Research on the molecular targets that drugs act on. Covers new antibiotic mechanisms, drug discovery competitions, bile-acid pathways, and Alzheimer's targets.
A phase 1 trial of ivosidenib against IDH1+ acute myeloid leukemia (AML) achieved an overall response rate of 41.9% and median progression-free survival of 8.2 months. Twenty-four percent of patients reached a complete response.
A novel data-driven approach called PanDrugs prioritizes cancer treatments based on patient genetic alterations. The method integrates pathway context and collective gene impact to provide therapeutic options for patients with limited druggable genes.
Scientists at Charité and Stanford University decipher the molecular step of cellular signal transmission involving G protein-coupled receptors (GPCRs) and arrestin. The study's findings could lead to the development of specific drugs targeting diseases like asthma, schizophrenia, hypertension, and cancer.
A study by University of Warwick and Manchester researchers reveals that small rises in temperature speed up a cellular 'clock' controlling the response to infections. This new understanding could lead to more effective drugs targeting A20 protein, which regulates inflammation.
Researchers have discovered a non-canonical pathway that triggers the opening or closing of ion channels through a zipper-like mechanism. This finding offers insights into how these channels regulate vital processes such as heartbeat and pain transmission.
New research reveals that young people in the US are increasingly choosing marijuana as their first drug, with a significant increase in use over the past decade. Young men from specific racial and ethnic groups are particularly affected.
Researchers at Scripps Research Institute have discovered a potential new strategy to treat spinal muscular atrophy (SMA) in infants. By understanding how the drug RG-7916 targets RNA mis-splicing, scientists may be able to design more effective therapies for genetic diseases.
Researchers at Hospital for Special Surgery are launching a two-year study to measure the bone effects of secukinumab in men and women with ankylosing spondylitis. The study aims to understand how the drug affects bone metabolism and identify potential benefits for patients with this condition.
A team of researchers has identified approximately 2,600 genes essential to the growth and survival of Plasmodium falciparum, a deadly malaria parasite. These findings could aid in the development of new or improved antimalarial drugs, highlighting key targets for future research.
Researchers found that palbociclib induces substantial changes in the proteasome, degrading proteins required for cell cycle progression and driving cells into senescence. The discovery suggests that proteasomal activity may be an additional mechanism by which palbociclib stalls proliferation.
Researchers developed a method to 'decorate' gold nanoparticles with proteins, allowing drugs to target specific areas in the body. This technology can improve drug delivery and overcome biological barriers.
A new study reveals that cold temperatures can transform white fat cells into 'beige' cells, which perform thermogenesis like brown fat cells. This process could potentially reduce the symptoms of metabolic diseases such as diabetes and obesity.
Researchers at Radboud University Medical Center have identified a unique protein in the malaria parasite's mitochondrion that could be targeted for a new vaccine. The protein, known as 'prohibitin,' plays a crucial role in the parasite's survival and is not present in human cells.
A modeling study estimates that US Food and Drug Administration (FDA) sodium reformulation targets will reduce cardiovascular disease cases, saving lives and costs. The study projects a gain of approximately 1.1 million discounted quality-adjusted life years (QALYs) and $19 billion in societal costs.
A team from Trinity College Dublin discovered two new families of proteins, PALI1 and PALI2, that are vital for embryonic development and controlling cellular identity in complex animals. These proteins help understand why cells look and act differently despite having identical genes.
Researchers solved the structure of a key nervous system protein in complex with various drugs, enabling precise targeting. The discovery aims to develop medications with regulated action and fewer side effects by controlling which proteins are affected.
Researchers successfully treat KRAS-driven lung cancer by targeting insulin and IGF-1 signaling, identifying metabolic vulnerabilities. The study shows that completely blocking the pathway can slow tumor growth, but a second step is needed to fully suppress it.
A massive international study of 520,000 people has identified 22 previously unknown genetic contributors to stroke, shedding light on the complex biology of the disease. The findings suggest that stroke shares common genetic influences with other vascular conditions and may lead to personalized treatments.
Researchers developed a new antimalarial drug that kills mosquitoes, reducing the spread of malaria. Adding high doses of ivermectin to existing drugs increased mosquito mortality by up to 61%.
A new University of Bristol study reveals that two types of nicotinic receptors play distinct roles in long-term memory retrieval and encoding. The study suggests that drugs targeting individual nicotinic receptors could lead to more effective therapies for dementia.
Researchers at the University of Minnesota Medical School discovered an unrecognized cyclic pathway structure within the folate biosynthesis pathway, allowing each drug to enhance the activity of the other. This new mechanism has the potential to open doors for identification of other synergistic drug combinations.
