Research on the molecular targets that drugs act on. Covers new antibiotic mechanisms, drug discovery competitions, bile-acid pathways, and Alzheimer's targets.
Scientists at the University of Exeter have discovered two new mechanisms for how a well-established antibiotic works, which could lead to potential development of new antibiotic treatment. The study found that doxycycline targets the bacterial ribosome by blocking protein exit channels or reconfiguring it into an inactive state.
Revolver Therapeutics, a spin-out company from the University of Bath, has won the Health category of the Royal Society of Chemistry's Emerging Technologies Competition with its proprietary discovery platform. The platform generates and tests millions of constrained peptide drug candidates directly inside living cells, selecting for mo...
A buildup of bile acids caused by an unhealthy gut can drive breast cancer to other parts of the body, researchers reveal. The study found that replenishing beneficial bacteria or treating patients with FDA-approved bile acid sequestrants may reduce breast cancer metastasis.
A USC interdisciplinary team has identified a promising pathway to turn off brain inflammation before it does its damage, increasing the risk of late-onset Alzheimer's. The Norman and Mary Pattiz Foundation funding will support research on novel drug discovery, early detection, and prevention of Alzheimer's disease.
A new study found that intranasal davunetide reaches the brain in higher amounts in female mice when estrogen peaks, with similar results seen in healthy adults. The findings suggest earlier trials mixing males and females may have obscured the true effects of the drug.
Researchers at the University of Houston have discovered adding salt can help release therapeutic material from endosomes, improving delivery efficiency for mRNA vaccines and gene therapies. The simple strategy overcomes a major obstacle in gene medicine, potentially making these therapies more efficient and accessible worldwide.
The University of Virginia has joined SPARK GLOBAL to leverage resources and expertise in accelerating the development of new medicines. This collaboration aims to reduce time from lab discoveries to clinic trials, delivering tangible healthcare solutions to patients.
The International Society for Stem Cell Research announces the launch of a global consortium focused on integrating human stem cell-derived models into preclinical drug development. Bayer is a founding member, aiming to establish standardized frameworks and regulatory guidance for these models.
Researchers identify key mechanisms and molecular targets to prevent tumor progression in patients with rhabdomyosarcoma, a rare soft tissue cancer affecting young children. Two new treatment possibilities have been highlighted, targeting the IRE1α-XBP1 signaling axis and protein TAK1.
Researchers found that temperature and calcium levels inside cells can dramatically impact how drugs interact with their targets. This discovery could lead to smarter medicine design and more effective treatments.
Researchers at EPFL have discovered a way to generate and screen large libraries of synthetic cyclic peptides that can both enter cells and block disease-related protein interactions. This breakthrough could broaden access to previously difficult targets for drug development, enabling the creation of orally available peptide drugs.
Researchers at UW Medicine Institute for Protein Design and Skape Bio used AI methods to create on-demand molecules that can toggle GPCRs. The approach enables precise control of GPCR signaling in cells, offering new insights into bodily functions and potential medicines for diseases.
The ISSCR Consortium supports the FDA's draft guidance on using new approach methodologies (NAMs) in drug development, emphasizing the need for a flexible and science-driven framework. The consortium recommends clarifying biological complexity alignment with context of use and expanding recognition of computational modeling and hybrid ...
Scientists have identified a new drug target for treating Fragile X syndrome by blocking the EPAC2 brain protein, which improves abnormal brain activity and behavioral symptoms. The study uses genetically engineered mice to simulate the condition and finds that EPAC2 levels rise gradually as the brain matures.
A team of researchers has uncovered a promising new target for antimalarial drug design, identifying an enzyme called aminopeptidase P from the Plasmodium falciparum parasite. The new inhibitors have been shown to bind more strongly and selectively than existing compounds, demonstrating potential as a new class of drugs to combat malaria.
A team of scientists has uncovered more than 1,700 new proteins known as peptideins, which are smaller than traditional proteins and may have unique biology. These proteins were found in the 'dark proteome', a section of DNA previously overlooked, and could have implications for human diseases like cancer.
A recent NIH-funded study reveals that oral small-molecule GLP-1 drugs can suppress eating for pleasure and reduce cravings by targeting a deeper brain region, potentially treating other dysfunctions in reward processing. The findings provide insight into the neural mechanisms underlying the effects of these medications.
Researchers have discovered a biomarker for chemotherapy resistance in small cell lung cancer, which can help identify cells that become more invasive and lead to treatment resistance. Targeting these cells with YAP1 may be a possible strategy to improve patient outcomes.
A new study published in Science Advances identifies IRS4 as a promising drug target for multiple solid tumors, offering hope for safer cancer treatments. By using AI and natural mutations, researchers prioritized targets with high therapeutic indexes to minimize toxicity.
