Research on the molecular targets that drugs act on. Covers new antibiotic mechanisms, drug discovery competitions, bile-acid pathways, and Alzheimer's targets.
Researchers combined CRISPR with drug discovery to understand how AML treatment works and which weaknesses can be exploited. The study revealed that LSD1-GFI1B relationship is critical for AML survival, enabling more targeted treatments.
A researcher at the University of Texas at Arlington has received a four-year grant to investigate protein regulation in programmed cell death. Understanding how these proteins are regulated could lead to drugs targeting specific cancer cells.
Researchers report a link between methamphetamine self-administration and imbalance of brain circuits responsible for addiction. Noninvasive brain stimulation may help control compulsive drug use in humans by targeting these circuit imbalances.
A new international protected area target is necessary to effectively conserve biodiversity, according to a study published in Science. The current target has led to perverse outcomes, with nations unable to account for real progress.
Researchers used CRISPR technology to disrupt every gene in over 300 cancer models from 30 cancer types, discovering thousands of key genes essential for cancer's survival. The team developed a system to prioritize and rank 600 drug targets promising for development into targeted cancer treatments.
A new cellular mechanism for amyotrophic lateral sclerosis (ALS) has been identified, suggesting a novel therapeutic strategy targeting the RNA degradation pathway. Researchers also found that an asthma drug called Tranilast could potentially rescue cells and fruit flies from C9orf72-induced neurotoxicity.
Hepatitis C transmissions in high-income countries could be reduced by up to 79%, with lower rates in low- and middle-income nations. New antiviral treatments have become available, but widespread adoption of prevention measures is crucial for meeting the World Health Organization's elimination targets.
A recent study in animal models of diffuse intrinsic pontine glioma (DIPG) has identified an experimental drug that effectively destroys DIPG cells by depleting cellular cholesterol. The researchers also found that the compound works by directly inhibiting lanosterol synthase, an enzyme involved in cholesterol production.
Researchers have captured the full-length structure of PDE6, an enzyme linked to vision problems and potential side effects from erectile dysfunction medications like sildenafil. The study reveals fish-hook-like regions that could be targeted for new inhibitors.
A new UK-based study reveals that alcohol, cannabis, MDMA, and cocaine are the most commonly combined with sex. The research found that people of all genders and sexual orientations engage in substance-linked sex, but gay and bisexual men are more likely to do so.
Researchers develop drugs targeting specific component of folate pathway, potentially addressing drug resistance and treatment time. The compounds are more effective than existing antifolate medication PAS, offering hope for new treatment options.
A team of researchers has identified a 'druggable' mechanism for pathological tau protein aggregation, which could lead to new treatment options for devastating neurodegenerative diseases. The discovery focuses on the RASD2 gene and its potential as a target for farnesyl transferase inhibitors.
Despite advancements in disease research, neglected tropical diseases and others continue to require attention, with only a small proportion of new drugs addressing these conditions. Novel treatments like bedaquiline and tafenoquine offer hope, but much work remains to be done.
A new test for identifying how single bacteria react to antibiotics could help slow down antibiotic resistance by targeting the right treatments more quickly. The test, which can analyze hundreds of bacteria at once, examines multiple properties and detects the signature of each bacterium in one hour.
A novel machine-learning model uses artificial neural networks to analyze protein sequence data, providing detailed information on protein structure, function and evolutionary features. The model can be used to design new proteins with desired functions and predict the future sequence evolution of proteins in living organisms.
Researchers have discovered a potential new drug that can minimize heart muscle death caused by stress signals after a heart attack. The treatment, targeting protein MAP4K4, reduced damage by 60% in mice and has the potential to be developed into an injection for routine clinical use.
Researchers have found a pair of old drugs that can disrupt disease-causing pathways in triple negative breast cancer. Metformin and heme, previously used to treat diabetes and porphyria, were tested in mice and produced encouraging results when combined.
Researchers identified S15 as a major immune suppressor in B7-H1-negative tumors resistant to current anti-PD therapies. A first-in-human Phase 1/2 clinical trial is underway for NC318, a monoclonal antibody targeting S15.
Researchers developed 13 bioluminescence sensors to measure intracellular signaling pathways and detect pharmaceutical action. The biosensors provide a complete answer, or 'signaling profile,' allowing for more accurate drug testing and development.
Researchers have successfully determined the crystal structure of the human type 2 cannabinoid receptor, enabling the design of more efficient drugs targeting this receptor or both CB1 and CB2 receptors.