Researchers found that targeted drug treatment before surgery can safely treat women with aggressive tumours using breast-conserving surgery, sparing patients from extensive surgery. This approach resulted in fewer long-term side-effects and improved quality of life for a significant proportion of patients.
Researchers discovered a key step in blood digestion that can be targeted to disrupt hookworm development. Testing quinolones disrupted hemozoin formation, interrupting development and reproduction, offering promise for new treatments against human-infecting hookworms.
A study found that triclosan inhibits target genes in the malaria parasite during two crucial stages of its lifecycle in humans. Triclosan also performed well in tests against resistant parasites, making it a potential dual specificity antimalarial.
Amino acid transporters are essential for capturing nutrients from blood vessels, which can be blocked by specific inhibitors to treat diseases. Blocking these functions can also enhance inhibition or excitation in the brain, a strategy used to treat chronic pain and possibly schizophrenia.
A study by EMBL researchers found that over a quarter of non-antibiotic drugs inhibit the growth of at least one species in the human gut microbiome. This can lead to unintended side effects and potentially contribute to antibiotic resistance. The research team screened 1000 marketed drugs against 40 representative bacteria, revealing ...
An international team of neuroscientists has discovered a basic molecular mechanism that better understands Lou Gehrig's disease, or amyotrophic lateral sclerosis (ALS), by investigating how cells change. This research could lead to new therapies for the debilitating disease.
Researchers have successfully fused living and non-living cells to harness the natural ability of biological cells to process chemicals while protecting them from the environment. This system can lead to applications such as cellular 'batteries' powered by photosynthesis, synthesis of drugs inside the body, and biological sensors that ...
Three NIDA Avant-Garde Award recipients will receive funding to investigate natural killer cell targeting, therapeutic dendritic cell vaccines, and a new platform for HIV-1 vaccine development. These innovative approaches aim to enhance the immune response against HIV in drug users.
A study published in Nature Neuroscience reveals that romidepsin, an FDA-approved anti-cancer drug, restored gene expression and alleviated social deficits in mice deficient in the Shank 3 gene, a key risk factor for autism. The treatment had a profound and prolonged effect, lasting several years in humans.
Scientists at Cold Spring Harbor Laboratory have discovered a way to rein in an overactive protein that drives some aggressive leukemias. The team's CRISPR-based system identifies two enzymes, LKB1 and salt-inducible kinase, as critical for the survival of certain AML cells.
The American College of Physicians recommends a moderate blood sugar control target of 7-8% for most patients with type 2 diabetes. Achieving this level balances long-term benefits with potential harms such as low blood sugar, medication burden, and costs.
A new drug, larotrectinib, has been shown to be effective in treating cancers with specific genetic mutations. The study found that 75% of patients with TRK fusion-positive cancers responded to the treatment, with 13% achieving a complete response.
Researchers have been working on optimizing and reformulating vaccines to target specific immune responses in individuals prone to relapse. Recent clinical trial data suggests that these new formulations may be effective in preventing opioid relapse.
Researchers used cryo-electron microscopy to discover the structure of an enzyme critical for maintaining adequate DNA building blocks in human cells. The human version differs from its bacterial counterpart, suggesting potential for designing antibiotics that selectively block the bacterial enzyme.
Researchers have discovered a key mechanism enabling stroke survivors to lift affected arms by tapping into 'backup' motor systems, bypassing damaged regions. This adaptive strategy involves involuntary activation of the elbow flexor muscles, which can be harnessed for improved movement control.
Researchers used molecular simulations to understand resistance to osimertinib, an effective anticancer drug for non-small-cell lung cancer. The study identified a novel mutation, L718Q, that causes drug resistance by changing the protein's structure, making it harder for the drug to bind.
A recent neutron analysis of glaucoma drugs and their interaction with human carbonic anhydrase II (hCA II) enzyme revealed the impact of temperature, pH, and electrical charge on drug targeting. The study provides new information about hydrogen-bonding networks in hCA II, which may aid in designing more effective cancer treatments.
A Bentley University study reveals the US government invested over $100 billion in basic research that led to 210 new medicines approved by the FDA. The National Institutes of Health (NIH) funded 84 innovative first-in-class drugs, which treat disease through novel biological mechanisms or targets.
Researchers have developed a new method using yeast to create camelid-free antibodies, allowing for faster and more efficient protein structure determination. This breakthrough enables scientists to study complex proteins without the need for llama or alpaca facilities.
