All Neurological disorders articles
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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.
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NewsOutSee wins Longitude Prize award for ALS target discovery
OutSee has secured a Discovery Award from the Longitude Prize on ALS, providing £100,000 in funding and access to genomic data from 9,000 patients. The company will deploy its AI-driven Nomaly platform to identify novel therapeutic targets for amyotrophic lateral sclerosis over a nine-month research programme.
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ArticleWomen in STEM: resilience and leadership in rare disease research
Rare neurological diseases remain one of the most challenging areas in drug discovery, with many patients still lacking treatment options. Dr Nitza Thomasson discusses returning to Servier to lead its rare neurology therapeutic area and explains why resilience, curiosity and persistence are essential for those looking to build a meaningful career in STEM.
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NewsAI-designed viral vectors achieve 50-fold brain enrichment over AAV9
WhiteLab Genomics has presented preclinical data showing that viral vectors designed using artificial intelligence achieved approximately 50-fold higher DNA enrichment in the brain compared to AAV9, with no detectable liver signal following intravenous administration in mice.
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ArticleTurning CRISPR hits into confident drug discovery decisions
Functional genomics is central to modern drug discovery, yet high attrition rates persist. In this article, Dr Salman Tamaddon-Jahromi, a postdoctoral research associate at the University of Cambridge, discusses how end-to-end CRISPR screening strategies, iPSC-derived neuronal models and layered quality control can convert functional genomics signals into actionable therapeutic hypotheses.
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NewsMachine learning identifies five distinct Parkinson’s disease subtypes
A new study from VIB and KU Leuven has revealed that Parkinson’s disease comprises five distinct molecular subtypes, each requiring tailored therapeutic approaches.
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ArticleWhy lower organisms matter for neurodegeneration drug discovery
In the wake of recent government policy aimed at actively replacing animal models in drug discovery, we consider a possible solution to the translational shortfalls of current cellular methodologies for neurodegenerative disease.
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NewsNasal spray reverses age-related brain inflammation in preclinical study
Researchers at Texas A&M University have developed a nasal spray delivering extracellular vesicles that reverses neuroinflammaging in preclinical models. Two doses significantly reduced brain inflammation, restored mitochondrial function and improved memory within weeks, with effects persisting for months. The therapy bypasses the blood-brain barrier and targets inflammatory pathways whilst reactivating cellular energy production.
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ArticleBeyond serendipity: rational design and AI’s expansion of the undruggable target landscape
For decades, drugging the ‘undruggable’ was thought to require luck rather than logic. Today, AI is transforming serendipity into strategy by enabling rational, data-driven approaches to previously inaccessible targets.