A new injectable bone healing drug has been developed to accelerate fracture healing by 50% and reduce recovery times. The technology, licensed through Purdue University's Research Foundation, has shown promising results in preclinical studies and is expected to transform treatment for broken bones.
Researchers at UCLA have developed a method to screen hundreds of drugs in patients' own cells, creating miniature tumor organoids. This approach can help identify effective treatments for rare and hard-to-treat cancers, and could guide treatment decisions.
Researchers found patients born without a CCR5 gene recover better from mild stroke than those with the gene. Maraviroc, a drug blocking CCR5, accelerated recovery in mice and boosted motor skills, language, and sensory function.
Researchers have developed a new approach to create enzyme-responsive nanostructures that can sense overexpression of MMP-9, helping diagnose cancer and autoimmune diseases. The design guidance enables the creation of smart-drug delivery vehicles with precisely tunable properties.
A research team at the Medical University of South Carolina has discovered a novel binding site for E1 inhibitors, which could lead to the design of more efficient anti-cancer drugs. The study reveals a new mechanism for promoting drug design and specificity.
A new medicine under development, vamorolone, shows improved safety in treating both inflammation and heart disease in experimental models of DMD. This is significant because the current standard of care, prednisone, reduces chronic inflammation but has harsh side effects.
Researchers at OHSU discovered a gene linked to lower expression in primates who consumed heavy amounts of alcohol, which reduced consumption by almost 50% when increased in mice. The study highlights a novel target for treating alcoholism and mood disorders.
A study by MGH researchers reveals that brain tumors use existing blood vessels to resist anti-angiogenic drugs, leading to compression and stimulation of angiogenesis. The study suggests targeting vessel co-option before using anti-angiogenic drugs could be an effective strategy for glioblastoma treatment.
A first-in-human study demonstrated the ability of a new class of antisense oligonucleotide therapeutics to target the liver, resulting in improved potency and safety at therapeutic doses. The study showed that the conjugated drug was up to 30-fold more potent than the parent antisense oligonucleotide.
Researchers at Brigham and Women's Hospital have discovered two new genetic targets, APEX2 and FEN1, which show promise as potential treatments for hereditary breast and ovarian cancers. The study's findings suggest that inhibiting these enzymes could complement existing Parp inhibitors and address drug resistance in BRCA-driven cancer.
A large-scale genetic study has uncovered 52 new genetic changes linked to osteoarthritis, which may help identify new treatment targets and repurpose existing medications. The study analyzed the genomes of over 77,000 people with osteoarthritis and found potential avenues for drug exploration.
Researchers identified a new process capable of generating antibiotic resistance in bacteria up to 600 million years ago. The discovery highlights the need for combined multidrug treatments and reduced agricultural use of antibacterials.
New research reveals a link between immune checkpoint inhibitors and the development of musculoskeletal and rheumatic diseases. The most commonly reported adverse events include arthralgia, myalgia, sicca syndrome, polymyalgia rheumatica, and systemic lupus erythematosus.
A new USC study found that blood-brain barrier breakdown is an early driver of dementia, with leaky capillaries in the brain signaling cognitive impairment before hallmark toxic proteins appear. The study suggests new targets for drugs to slow down or prevent dementia's onset.
A landmark study published in the New England Journal of Medicine has found that ibrutinib is significantly more effective than standard therapy in treating CLL, causing fewer side effects and requiring less frequent patient visits. Ibrutinib was shown to improve survival rates by 13% compared to standard treatment.
Scientists have successfully mapped the active-state structure of the angiotensin II type 1 receptor, a critical drug target for regulating blood pressure and kidney function. The study provides insights into how this receptor activates and offers clues for developing new medications that activate rather than block the receptor.
A Penn study reveals that individual genetic variation can predict how well patients respond to certain anti-diabetes drugs, including thiazolidinediones. The researchers identified a specific genetic variation associated with increased cholesterol levels in response to rosiglitazone treatment.
A recent study characterizes silent seizures in a mouse model of Dravet syndrome and identifies the thalamus as a potential target to stop them. The researchers developed two new strategies to prevent non-convulsive seizures, which can disrupt consciousness and occur hundreds of times daily in affected children.
Recent advances in computational methods enhance the accuracy and reproducibility of label-free quantification (LFQ) in cancer proteomics, enabling discovery of anti-cancer targets and drugs. The study evaluates popular acquisition techniques and state-of-the-art quantification tools to improve LFQ performance.
A class of breast cancer drugs could potentially benefit patients with EGFR-mutant lung cancers that have become resistant to treatment. Lung tumours in mice caused by mutations in a gene called EGFR shrunk significantly when a protein called p110α was blocked.
