Research on the molecular targets that drugs act on. Covers new antibiotic mechanisms, drug discovery competitions, bile-acid pathways, and Alzheimer's targets.
A new computational tool, parabolic personalized dosing (PPD), successfully tested in a pilot trial accurately predicts the optimal drug dose for an individual patient. PPD harnesses clinical data to account for various factors influencing drug response, including age, ethnicity, and genetics.
A high-throughput RNA interference screen identified fibrillarin as a crucial host protein required for live henipavirus infection in human cells. This discovery suggests that methyltransferase enzymes, including fibrillarin, represent a potential target for the development of anti-henipavirus drugs.
Researchers identified a human enzyme called fibrillarin as crucial for henipavirus infection, suggesting it as a potential therapeutic target. The study's findings have implications for the development of an anti-henipavirus drug, and may also offer broader use against other paramyxoviruses.
Researchers have developed a drug that can erase epigenetic markers on chromatin to restore the original state of stem cells in mice. The study shows that over half of mouse epiblast stem cells treated with the drug regained embryonic pluripotency, opening up new possibilities for regenerative medicine.
Tracy Clinton and Michael Thompson received the Protein Society's Year 2015 'Best Paper' awards for their work on Ebola drug target mimics. The researchers, who came from diverse backgrounds, were chosen for their innovative approaches to solving complex protein problems.
A new study from MIT reveals that certain cancers develop a backup system to evade treatment, which can be detected in blood samples. Combining kinase inhibitors with another drug shows promise in improving results, according to researchers.
Researchers found a maximum reduction of 52.4% in dry-eye symptoms using the repurposed drug, with some patients reporting complete resolution of symptoms. The treatment has antiangiogenic effects and offers distinct advantages over existing surgical removal methods.
Scientists at University of Utah Health discovered that spironolactone, a medicine for heart failure, can also inhibit Epstein Barr virus by blocking a key step in viral infection. This finding has broader implications, suggesting that spironolactone could be developed into a new class of anti-herpesvirus drug.
Researchers developed insights to guide powerful new anti-influenza drug development by analyzing artificially created drug-resistant flu strains' molecular details. The compounds target the endonuclease active site, a region where viruses can mutate to evade existing drugs.
A new study is exploring whether the drug Omalizumab can be targeted at specific patients with severe asthma, who are expected to benefit most from the treatment. This research aims to identify biomarkers that will enable more effective treatment and improve symptoms for those suffering from severe asthma.
Researchers develop vaccine to prevent fentanyl overdose and addiction by targeting its molecular structure, which can be tailored to neutralize various variants of the drug. Successful preclinical tests show the vaccine protects against lethal doses of fentanyl, providing new hope in combating the opioid crisis.
Researchers at Vanderbilt University Medical Center have discovered a new compound that is more effective against the tuberculosis enzyme and maintains activity against resistant forms. The findings could lead to a more effective treatment for the deadly disease, which killed 1.5 million people in 2014.
UPMC's PreCISE-Rx program uses pharmacogenomics to individualize patient treatments, targeting the right drug for each patient based on their genes. The goal is to reduce hospital readmissions and recurrent illness while improving treatment outcomes.
A new robotically driven experimentation system has been developed to determine the effects of a large number of drugs on many proteins, reducing the number of necessary experiments by 70%. The model uses an active learning approach to identify patterns and make predictions about unmeasured experiments with high accuracy.
A team of scientists has produced a structural movie showing the creation of S-Adenosylmethionine (SAMe), a major methyl donor in the body that plays a role in some cancers. The research provides insight into how this enzyme synthesizes SAMe and highlights it as an excellent therapeutic target for cancer treatment.
A team of Dutch investigators has identified NS4B as a potential target for antiviral drug development against dengue virus. A metabolite of acetaminophen, AM404, inhibits replication of the virus. The researchers found that mutations in the viral NS4B protein render the virus insensitive to AM404.
Researchers are exploring the anti-inflammatory drug's ability to improve cognition in patients with stable schizophrenia or schizoaffective disorder. The study aims to enroll 30 patients and examine the effects of siltuximab on cognitive impairment, which is a significant unmet need in treating schizophrenia.
Depression patients with systemic inflammation have elevated glutamate levels in motivation regions of the brain, which may guide personalized treatment. Elevated glutamate and myo-inositol levels are associated with anhedonia and slow motor function, suggesting a potential target for depression treatment.
Scientists have developed a new strategy for constructing highly specific synthetic pores using DNA, which can control the release of therapeutics. The pores are designed to act like doors, opening only when provided with the right key, allowing for precise targeting of drugs to specific tissues.
