Research on the molecular targets that drugs act on. Covers new antibiotic mechanisms, drug discovery competitions, bile-acid pathways, and Alzheimer's targets.
Despite its promise, cancer immunotherapy faces several hurdles, including limited efficacy and variable outcomes. Researchers are exploring new therapeutic targets and combination strategies to overcome resistance and improve patient outcomes.
A team of MIT engineers has developed a drug delivery capsule that can inject large quantities of monoclonal antibodies and other proteins into the stomach lining after being swallowed. The capsule overcomes a major challenge in delivering these drugs orally, which are usually administered via injections.
Scientists at Kyoto University developed a chemical compound that can tag and remove mutant DNA sequences from mitochondria, potentially treating mitochondrial diseases. The approach overcomes existing problems with genetic material injection and antioxidant drugs.
A genetic analysis of humans and rhesus macaques identified the NPSR1 gene as a risk factor for endometriosis. The study found that rare variants in this gene are associated with stage III/IV disease, leading to potential nonhormonal treatment targets for improved therapy.
A study from Linköping University found that the tumour-inhibiting gene TET2 is silenced in most cases of acute lymphoblastic leukemia (ALL) in children. The gene can be reactivated by treatment with an existing drug, 5-azacytidine, suggesting a targeted therapy for ALL in children.
Researchers have identified a new potential treatment for neuroblastoma by targeting the ALT mechanism, which is responsible for chemotherapy resistance. The study found that activating ATM kinase at telomeres promotes chemotherapy resistance in ALT neuroblastoma and suggests a cancer-specific approach to treating this disease.
Researchers reverse lung fibrosis in a mouse model using Bcl-2 inhibitor ABT-199, suggesting a novel therapeutic target to treat idiopathic pulmonary fibrosis. The study finds that monocyte-derived macrophages play a key role in fibrosis progression and that targeting the Cpt1a-Bcl-2 interaction modulates apoptosis resistance.
Researchers at Tel Aviv University successfully printed the first entirely active and viable glioblastoma tumor using a 3D printer. The 3D-bioprinted model includes functional blood vessels that simulate a real tumor, making it a promising tool for predicting treatment efficacy and drug development.
Researchers at Massachusetts General Hospital have identified two separate genetic alterations that enable triple-negative breast cancer cells to develop resistance to a highly effective drug. The findings could help improve therapy and prolong survival for patients with this aggressive form of breast cancer.
Fels and Fox Chase researchers found specific TET2 and DNMT3A mutations in leukemia patients that affect DNA repair pathways. These mutations make leukemia cells sensitive to PARP inhibitors, a type of targeted therapy, while others are resistant. The study aims to develop personalized therapies for patients with these mutations.
Researchers have determined the structure of human leukotriene B4 receptor 1 (hBLT1), a protein involved in inflammation and disease. The analysis reveals how the receptor recognizes its binding partners and interacts with them, opening up avenues for designing better drugs.
Despite guidelines and legal requirements, women remain underrepresented in cardiovascular clinical trials, limiting treatment data on cardiovascular disease. Research shows that women respond differently to drugs than men, highlighting the need for sex-specific data in optimal care.
The article considers the ethical issues surrounding enrolling children with neurodevelopmental conditions, such as autism spectrum disorder and fragile X syndrome, in clinical trials. Parents may face difficult decisions about whether to enroll their children due to concerns about potential loss of positive aspects of their condition.
Researchers have identified a promising therapeutic target, METTL1, to treat aggressive cancers by inhibiting an RNA-modifying protein. The study found that targeting METTL1 effectively destroys cancer cells in laboratory models and mice while leaving healthy cells unharmed.
Researchers at Johns Hopkins Medicine have discovered that mebendazole, an anti-parasitic drug, can prevent and slow the growth of pancreatic cancer in genetically engineered mice. The study suggests that mebendazole may act similarly to collapsing cancer cells' structure and reducing inflammation.
A large-scale genetic study has identified new drug targets to treat type 1 diabetes by examining 61,427 participants and pinpointing 78 regions on the chromosomes that influence risk. The researchers found 36 previously unknown regions and specific gene variations that can be used to identify potential drug targets.
A new technique called HT-MEK enables the simultaneous performance of thousands of enzyme experiments, allowing scientists to deeply probe into enzyme functions and structure. This could reveal clues about how enzymes work together to achieve their remarkable reactivity, enabling researchers to 'do enzymatic tricks' themselves.
Researchers have developed a new type of nanocarrier that combines photothermal, chemo-therapy, and chemodynamic therapy to target and destroy cancer cells. The nanoclusters, which are responsive to near-infrared light, show a significant inhibitory effect on mouse tumor growth in vivo experiments.
