molecular neurobiologists, computational biologists, biochemists, pharmacologists, and clinical researchers, this collection aims to systematically address the critical roadblocks in the translational pipeline. We strive to explore how researchers can safely, effectively, and rapidly transition promising preclinical candidate molecules, cellular pathways, and technological platforms into active clinical trials, eventually delivering tangible, real-world bedside therapies to patients in need.ScopeWe welcome original research articles, comprehensive reviews, mini-reviews, perspectives, and clinical trial reports that advance our understanding of drug discovery and translation in neurology and psychiatry. This collection focuses on bridging the gap between molecular mechanisms and therapeutic outcomes.Our specific scope focuses on key thematic areas, including:o Systematic identification, validation, and computational modeling of novel molecular and cellular targets within diseased brain circuits.o Pathophysiological targeting of neuroinflammation, oxidative stress, mitochondrial dysfunction, cellular signaling, and synaptic failure or cognitive decline.o Emerging pharmacological interventions, including small molecules, biologics, natural products, and pathway-specific modulators.o Innovative cell-based (including stem cell therapies), gene-based, and RNA-based therapeutic approaches.o Neuroprotective and disease-modifying strategies designed to mitigate acute brain injuries or slow chronic neurodegenerative processes.o Advanced translational models, diagnostic and prognostic biomarkers, and multi-omics approaches (genomics, proteomics, metabolomics, transcriptomics) for therapeutic discovery.o Critical analyses of preclinical-to-clinical translation, outlining the major hurdles in safety, blood-brain barrier penetration, drug delivery, and clinical trial efficacy.Through this multi-faceted exploration, we hope to establish a robust framework that guides future drug-development strategies and enhances clinical success rates. Topic editors: Arup Acharjee, PAPIA ACHARJEE, Raj Kumar Koiri, THAMIL MANI SIVANANDAM Source: https://www.frontiersin.org/research-topics/82936/emerging-therapeutics-for-brain-disorders-from-molecular-targets-to-translational-interventions