Researchers have identified a gene, eIF4G2, crucial for keeping adult intestinal stem cells stable and functional. The study reveals that the gene plays a vital role in regulating protein production and maintaining stem cell identity.
A research consortium has developed mini-antibodies that can reactivate mutated p53, a key tumor suppressor protein. These DARPins bind selectively to p53 mutants, restoring stability and functionality, making them potentially useful against various types of tumors.
The Phase I MYTHIC trial demonstrated a strong synergy between zedoresertib and lunresertib, showing durable regressions and consistent tumor shrinkage in patients with ovarian cancer. The combination achieved an overall disease control rate of 68.5% and a molecular response rate of 47%.
Researchers Dr Íris Luz Batalha and Dr Maria Shchepinova from the University of Bath have been awarded funding to test new ideas in tackling global health challenges. They will develop precision-targeted therapies for antimicrobial resistance and investigate why treatments for Type 2 diabetes don't work for everyone.
Scientists at the University of Virginia Health System have developed a suite of AI-powered tools, called YuelDesign, YuelPocket and YuelBond, to transform how new drugs are created. These tools can design drug molecules tailored to fit their protein targets exactly, even accounting for protein flexibility.
Researchers have identified a framework to characterize RNA structure-changing small molecule drugs, which could lead to the development of better treatments targeting RNA directly. The study found that molecules binding to RNA rarely affect its function, while those altering its structure have a bigger impact.
Lawrence Toll has been named a senior member of the National Academy of Inventors for his pioneering work on pain pathways, reward systems, and potential treatments. His research focuses on identifying new drug targets and therapeutic strategies to combat chronic pain and addiction.
Researchers developed a machine-learning system that predicts how molecules form, cutting lab work time from months to days and reducing costs. The system uses asymmetric cross-coupling reactions to build complex compounds and can be applied across fields, deepening our understanding of chemistry.
The Alliance for Clinical Trials in Oncology is spotlighting new trials for colorectal cancer in March, focusing on early detection methods and treatments for treatment delays and loss of appetite. The trials aim to improve patient outcomes, with several enrolling patients with newly diagnosed colon or rectal cancer.
Researchers at Goethe University are developing non-hormonal contraceptives to address declining pill use and side effects. The PREVENT project aims to create safe and effective alternatives, focusing on small molecules that block proteins in sperm or egg cells.
A study from The University of Osaka reveals a molecular pathway connecting liver congestion to liver fibrosis, portal hypertension, and liver tumorigenesis. Researchers identified increased activity of YAP and CTGF in liver sinusoidal endothelial cells as key molecules involved in this signaling pathway.
A new study finds that a novel GLP-1 receptor agonist, Exendin-4-Phe (Ex-Phe-1), preserves glycemic control while reducing malaise and vomiting behaviors in preclinical models. The compound uses biased agonism to selectively activate certain signaling pathways, achieving desired effects without triggering others.
Researchers developed a novel method to immobilize proteins onto magnetic microbeads, allowing precise measurement of binding strength and efficient selection of target peptides. The technique achieved a 10,000-fold concentration in a single sorting step, significantly enhancing the efficiency of drug discovery research.
A study published in PNAS successfully eliminated pancreatic tumours in mice completely and durably, with no significant side effects. The treatment, combining three molecular targets, induced robust regression of experimental PDACs without causing tumor resistance.
Researchers are expanding options for patients with hard-to-treat cancers using a personalized approach that identifies effective therapies by testing large libraries of drugs on living tumor cells. The approach has shown stronger responses than initial findings, suggesting it can reveal opportunities missed by traditional methods.
Researchers uncover a key ion channel, TRPM4, that regulates intestinal fluid balance and identify a new druggable site. This discovery provides a blueprint for designing targeted treatments for gastrointestinal disorders.
A new nanodrug called Nano-273 could offer improved survival for patients with pancreatic and lung cancers by activating the immune system and blocking tumor growth. The drug, developed by University of Houston researcher Wei Gao, has shown promising results in early studies.
Researchers at Harvard Medical School have uncovered crucial insights into how a new class of antiviral drugs works, shedding light on an important tool for fighting drug-resistant strains of herpes simplex virus. The discovery may lead to new pathways for treating herpesviruses and other kinds of DNA viruses.
Researchers develop experimental drugs that encourage mitochondria in cells to work harder and burn more calories. The findings offer a framework for designing safe and effective weight-loss treatments with potential benefits for metabolic health and neurodegenerative diseases.
Researchers at the University of Exeter have discovered a genetic process in Candida auris that could unlock new ways to treat the deadly fungal infection. The study found that genes activated during infection include those that code for nutrient pumps, potentially making it vulnerable to iron-based drugs.
In a new study, Northwestern scientists identified a previously unknown toxic sub-species of amyloid beta oligomers that drive brain changes in Alzheimer's disease. NU-9 decreased this toxin and reduced damage in a mouse model, suggesting it could prevent or delay the cascade of toxic events that destroy neurons.