Researchers at St. Jude Children's Research Hospital identified a metabolic control pathway that regulates T follicular helper cells, offering targets for drugs to stimulate the adaptive immune response and treat autoimmune diseases such as lupus. The finding holds promise for developing new vaccines and treatments.
A new study reveals 200 previously approved drugs that could be repurposed to treat COVID-19, with 40 already undergoing clinical trials. The research identified key proteins targeted by these drugs and found potential antiviral compounds, including proguanil and sulfasalazine.
Researchers at the University of Cambridge have identified 200 approved drugs that could be effective against COVID-19, including 160 previously unlinked to the infection. The study used computational biology and machine learning to create a comprehensive map of proteins involved in SARS-CoV-2 infection.
A study at ECCMID identifies existing drugs that could be repurposed to treat gonorrhoea by targeting efflux pumps, which remove toxic compounds from bacterial cells. The research found 57 potential drug candidates, including dequalinium and doxorubicin, which may serve as lead compounds for new gonorrhea treatments.
A novel drug candidate, KNT-127, has been developed to target glutamate-induced neurotransmission and reduce anxiety-like behavior in mice. The study proposes a new mechanism of action for treating anxiety disorder, which could lead to the development of evidence-based antipsychotics with a new mechanism.
The national tender system in Norway has significantly reduced the cost of biologic and targeted drugs for rheumatoid arthritis patients. Over a 10-year period, the average annual costs were lowered by approximately 50%, with current users experiencing a 50% reduction and new patients seeing a 75% decrease.
A study found that baseline use of biologic medications like rituximab and JAK inhibitors is associated with worse COVID-19 outcomes in people with rheumatoid arthritis. This association was seen even after adjusting for potential confounding factors.
Researchers found a new treatment approach that targets the body's cellular response to SARS-CoV-2, significantly reducing virus replication. The combination of two drugs reduces virus production in cells by up to 99.5%, making COVID-19 symptoms milder and recovery times faster.
Researchers at Northwestern University have identified a novel target for a drug to treat SARS-CoV-2 and potential future coronaviruses. The new therapy could be taken early in the disease to prevent severe symptoms.
Scientists at Rockefeller University have identified a new class of therapeutics that destroy fibrolamellar tumor cells growing in mice. The team tested over 5,000 compounds to find these effective treatments, which could potentially transform the landscape of precision medicine by tailoring treatment options for individual patients
A new study by Ludwig Institute researchers has developed a method to quickly identify potentially effective drug combinations for personalized cancer therapy. By using dynamic BH3 profiling, the team found specific metabolic dependencies in triple-negative breast cancer cells that could be targeted with existing drugs.
Researchers have identified a potential new treatment for ALS using motor neurons created from ALS patients. The high-throughput platform confirmed two known targets and found an existing class of drugs that could reduce hyperexcitability, a key feature of the disease.
Researchers have developed a new categorization system for vasculitis based on its underlying immunological causes, which may lead to more effective treatments and better patient outcomes. The study identified key differences in disease response to various treatments, paving the way for new clinical trials.
A live-cell screening strategy identified Kv7.2/7.3 as a key target for treating hyperexcitability-induced neurodegeneration in ALS patients. QurAlis' therapeutic candidate QRL-101 (QRA-244) shows promise in decreasing spinal and cortical motor neuron excitability.
The FDA-approved drug sotorasib has been shown to reduce tumor size and improve survival among patients with non-small-cell lung cancer caused by a specific DNA mutation. The study involved 126 patients who showed significant responses to the treatment, including 34% achieving partial response and 3% complete response.
University of Minnesota Medical School researchers found that senescent immune cells drive tissue damage and shorten lifespan. The team identified these cells as the most dangerous type of senescent cell, paving the way for developing senolytic drugs targeting them.
Researchers will use whole genome sequencing to identify genetic variants associated with plaques and tangles in the brain, which begin accumulating years before symptoms appear. The goal is to develop early diagnosis methods and potential treatments for Alzheimer's disease.
Chemists create supramolecular cage using Pd6(TPT)4 cages that can transport and release pharmaceutically active molecules like ibuprofen and progesterone. The system uses ultrasonification to break the bonds, releasing the drugs at the desired location.
A new study published in Blood Advances has identified a gene variant, mesothelin (MSLN), that is abnormally expressed in more than one-third of childhood and young adult AML cases. This discovery could lead to the development of precision medicine treatments for children with AML.
A new compound AAZ-A-154 has been identified with potential to act on beneficial pathways in the brain without hallucinogenic effects. The discovery was made possible by a genetically encoded fluorescent sensor called psychLight that screens for hallucinogenic potential.
Researchers at NUS have identified two new proteins that contribute to EV-A71's ability to invade the central nervous system, making them potential targets for treating severe HFMD cases. This discovery could lead to the development of more effective treatments, particularly for young children affected by this illness.