A Salk Institute study has discovered that nearly 80 percent of genes exhibit daily fluctuations, with the most rhythmic genes peaking in early morning and late afternoon. This finding has significant implications for understanding circadian disruption's impact on diseases such as depression and heart disease.
A new study suggests adapting the WHO eradication strategy for yaws by implementing multiple rounds of mass drug administration and targeting a broader geographic area. The research found that this approach can capture those not present during the first round and prevent the spread of antibiotic-resistant strains.
A team of researchers from Case Western Reserve University School of Medicine has used cryo-electron microscopy to capture the structure of full-length serotonin receptors for the first time. This breakthrough provides valuable insights into molecular binding sites that could lead to more precise drug design and targeted therapies.
Researchers at H. Lee Moffitt Cancer Center identified a new target to reduce the risk of graft-versus-host disease (GVHD) in stem cell transplants. The JAK2 inhibitor pacritinib has been shown to minimize GVHD while maintaining antitumor response.
A study by Toyohashi University of Technology found that cell membrane components form isolated domains within an artificial lipid bilayer, separated from the surrounding membrane. The findings provide valuable information for understanding membrane protein functions and developing experimental techniques.
A new study published in the Journal of General Internal Medicine found that long-term opioid prescribing among US military veterans decreased by 30% between 2010 and 2016. The decline was largely due to a reduction in long-term opioid use, rather than a decrease in short-term use.
Researchers at Moffitt Cancer Center have discovered a new way to stimulate the immune system to target cancer cells. The study found that activating dendritic cells with a TIM-3 antibody indirectly stimulates T cell anti-cancer activity, leading to reduced tumor growth in mice.
A new study published in eNeuro found that a drug currently being evaluated for depression and suicidal tendencies also alleviates anxiety, fearful behaviors, and memory deficits in rats exposed to blast injuries. The drug, BCI-838, promotes the generation of new brain cells in the hippocampus.
Researchers have discovered that three distinct varieties of the protein AKT play different roles in brain health, with AKT2 targeting brain cancer and AKT1 promoting memory formation. The study's findings hold promise for developing targeted therapies for conditions like Alzheimer's disease and schizophrenia.
A team of MIT researchers has created a miniaturized system that can deliver tiny quantities of medicine to specific brain regions, allowing for more precise treatment of neurological disorders. The device consists of several tubes contained within a needle and can deliver targeted doses with precise control.
Researchers have discovered that acesulfame potassium, an artificial sweetener widely used in foods and drinks, could lead to the development of a more selective therapy for aggressive cancers. The compound is more selective towards carbonic anhydrase IX than previously studied saccharin, potentially offering fewer side effects.
Researchers at Uppsala University have discovered a new strategy to shut down specific enzymes that can help fight cancer. By studying the native structures and mechanisms of dehydroorotate dehydrogenase, they found that lipids play a crucial role in binding drugs to this enzyme.
A team of researchers has described atom-by-atom changes in a family of proteins linked to amyotrophic lateral sclerosis (ALS), a group of brain disorders. The study suggests that small chemical changes can lead to big changes in assembly and disease-associated aggregation, offering new insights into disease mechanisms.
A human protein called prohibitin may play a significant role in infection of the nervous system by EV71 virus. The study found that inhibitin is required for entry and replication of the virus within cells.
A study found that desmoplastic melanoma patients respond significantly to anti-PD-1/PD-L1 therapies, with a 70% response rate. The tumors have high levels of DNA mutations and pre-existing immune cells necessary for an immune response.
A recent study by Queen Mary University of London found that protein markers are a more accurate predictor of patient responses to midostaurin, a new leukemia treatment. This breakthrough could lead to double the number of patients being treated and improve outcomes for those with acute myeloid leukemia.
Researchers have discovered a link between ADCY3 gene mutations and severe obesity, which can lead to appetite control abnormalities, diabetes, and anosmia. This finding offers new hope for the development of targeted treatments for obesity.
A team of researchers at Stanford University School of Medicine identified four on-the-market drugs that successfully quelled symptoms triggered by several overabundant proteins in patients with a rare eye disease. The findings demonstrate the potential of treating rare and complex eye conditions using precision health approaches.
IRB Barcelona researchers have discovered a new target for treating advanced prostate cancer, the TFIIF protein. Removing this protein's interaction with the androgen receptor could make treatment effective again in resistant tumors.
A study has identified an agent that can reverse resistance to a targeted therapy in some cases of leukemia. Azacitidine was found to reactivate cells that had become resistant to the treatment SL-401 by reversing DNA methylation, making them vulnerable to the drug again.