Researchers suggest GPR119 could treat nonalcoholic fatty liver disease, with administration of its ligand reducing hepatic lipid accumulation in mice. This discovery opens the door to developing new treatments for this increasingly prevalent disease.
Researchers discovered a novel mechanism of resistance and re-sensitization to first-generation ALK inhibitors in a patient with metastatic non-small-cell lung cancer. The study highlights the importance of repeat biopsies and molecular profiling to uncover novel resistance mechanisms.
A global research team has identified 52 genetic variants associated with age-related macular degeneration (AMD), a disease affecting 150 million people worldwide. The discovery may lead to personalized medicine approaches and new treatments for this debilitating condition.
A team at Cold Spring Harbor Laboratory has discovered a novel drug that targets an abnormal RNA molecule called Malat1 in metastatic breast cancer. The drug reduces tumor growth by 70% and prevents metastasis, suggesting that targeting this RNA may be a promising therapeutic approach for treating aggressive breast cancer.
Researchers have developed models to rationalize the action of chemotherapeutic molecules on body cells, enabling better understanding of how drugs interact with biological targets. The study aims to improve the design of new therapeutic molecules, resulting in more effective and side-effect-free drugs.
Cardiff University scientists have engineered a new dual 'homing' agent that inactivates the complement system in the brain, reducing inflammation and aiding recovery. The treatment has shown promise in mice with traumatic brain injuries, potentially leading to life-changing benefits for humans.
A new study suggests that pomaglumetad methionil, a glutamate pathway target, may be an effective treatment for individuals in the early course of schizophrenia. Researchers found a statistically significant response in patients who are early-in-disease or have not already been exposed to other antipsychotic medications.
The Target Validation Platform provides a single, robust infrastructure that integrates high-level information from key sources of evidence. It enables communities to work together, making the hand-off from basic research to drug discovery smoother, and is expected to grow substantially as it integrates experimental project data.
A recent study published in the New England Journal of Medicine found that ibrutinib, a targeted drug, was superior to chlorambucil in treating chronic lymphocytic leukemia (CLL) patients. The study showed improved progression-free survival, overall response rate, and overall survival rates for patients taking ibrutinib.
Researchers have created a detailed image of pneumolysin, a toxin linked to deadly infections like pneumonia and meningitis. The structure reveals how the toxin assembles on cell surfaces, enabling the development of novel therapeutic approaches to block its formation.
A University of South Florida research team developed a novel chemogenomic profiling approach to identify drug targets for the most lethal strain of malaria. The study identified six new genes critically involved in P. falciparum's response to artemisinin, providing insights into the drugs' mechanisms of action.
Scientists at UNC and UCSF developed a general tool to find homes for 'orphan' cell-surface receptors, illuminating their roles in behavior. The creation of the research tool allows researchers to learn how orphan receptors interact with molecules inside the body or with drugs.
Scientists at UNC and UCSF create general tool to probe orphan receptor activity, illuminating their roles in behavior. They found a novel probe molecule that can modulate the orphan G protein-coupled receptor 68 (GPR68), which is highly expressed in the brain.
Recent SPRINT trial results suggest lowering systolic blood pressure below 120 may be associated with a lower incidence of cardiovascular disease and reduced mortality by 25%. The study's findings, published in the New England Journal of Medicine, may lead to a reevaluation of blood pressure goals for non-diabetic individuals over 50
A new study suggests that protected areas are failing to meet biodiversity goals due to inadequate science and management. The Wildlife Conservation Society proposes a three-point plan to improve the effectiveness of protected areas, including establishing ecologically sensible targets and identifying performance metrics.
Scientists have made a breakthrough in understanding how the flu virus works by studying its M2 proton channel. Using advanced MRI technology, researchers gained insight into the virus's replication process, which could lead to the development of new prescription drugs.
A study published in JAMA Oncology found that superlatives like 'breakthrough' and 'miracle' were used to describe unapproved cancer drugs, often without clinical data. Targeted therapy was the most commonly referenced class of drugs, with immunologic checkpoint inhibitors also being frequently described as 'game-changers'.
Researchers have discovered a new combination of drugs that may be effective against resistant, BRAF-mutant melanoma by targeting different signaling pathways. The combination showed synergistic effects in tumors resistant to BRAF inhibition.
A study using ^90Y-daclizumab, an anti-CD25 monoclonal antibody, achieved complete and partial responses in 50% of patients with relapsed Hodgkin's lymphoma. The treatment showed minor toxicity, but six patients developed myelodysplastic syndrome, limiting its use as a standalone therapy.
Scientists tracked proteins from the C9orf72 gene, finding a specific protein helps transport essential proteins into motor neuron cells' nuclei. Misplacement of this protein can lead to cell death in diseases like ALS and FTD.
