All Neurological disorders articles
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NewsPatient-specific brain tumour chip models vascular drug barriers
A microfluidic chip combining patient-derived glioblastoma cells with their tumour vascular environment could give drug developers a more realistic preclinical model for predicting treatment response – and may help explain why genetically similar patients respond differently to the same therapy.
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NewsAutomated MRI pipeline standardises preclinical stroke damage measurement
An automated imaging pipeline developed at USC’s Stevens Neuroimaging and Informatics Institute can measure stroke-related brain tissue damage from MRI scans with accuracy matching human experts, offering a scalable, standardised tool for preclinical drug evaluation across multi-site research networks.
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NewsMeis2 gene identified as potential therapeutic target for neurodevelopmental disorders
A gene therapy targeting parvalbumin inhibitory neurons has restored experience-dependent plasticity and reduced seizure frequency in a mouse model of neurodevelopmental disorders, offering a potential new avenue for treating autism spectrum disorder and epilepsy.
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NewsNanomedicine for glioblastoma: overcoming the blood-brain barrier
A new review published in Chinese Neurosurgical Journal examines how nanoscale drug delivery systems could help overcome one of the most persistent obstacles in glioblastoma treatment – getting therapeutics across the blood–brain barrier and into tumour tissue.
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NewsVY1706 Alzheimer’s gene therapy achieves 75 percent tau reduction in preclinical study
Voyager Therapeutics has presented six-month GLP toxicology data for VY1706, its investigational tau-targeting gene therapy, showing durable tau reductions of up to 75 percent and a clean safety profile in non-human primates, as the company prepares to begin first-in-human dosing in the second half of 2026.
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NewsGlymphatic gene therapy platform targets brain’s glial cells precisely
A new gene therapy platform exploiting the brain’s glymphatic transport system and engineered AAV5 vectors could offer a targeted treatment route for multiple sclerosis, Huntington’s disease and rare white matter disorders.
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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.