All Neurological disorders articles
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ArticleWhy the blood–brain barrier blocks most drugs – and how ultrasound could help
The blood–brain barrier protects the brain from harmful substances, but it also prevents many medicines from reaching their target. Researchers are investigating whether focused ultrasound could safely improve drug delivery.
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NewsBlood test for protein misfolding identifies Alzheimer’s risk years before symptoms
A blood-based biomarker measuring amyloid beta protein misfolding has demonstrated superior predictive accuracy for pre-symptomatic Alzheimer’s disease compared with p-tau 217, achieving an AUC of 0.87 when combined with genetic and demographic data.
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NewsLandmark fibromyalgia study highlights neurological targets for new therapies
A landmark genome-wide study of more than 2.5 million adults has identified 26 genomic regions linked to fibromyalgia, confirming a neurological basis for the condition and opening new avenues for targeted drug discovery.
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News3D genome reorganisation identified as Alzheimer’s disease molecular hallmark
An international study combining single-cell genomics, spatial transcriptomics and artificial intelligence has identified large-scale changes in three-dimensional genome organisation in Alzheimer’s-affected brain cells, establishing chromatin architecture as a previously overlooked component of the disease’s molecular pathology and a potential framework for future therapeutic targeting.
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NewsInsilico Medicine nominates ISM9528 non-opioid chronic pain candidate using AI
Insilico Medicine has nominated ISM9528, an orally available, brain-penetrant non-opioid candidate targeting a previously unrecognised pain mechanism, as its 31st AI-generated preclinical compound since 2021, with efficacy exceeding morphine in some preclinical models.
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ArticleHow AI and human biology data led to a new addiction drug candidate
How can human biology data improve target selection? Learn how one discovery programme identified a potential new treatment for opioid use disorder.
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NewsNew drug SB-0110 targets levodopa’s two biggest clinical limitations for Parkinson’s
A computational biology-derived compound has shown promise in preclinical models by simultaneously improving levodopa response and reducing dyskinesia — two of the most significant challenges in long-term Parkinson’s disease management.
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NewsGene therapy restores key features in fragile X syndrome mouse model
Researchers at Cincinnati Children’s have demonstrated that AAV-mediated delivery of the human FMR1 gene can restore FMRP expression and reverse multiple disease-related features in a fragile X syndrome mouse model, offering a promising preclinical foundation for future clinical trials.
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NewsKCL-286 targets multiple Alzheimer’s pathways in preclinical study
A repurposed small molecule originally developed for spinal cord injury has demonstrated the ability to address multiple disease-relevant pathways in Alzheimer’s disease, including DNA damage and neuroinflammation, offering a potential route to disease-modifying therapy.
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News15-PGDH enzyme inhibition protects brain cells in Parkinson’s disease models
Researchers have identified 15-PGDH enzyme inhibition as a neuroprotective strategy in Parkinson’s disease, with repurposable compounds already in clinical development offering a potential shortcut to disease-modifying therapies.
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NewsXL20 drug candidate shields motor neurons from ALS-linked TDP-43 damage
A newly identified experimental compound, XL20, has demonstrated neuroprotective effects in mouse models and human motor neurons by targeting a conserved disease-linked region of TDP-43, a protein central to ALS pathology.
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NewsATRX mutations reprogram chromatin to drive glioma progression
Scientists at MD Anderson Cancer Center have revealed how ATRX mutations restructure chromatin and activate oncogenic developmental pathways in glioma, pointing towards novel therapeutic targets including the HOXA signalling axis.
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NewsCerebral organoids identify Ebola virus persistence in neural tissue
Researchers have used human brain organoids to demonstrate that Ebola virus can replicate in neural tissue for up to 120 days, offering new insights into viral persistence mechanisms in immune-privileged sites and late-stage inflammatory complications in survivors.
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NewsThalidomide shows potential for treating rare brain vascular malformations
Researchers in China have reported encouraging preclinical and early clinical evidence that thalidomide may help stabilise and reduce rare vascular malformations affecting the brain and spinal cord, potentially offering the first medical treatment option for patients with central nervous system arteriovenous malformations who are unsuitable for invasive procedures.
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NewsAI-powered $6M project targets new Alzheimer’s treatments
A $6 million NIH-funded collaboration between Indiana University School of Medicine and Luddy School of Informatics aims to deploy AI and machine learning to identify promising Alzheimer’s drug candidates, screening billions of compounds to overcome traditional discovery bottlenecks.
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NewsCopper drug Cu(ATSM) reduces Alzheimer’s proteins by 42 percent in preclinical study
A copper-delivering compound has demonstrated the ability to restore blood-brain barrier clearance mechanisms, reducing amyloid-beta accumulation by 42 percent and improving spatial learning by 44 percent in Alzheimer’s disease models, according to research published in ACS Chemical Neuroscience.
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NewsCytokine-armoured CAR T cells target glioblastoma while reducing treatment toxicity
UCLA Health researchers have developed cytokine-armoured CAR T cells that directly attack glioblastoma tumours whilst recruiting the body’s wider immune system. The engineered cells showed improved tumour control in mouse models and could address antigen heterogeneity challenges that have limited CAR T therapy success in solid tumours.
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NewsStudy suggests senolytic therapies could slow spinal disc degeneration
Thomas Jefferson University researchers have demonstrated that senolytic therapies targeting cellular senescence may delay early intervertebral disc degeneration, a major cause of chronic back and neck pain.
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NewsOrganoid study reveals valproate’s impact on developing brain
German researchers have used cerebral organoids to investigate how the epilepsy medication valproate interferes with early brain development, identifying significant disruption to the extracellular matrix and neuronal maturation that may explain increased neurodevelopmental risks in exposed foetuses.
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ArticleBeyond tangles: why soluble intracellular tau should guide drug discovery
Tau tangles are a hallmark of Alzheimer’s disease and related disorders, but evidence suggests the real damage may come from rare, soluble tau species inside neurons. Targeting these hidden drivers of circuit dysfunction could be key to restoring memory and cognition.