SynthesisBMJ open2023
Systematic, comprehensive, evidence-based approach to identify neuroprotective interventions for motor neuron disease: using systematic reviews to inform expert consensus.
Synthesis in BMJ open, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04302870 (Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial), which is not on this map. Cited by 10 papers, 1 of them a synthesis that pooled it.
What it found
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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.
Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial
Who cites it
10 citing papers in PubMed, 1 synthesis or guideline pooled it, 14 citations in OpenAlex.
- Systematic online living evidence summaries: emerging tools to accelerate evidence synthesis.Clinical science (London, England : 1979) · 2023Pooled it
- Trial
- Developing Interim Outcome Measures for Multi-Arm Multistage Clinical Trials in Multiple Sclerosis.Neurology · 2026Article
- Repurposed drug prioritization pipeline for a multi-arm platform trial in clinical Alzheimer's disease.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2026Article
- Ageing, Neurodegeneration and the Endocannabinoid System.Current topics in behavioral neurosciences · 2026Review
- Treatment Selection and Prioritization for the EJS ACT-PD MAMS Trial Platform.Movement disorders : official journal of the Movement Disorder Society · 2025Article
- Transforming Alzheimer's Treatment: Unveiling New Potential with Drug Repurposing Strategies.Current medicinal chemistry · 2025Review
- Preclinical assessment for translation to humans: The PATH approach for assessing supporting evidence for early-phase trials and innovative care.Med (New York, N.Y.) · 2024Review
- In Vitro Models of Amyotrophic Lateral Sclerosis.Cellular and molecular neurobiology · 2023Review
- From the Bush to the Brain: Preclinical Stages of Ethnobotanical Anti-Inflammatory and Neuroprotective Drug Discovery-An Australian Example.International journal of molecular sciences · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
50 authors at 12 institutions in 1 country.
Funding
Abstract
objectivesMotor neuron disease (MND) is an incurable progressive neurodegenerative disease with limited treatment options. There is a pressing need for innovation in identifying therapies to take to clinical trial. Here, we detail a systematic and structured evidence-based approach to inform consensus decision making to select the first two drugs for evaluation in Motor Neuron Disease-Systematic Multi-arm Adaptive Randomised Trial (MND-SMART: NCT04302870), an adaptive platform trial. We aim to identify and prioritise candidate drugs which have the best available evidence for efficacy, acceptable safety profiles and are feasible for evaluation within the trial protocol.
methodsWe conducted a two-stage systematic review to identify potential neuroprotective interventions. First, we reviewed clinical studies in MND, Alzheimer's disease, Huntington's disease, Parkinson's disease and multiple sclerosis, identifying drugs described in at least one MND publication or publications in two or more other diseases. We scored and ranked drugs using a metric evaluating safety, efficacy, study size and study quality. In stage two, we reviewed efficacy of drugs in MND animal models, multicellular eukaryotic models and human induced pluripotent stem cell (iPSC) studies. An expert panel reviewed candidate drugs over two shortlisting rounds and a final selection round, considering the systematic review findings, late breaking evidence, mechanistic plausibility, safety, tolerability and feasibility of evaluation in MND-SMART.
resultsFrom the clinical review, we identified 595 interventions. 66 drugs met our drug/disease logic. Of these, 22 drugs with supportive clinical and preclinical evidence were shortlisted at round 1. Seven drugs proceeded to round 2. The panel reached a consensus to evaluate memantine and trazodone as the first two arms of MND-SMART. DISCUSSION: For future drug selection, we will incorporate automation tools, text-mining and machine learning techniques to the systematic reviews and consider data generated from other domains, including high-throughput phenotypic screening of human iPSCs.
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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.