Scientists have discovered a shape-shifting molecular valve in PANX1, a cellular gate that controls the flow of chemical messages. Researchers found that a common antimalarial drug can enhance or inhibit this gate's activity, paving the way for precision therapies.
A specific protein, RASH3D19, activates the RAS signaling pathway involved in aggressive tumor growth and resistance to KRAS inhibitors. Blocking RASH3D19 improves outcomes in preclinical models, suggesting a potential therapeutic strategy.
A new computational tool called DeepTarget predicts direct and indirect targets of cancer drugs, revealing that small molecules can have different targets and effects depending on the disease and cell type. The study demonstrates the tool's superior performance in real-world scenarios, highlighting its potential to accelerate drug deve...
Researchers at UVA School of Medicine found that the breakdown of protective brain structures called perineuronal nets leads to social memory loss in Alzheimer's patients. Preventing the degradation of these nets may lead to new treatments to prevent or delay the disease.
The FDA has approved elinzanetant for treating hot flashes and night sweats in postmenopausal women. The drug significantly reduces the frequency and severity of symptoms while improving sleep quality and quality of life.
A University of Houston professor has received funding to develop effective treatments for Cryptosporidium infections, which cause severe diarrhea and have no existing cure. The team aims to design drugs that target the parasite's enzyme CDPK1 to minimize systemic exposure and maximize efficacy.
Researchers at the University of Bath develop a peptide fragment that locks alpha-synuclein into its healthy shape, blocking toxic clumps that cause nerve cell death. The breakthrough demonstrates the potential of rational peptide design to transform large proteins into compact drug-like molecules.
Researchers identified a promising treatment for PTSD, chronic pain, and alcohol misuse in rats. The study found that PPL-138 selectively reduced anxiety-like behavior, pain responses, and alcohol consumption in those with trauma-related anxiety.
A large international clinical trial found that elinzanetant significantly reduces hot flashes and night sweats in postmenopausal women by over 73%. The drug also shows secondary benefits such as improved quality of life and reduced sleep disturbances, with no harmful effects on the liver or bone density.
A Kobe University study found that metformin reduces copper and iron levels and increases zinc levels in patients with type 2 diabetes. This suggests a possible mechanism for the drug's beneficial effects beyond lowering blood sugar levels.
Scientists at CUNY ASRC develop novel synthetic carbohydrate receptors that block infection from seven different viruses across five unrelated families, including Ebola and SARS-CoV-2. The breakthrough offers a promising path toward the development of broad-spectrum antivirals.
A new imaging approach allows researchers to visualize how dual-acting diabetes and obesity drugs like tirzepatide interact with cells in the pancreas and brain. The tool, called daLUXendins, provides insight into the metabolic effects triggered by these drugs, which stimulate both GLP-1R and GIPR receptors.
Elevated cdc42 activity is a critical initiation event leading to proteinuria in nephrotic syndrome. Suppression of cdc42 activity could be a promising therapy.
Researchers validated panels of antibodies targeting clinically relevant nucleic acid modifications to visualize antisense oligonucleotides in both in vitro and in vivo studies. The tools enable detection of modified nucleic acids irrespective of sequence, facilitating multiple clinical and pre-clinical workflows.
Researchers at the University of Arkansas have created a new controlled release system that uses cellulose nanocrystals and alginate to deliver bioactive compounds to specific areas of the body. The system protects medications from acid in the stomach and releases them in alkaline environments, such as the intestines.
A new treatment for corneal scarring is being developed by University of Houston optometry researcher Tarsis G. Ferreira. The treatment uses a natural protein called decorin to block scarring and unwanted blood vessel growth, offering hope for people with injured corneas.
A potent new dual lipid kinase inhibitor, RMC-113, selectively inhibits PIKfyve and PIP4K2C, providing a potential strategy to combat emerging viruses. The study reveals that PIP4K2C plays a crucial role in SARS-CoV-2 entry, replication, and assembly/egress, making it an understudied kinase with significant antiviral potential.
Scientists from the University of Bath have identified two new families of chemical compounds that inhibit alpha-methylacyl-CoA racemase (MCR) in Mycobacterium tuberculosis, a key enzyme for TB survival. This breakthrough could lead to new treatments for TB and potentially other diseases like prostate cancer.
Researchers at King's College London used cryo-electron microscopy to study the flagellum in unprecedented detail, revealing its architecture and identifying potential drug targets. This breakthrough could lead to the development of new treatments for bacterial infections without driving resistance.
Dr. Danielle Beckman's research uses animal models to understand how viruses like COVID-19 trigger neurological damage and accelerate Alzheimer's disease. Her work has established critical connections between viral infections and neurodegenerative processes.