ReviewNeuropharmacology2017
The need for new approaches in CNS drug discovery: Why drugs have failed, and what can be done to improve outcomes.
Review in Neuropharmacology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 182 papers, 4 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
182 citing papers in PubMed, 4 syntheses or guidelines pooled it, 354 citations in OpenAlex.
- Meta-analysis of the make-up and properties of in vitro models of the healthy and diseased blood-brain barrier.Nature biomedical engineering · 2025Pooled it
- Safety, Efficacy and Clinical Applications of Focused Ultrasound-Mediated Blood Brain Barrier Opening in Alzheimer's Disease: A Systematic Review.The journal of prevention of Alzheimer's disease · 2024Pooled it
- Placebo response in pharmacological and dietary supplement trials of autism spectrum disorder (ASD): systematic review and meta-regression analysis.Molecular autism · 2020Pooled it
- Engineered tissues and strategies to overcome challenges in drug development.Advanced drug delivery reviews · 2020Pooled it
- Establishment of an in vitro human blood-brain barrier model for antiviral screening against neurotropic viruses.Fluids and barriers of the CNS · 2026Article
- New approach methodologies in neurotherapeutics development.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026Review
- Biodistribution, Radiation Dosimetry, and Kinetic Modeling of [Pharmaceuticals (Basel, Switzerland) · 2026Article
- DeepBBB: A Data-Composition-Aware Graph Screening Workflow for BBB-Focused CNS Library Construction and Prospective PAMPA-BBB Evaluation.Pharmaceuticals (Basel, Switzerland) · 2026Article
- Comparative analysis of surface coatings for human neural co-cultures identifies fibronectin as a robust MEA substrate.Scientific reports · 2026Article
- Cross-species characterisation of microglial morphology and proliferation during ageing.GeroScience · 2026Article
- Functional Mapping of Neurodevelopmental Disease Pathways to Key Neurodevelopmental Processes Represented in the Developmental Neurotoxicity In Vitro Testing Battery.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Review
- Artificial intelligence revolutionizing CNS drug discovery and development.Drug discovery today · 2026Review
- Uncovering putative neural mechanisms of neurotherapeutic impacts on EEG using the Human Neocortical Neurosolver.bioRxiv : the preprint server for biology · 2026Article
- Ependymoma group-specific blood-brain barrier differences uncovered by a multi-omics approach.Scientific reports · 2026Article
- Lipid droplets in neurodegenerative diseases: pathological drivers and therapeutic vulnerabilities.Cell death discovery · 2026Review
- From In Silico Hypothesis to Validation: The Role of Real-World Evidence in the Preliminary Verification of AI-Generated Drug-Repositioning Candidates: A Comprehensive Review.Journal of clinical medicine · 2026Review
- Evaluating the Translation Value of TwoCancers · 2026Article
- From General-Purpose to Disease-Specific Features: Aligning LLM Embeddings on a Disease-Specific Biomedical Knowledge Graph for Drug Repurposing.bioRxiv : the preprint server for biology · 2026Article
- From Polyphenols to Prodrugs: Bridging the Blood-Brain Barrier with Nanomedicine and Neurotherapeutics.International journal of molecular sciences · 2026Review
122 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
An important goal of biomedical research is to translate basic research findings into useful medical advances. In the field of neuropharmacology this requires understanding disease mechanisms as well as the effects of drugs and other compounds on neuronal function. Our hope is that this information will result in new or improved treatment for CNS disease. Despite great progress in our understanding of the structure and functions of the CNS, the discovery of new drugs and their clinical development for many CNS disorders has been problematic. As a result, CNS drug discovery and development programs have been subjected to significant cutbacks and eliminations over the last decade. While there has been recent resurgence of interest in CNS targets, these past changes in priority of the pharmaceutical and biotech industries reflect several well-documented realities. CNS drugs in general have higher failure rates than non-CNS drugs, both preclinically and clinically, and in some areas, such as the major neurodegenerative diseases, the clinical failure rate for disease-modifying treatments has been 100%. The development times for CNS drugs are significantly longer for those drugs that are approved, and post-development regulatory review is longer. In this introduction we review some of the reasons for failure, delineating both scientific and technical realities, some unique to the CNS, that have contributed to this. We will focus on major neurodegenerative disorders, which affect millions, attract most of the headlines, and yet have witnessed the fewest successes. We will suggest some changes that, when coupled with the approaches discussed in the rest of this special volume, may improve outcomes in future CNS-targeted drug discovery and development efforts. This article is part of the Special Issue entitled "Beyond small molecules for neurological disorders".
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.