A team of scientists discovered a molecular switch that regulates the body's circadian clock, allowing it to keep time. The 'phosphoswitch' maintains clock speed despite temperature changes and metabolic signals.
A combination of dabrafenib and trametinib significantly prolongs median overall survival time for advanced melanoma patients to over two years, with 51% remaining alive after two years. The treatment also improves disease-free progression and quality of life compared to vemurafenib alone.
Researchers at St. Jude Children's Research Hospital have identified a mechanism underlying the formation of stress granules in cells under stress, which are linked to degenerative diseases such as ALS. The study reveals that mutations in proteins involved in stress granule assembly can lead to toxic fibrils and disease progression.
BACE1 and APH1B-y-secretase control axonal guidance by regulating growth cone collapse and restoration. Inhibition of these proteases may have physiologically opposite effects, suggesting cautious combination therapies to mitigate side effects.
Studies suggest that the insular cortex, damaged during a stroke, can lead to increased likelihood of quitting smoking and reduced withdrawal symptoms. Smokers who experienced strokes in this area were almost twice as likely to quit smoking compared to those with strokes in other parts of the brain.
Acute pancreatitis is a sterile inflammation triggered by false alarm molecules, leading to the formation of harmful DNA-protein structures called NETs. Researchers found that neutrophils can activate these structures, causing pancreas damage.
A University of Iowa study reveals that disabling a critical biological checkpoint, Mitochondrial Pyruvate Carrier (MPC), reduces blood sugar levels in mouse models of type 2 diabetes. The research suggests that inhibiting MPC activity could be a new target for drugs to treat diabetes.
Imetelstat, a novel telomerase targeting drug, demonstrates potential value in treating patients with myelofibrosis by inducing morphologic and molecular remissions in some patients. The study shows selective anti-clonal activity, inhibiting the growth of cancer cells.
Researchers identified a nanobody that stabilizes an enzyme essential for parasite invasion and reproduction. The discovery reveals a previously unappreciated feature of the enzyme's activation, making it a potential target for prevention and treatment of diseases like malaria.
A study analyzing 10 childhood-onset autoimmune diseases found 22 genome-wide signals shared by two or more diseases, suggesting potential new targets for therapy. The research identified genes with biological relevance to specific diseases, offering opportunities for targeted treatment and repurposing existing drugs.
Researchers have identified a novel virulence factor that could be ripe for drug development, targeting the electron transport process in TB. The discovery of MenJ, a gene essential for the organism's proliferation, offers hope for rapid treatment of the disease.
Northeastern University researchers introduce a new metric called 'control energy' to characterize the effort needed to control real-world complex systems. This enables a kind of network cost-benefit analysis, allowing scientists to identify the minimum number of driver nodes required for input signals and optimize network control.
Researchers discovered that blocking a specific ion channel in medulloblastoma can impede tumor cells from proliferating and spreading. The development of targeted treatments could improve outcomes for patients with this disease, which is a common cause of death in children.
A new algorithm developed by InSilico Medicine has the potential to improve the effectiveness of targeted therapy for cancer patients. The algorithm predicts whether a specific drug will work for an individual patient based on activation of intracellular regulatory pathways.
Researchers found that Ebola and Marburg viruses use G protein-coupled receptors to enter cells, which can be blocked by existing drugs targeting these receptors. The study identified 20 GPCR antagonists effective against the two viruses, paving the way for potential therapeutics.
A new tool called the Kinase Addiction Ranker (KAR) improves the ability to match drugs to disease by predicting what genetics are truly driving a patient's cancer. The tool clarifies the best drug or combination of drugs that targets specific genetic abnormalities, leading to more effective treatment options.
Researchers have established the safety and dosing of a new drug for treating blood cancers, targeting dormant cancer stem cells. The drug coaxes these cells to differentiate and exit the bone marrow, where they can be destroyed by chemotherapy agents.
Researchers urge caution when using biodiversity offsets to compensate for development damage, as it can lead to 'stealth' reneging on existing conservation goals. Properly designed offsets can help reconcile development with conservation, but improper use can cause more harm than good.
The CRISPR/Cas system has revolutionized biological research, enabling targeted DNA changes in living cells. This innovation is now being applied to clinical medicine, promising significant advancements in drug therapies, agriculture, and food products.
Researchers developed a wireless device implanted in mice brains that can deliver drugs via remote control. The technology targets specific brain circuits, promising fewer side effects for neurological disorders.
Researchers discovered that combining Roux-en-Y gastric bypass with specific medication can augment weight loss benefits. The study found independent pathways contributing to weight loss, suggesting potential for combination treatment.