The company's Abiprot technology identifies antibody binding sites on native-state, disease-relevant proteins at high resolution. The platform has the potential to bring game-changing benefits to large populations of patients suffering from uncurable diseases.
Researchers at Tokyo Medical and Dental University developed an antibody-drug conjugate that selectively targets human monocyte progenitors to combat chronic myelomonocytic leukemia (CMML). This strategy effectively blocks malignant cell proliferation with minimal collateral damage to other cell lineages.
Scientists have discovered how a unique molecular switch in the brain causes feelings of fullness and may help develop improved anti-obesity drugs. The melanocortin receptor 4 is activated by setmelanotide, which binds to its binding pocket and enhances binding with calcium ions.
A new study suggests prioritizing clinical trials of drugs targeting these two proteins to manage COVID-19 in its early stages. Researchers used large-scale human genetic studies to identify existing drugs that can be repurposed for the treatment.
Researchers discovered a biochemical pathway triggered by SARS-CoV-2 in lung cells, which may explain the disease's difficulty to treat. The pairing of antiviral drugs with inhibitors of this process showed promise in inhibiting the immune response.
Researchers identified Mycobacterium tuberculosis' use of rubredoxin B to survive in iron-deficient conditions, helping the bacterium evade the immune system. The study provides new insights into the development of drug resistance and potential targets for therapeutic agents.
A team of University of Alberta researchers has identified a protein that blocks the body's ability to clear low-density lipoprotein (LDL) cholesterol from the blood. This discovery paves the way for developing a new drug to boost existing statin drugs and prevent heart disease.
Researchers at NYU Langone Health identified hundreds of potential new treatment targets for epilepsy by analyzing adult human brain tissue. Altered levels of brain proteins were found predominantly in the hippocampus and frontal cortex, suggesting that these regions may play a role in the development of the disorder.
A breakthrough discovery has pinpointed the mechanism by which normal proteins convert to diseased forms, causing conditions like CJD and Kuru. The research could lead to the development of new drugs to combat these devastating diseases.
Researchers discover histone-lysine N-methyltransferase (NSD3) as a main driver of squamous cell carcinoma lung cancer. Targeting NSD3 could improve treatment and survival rates.
A team at Massachusetts General Hospital developed an artificial intelligence-based method to screen currently available medications as possible treatments for Alzheimer's disease. The analysis yielded a ranked list of candidates, including anti-inflammatory drugs used to treat rheumatoid arthritis and blood cancers.
A new study by Johns Hopkins Bloomberg School of Public Health found that Medicare Part D program lost $1.7 billion due to requests for brand name over generic prescription drugs in 2017. Opting for generic drugs could save hundreds of millions annually.
A team of international researchers has discovered a promising therapeutic drug target, SARM1, which is activated in response to nerve fibre damage. This finding offers hope for developing effective treatments for neurodegenerative disorders such as Parkinson's and Alzheimer's disease.
Researchers at Hong Kong Baptist University develop a peptide-linked drug targeting two viral proteins produced by Epstein-Barr virus, reducing cancer cell damage and increasing drug uptake rates. The novel drug has shown efficacy in animal models and is being further developed for clinical trials.
Researchers are exploring a novel approach to target disease-causing proteins in human cells. This method, utilizing ubiquitin ligases, aims to overcome traditional drug discovery limitations by targeting more disease-causing proteins, offering new therapeutic possibilities for various conditions.
A machine-learning approach has been developed to identify repurposed drugs for COVID-19 treatment in elderly patients. The system accounts for changes in gene expression in lung cells caused by both the disease and aging, pinpointing the protein RIPK1 as a promising target.
The combination of lenvatinib and pembrolizumab significantly improved overall survival, progression-free survival, and response rates compared to sunitinib in untreated patients with metastatic kidney cancer. The study results demonstrate the importance of immune checkpoint inhibitors in first-line treatment.
A new class of antibiotic-drug conjugate (ADC) drugs has been found to significantly increase the survival rate of patients with bladder cancer. The study, led by Queen Mary University of London, showed that the drug reduced the risk of death by 30% compared to chemotherapy, with a median survival time of approximately 13 months.
A recent study found that protected areas reduce the rate of deforestation by 41%, but nearly one-third of these areas are under intense human pressure. The study suggests that just 6.5% of the Earth's woodlands are truly protected, well below the UN's 2020 target of 17%.
Researchers at UT Health San Antonio have identified a human protein called SAMHD1 that can recognize and respond to the HIV virus. This recognition mechanism may lead to a new approach for treating HIV/AIDS by targeting the protein with a specific drug.
A new biologically active porphyrin compound was isolated from Ophiura sarsii, which may be used to make photodynamic therapy more affordable. The compound has been shown to have antitumor effects and could potentially be used to treat triple-negative breast cancer and other cancers.