All Drug Targets articles
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ArticleOrgan chips move closer to drug discovery pipelines
From uncovering new drug targets to predicting human toxicity, organ chips are showing what they could bring to drug discovery. Professor Donald Ingber of Harvard University discusses where the technology is heading next.
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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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NewsPeptide vaccine targeting PCSK9 proves effective in cardiovascular disease models
A peptide vaccine targeting PCSK9 has demonstrated sustained antibody responses and reductions in LDL cholesterol and atherosclerotic plaque formation in animal models, offering a potential long-term alternative to current lipid-lowering therapies.
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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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NewsMETTL3 protein finds new mechanism driving breast cancer metastasis
Scientists at Umeå University have uncovered a previously unknown function for the RNA-modifying protein METTL3, revealing it plays a distinct role in enabling breast cancer cells to invade surrounding tissue and form metastases – findings that could open new avenues for therapeutic targeting.
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NewsHidden vulnerability in persister cells could prevent KRAS cancer relapse
Scientists at Chiba University have identified a metabolic vulnerability in drug-tolerant cancer cells that survive KRAS-targeted therapy, opening a potential route to combination treatments designed to prevent disease recurrence in lung, pancreatic and colorectal cancers.
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NewsStreptomyces antibiotic ‘megacluster’ targets biotin to combat drug-resistant infections
Researchers at McMaster University have identified an unprecedented antibiotic ‘megacluster’ in Streptomyces bacteria, harbouring four co-located gene clusters that produce compounds collectively targeting bacterial biotin biosynthesis, uptake and utilisation — with two proving effective against multidrug-resistant E. coli in animal models.
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NewsBlocking cathepsin B protein enhances CAR T-cell therapy effectiveness
University of Maryland researchers have discovered that blocking cathepsin B protein prevents CAR T-cells from losing effectiveness, potentially improving long-term outcomes for blood cancer patients. The preclinical findings reveal that engineered immune cells inadvertently weaken themselves by acquiring tumour fragments, a process that can be prevented through targeted protein inhibition.
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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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NewsMembrane lipid composition directly regulates EGFR activity in cells
MIT researchers have demonstrated that cell membrane composition directly influences EGFR activity, with elevated negatively charged lipids locking the receptor into a growth-promoting state.
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ArticleSmall tags, big advances: studying endogenous proteins in their native context
By combining CRISPR knock-in with small peptide tags, researchers can study proteins in their native cellular context, generating more predictive data for translational drug discovery.
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NewsEuropean initiative aims to bring molecular dynamics into mainstream drug discovery
What if the vast amounts of data generated by molecular dynamics simulations could be routinely shared and reused? A new €10 million European initiative aims to do just that, helping researchers gain a deeper understanding of protein behaviour and drug-target interactions.
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NewsPim1 kinase identified as therapeutic target for inflammatory arthritis
Researchers have identified Pim1 kinase as a critical driver of inflammatory arthritis through its regulation of Th17 cell differentiation via mitochondrial metabolism.
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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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NewsNew kidney water regulation mechanism could improve polycystic kidney disease treatment
Scientists at Mayo Clinic have discovered a previously unknown mechanism by which kidneys regulate water balance, offering new therapeutic possibilities for polycystic kidney disease patients experiencing severe side effects from current treatments.
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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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ReportContext is everything: how spatial biology is changing our understanding of disease
Studying individual cells has revolutionised biomedical research, but it doesn’t tell the whole story. Discover how spatial biology is revealing disease mechanisms with implications for biomarkers, immunotherapy and drug development.
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NewsGlycine transporter suppression restores NMDAR function in autism models
Researchers have identified a novel therapeutic strategy for autism spectrum disorder by suppressing the glycine transporter SLC6A20, successfully restoring NMDA receptor function in both mouse models and human cortical organoids carrying SHANK2 and SHANK3 mutations.