Research reveals flavonoids as potent P-gp modulators with high binding affinity and low toxicity, offering a new generation of drug candidates to reverse multidrug resistance. Synthetic flavonoid dimer FD18 specifically targets the pseudodimeric structure of the drug transporter.
Research identifies GM3S and GM3 ganglioside as crucial for intestinal cholesterol uptake, offering a new therapeutic target for reducing high blood cholesterol. A high-cholesterol diet-fed mouse model with deficient GM3S shows lower susceptibility to high blood cholesterol.
Researchers developed a new dielectric blood coagulometry (DBCM) method to assess Factor Xa (FXa) activity in patients treated with FXa inhibitors. The study showed that DBCM detected FXa inhibitor-specific changes in a manner similar to more complicated methods, offering a promising easy-to-use clinical treatment option.
Researchers have reviewed recent developments in monoamine transporter inhibitor design, including structure-activity relationships and binding modes. The study highlights potential applications for these inhibitors in treating depression and behavioral disorders.
Researchers discovered that a drug initially developed for Alzheimer's disease has antibacterial properties, potentially breaking antibiotic resistance in bacteria. The findings suggest the drug could be repurposed to treat infectious diseases.
A comprehensive review highlights novel approaches to slow or prevent Alzheimer's disease, focusing on the effects of aging on the brain. Combination therapy is deemed necessary for better treatment outcomes, similar to other major diseases.
Biologists have visualized the inner workings of cellular 'undertaker' proteasomes using cryo-electron microscopy. The study reveals how ATP powers movements within the motor that enable it to pull in proteins, providing insights into neurogenerative diseases like Parkinson's and Alzheimer's, as well as cancer therapy.
A study led by Flinders University found that removing the RCAN1 gene in mice prevented weight gain despite a high-fat diet. Blocking this gene may transform unhealthy white fat into healthy brown fat, presenting a potential treatment method against obesity. Further studies are needed to confirm these results in humans.
Scientists at USC and SLAC develop a 3D map of the structure of a cell receptor as it binds to misoprostol, a key drug for women's health. This research provides a starting point for new drug discoveries that could reduce maternal mortality rates.
Researchers have created an in silico inventory of proteins on cell surfaces using machine learning, predicting the presence of over 2,900 proteins on human cell surfaces. The study reveals a wide variety of surface proteins across different cell types, with primary stem cells showing the greatest diversity.
Researchers have created a new drug that targets the colon and limits its exposure, offering hope for treating Familial Adenomatous Polyposis (FAP) and preventing colon cancer. The treatment could be effective in reducing life expectancy by nearly 50% for children with FAP.
Researchers identified a gene mutation in SGLT1 that helps protect against spikes in glucose levels, resulting in lower incidence of obesity, diabetes, death, and heart failure. The study's findings suggest potential therapeutic targets for metabolic disease.
A recent trial found that low-dose methotrexate did not lower cardiovascular event rates in patients at high risk for heart disease and stroke. The study, which included over 4,700 participants, showed no significant reduction in major adverse cardiovascular events compared to placebo.
Researchers developed a targeted therapy that enhances fracture repair in vivo, reducing healing time by 60% without impacting surrounding healthy tissue. The therapy, DAC, was found to double bone density and improve treatment intervals, offering potential benefits for the orthopedic community.
Researchers developed a method to release drugs from nanoparticle cages in small areas of the brain, reducing neural activity. The technique uses focused ultrasound to jiggle drug molecules loose and has potential for treating various conditions, including cancer.
The largest genomic study of parasitic worms has identified hundreds of thousands of new genes and predicted many new potential drug targets. The research could lead to new de-worming treatments to help prevent and treat the diseases caused by parasitic worms worldwide.
Researchers have identified a new way to target and degrade a class of proteins called zinc finger transcription factors, which play critical roles in health and disease. By modifying thalidomide analogs, scientists can selectively degrade specific zinc fingers, offering a promising lead for developing new cancer treatments.
Researchers identified potential cancer drugs that disrupt normal chromosome division in cancer cells and induce errors when dividing DNA, posing double trouble for tumor growth. This new approach could inform the development of more effective therapies targeting various types of cancers.
Research suggests that prenatal exposure to most common medications does not increase the risk of autism, contrary to previous studies. Instead, a mother's general health before pregnancy appears to play a more significant role in her child's development.
Researchers have identified potential drug targets for rhabdomyosarcoma, a rare and aggressive cancer affecting mostly children. A new compound, adavosertib, showed promising results when combined with chemotherapy.