Research on discovering and developing new drugs. Covers structure-revealing tools, eye-disease mechanisms, protein screening, and once-daily oral medicines.
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.
Early-stage drug development worldwide has nearly doubled over the past decade, with China's share increasing from 8% to 32%, according to Georgetown University researchers. The number of early-stage programs increased by 82.4% from 10,417 in 2015 to 18,999 in 2024.
Researchers have developed a low-cost fake drug detector that uses an infrared sensor to track pill dissolution rates. The device can identify legitimate medications from counterfeit ones with high accuracy, making it a valuable tool in the fight against global health threats.
Researchers at the University of Edinburgh have developed a method to transform post-consumer plastic into L-DOPA, a frontline medication for Parkinson's disease. The process harnesses the power of bacteria to break down PET waste and produce L-DOPA, providing a sustainable solution to the production of pharmaceuticals.
ToxIndex integrates AI agents to access and orchestrate toxicological resources, providing comprehensive risk assessments in hours. The platform addresses a critical need in chemical and drug safety, meeting the vision of the 2007 NRC report for 21st-century safety testing.
A new clinical trial will investigate whether adding the oral medication vorasidenib to standard chemotherapy improves progression-free survival for people with newly-diagnosed, grade 3 IDH-mutant astrocytoma. The study aims to recruit 400 individuals with this type of brain cancer and evaluate the safety and side-effect profile of the...
ToxIndex integrates three tiers of New Approach Methodologies, leveraging AI agents to access and orchestrate toxicological resources, and providing comprehensive, source-traceable risk assessments in a fraction of the time required by traditional methods.
Researchers have identified and resolved molecular bottlenecks to produce doxorubicin, a vital chemotherapy agent, resulting in a 180% increase in production. This breakthrough enables cost-effective manufacturing of essential antibiotics and anti-cancer agents, promising a cleaner and more reliable supply of life-saving medicines.
A preclinical study reveals a new topical cream that activates the skin's immune defenses and suppresses tumor growth in two models of cutaneous squamous cell carcinoma. The cream works by blocking LSD1, an enzyme that suppresses immune-activating pathways in the skin, prompting immune cells to attack tumors.
Researchers discovered yaku'amide B induces CD9 degradation, a cancer stem cell-related protein, in addition to inhibiting ATP synthase, leading to cellular energy depletion and cancer cell suppression. This natural compound has potential as a new therapeutic approach for cancer treatment.
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.
Researchers have developed a bacterial system to create millions of potential drug molecules that can target difficult-to-treat cancers. The approach combines chemical peptide stabilisation with the TBS assay to screen for effective peptides, which can then be tested in more complex tissue models and animal studies.
The European Medicines Agency has granted a positive opinion to Acoziborole Winthrop as a single-dose oral treatment for both early- and advanced-stage gambiense sleeping sickness in adults and adolescents. The medicine, co-developed by DNDi and Sanofi, could provide a significant advance over current therapies.
Researchers at Osaka Metropolitan University found that targeting the glutamine transporter ASCT2 can suppress cell growth and induce apoptosis in synovial sarcoma cells. This study suggests a new approach for treating this aggressive malignant tumor by cutting off its nutrient supply.
Approximately 40 cancer patients will receive LMP744 for five consecutive days, with biological analyses conducted on brain tissues before and after treatment. If results are favorable, treatment will continue for 12 cycles to evaluate parameters such as progression-free survival and overall survival.
The project aims to test whether reducing chronic inflammation triggered by DNA can help older adults stay healthier. The research focuses on retrotransposons, which become increasingly active with age, leading to tissue decline.
A UC Santa Barbara research team has developed a method to efficiently synthesize non-natural amino acids and apply them to peptide construction. This technique provides greater access to amino acids beyond the 22 found in nature, opening up new possibilities for biochemists, medical researchers, and materials scientists.
Chemists at the University of Münster developed a new method to produce high-grade housane molecules, which are small tri- or quadripartite ring molecules crucial for drug development and materials science. The reaction is triggered by photocatalysis, enabling efficient access to valuable products.
Researchers are developing a new compound that can break down MDM2, a cancer-driving protein often found in triple-negative breast cancer, showing promise in shrinking tumors. The goal is to create an entirely new class of therapies for this aggressive and difficult-to-treat form of the disease.
The collaboration aims to integrate Mayo Clinic's clinical insights and genomic data with Merck's AI capabilities to support drug discovery and development. The partnership will focus on high-need therapeutic areas, leveraging multimodal data sets and advanced analytics to advance progress in disease treatment.
Researchers developed a free-to-use software tool, PSBench, to verify the accuracy of artificial intelligence-based protein structure predictions. The database includes 1.4 million annotated protein models, verified by experts, and provides reliable information for building more accurate AI systems.
MIT researchers used a large language model to optimize the genetic sequences of proteins manufactured by yeast, reducing production costs. The new model predicted which codons would work best for manufacturing six different proteins, including human growth hormone and a monoclonal antibody, with successful results.
Kobe University scientists have engineered bacteria to produce a group of compounds with promising pharmacological activities. The breakthrough uses a rational design strategy to create a platform for industrial production of drug candidates.
A recent study reveals that hydration and terminal chemistry of poly(ethylene) glycol (PEG) polymers influence immune recognition, providing insights into why some PEGylated drug coatings fail to evade the immune system. The findings pave the way for novel drug coatings with improved performance.
A new HPV cancer vaccine developed by Northwestern University scientists has shown promising results in a preclinical model. The vaccine's carefully organized structure dramatically enhances the immune system's ability to attack tumors, shrinking them and extending animal survival.
The University of Missouri has launched its first human clinical trial using Eye90 microspheres, a radiopharmaceutical breakthrough device manufactured on campus. The study aims to assess the safety and effectiveness of Eye90 in treating unresectable liver tumors, including hepatocellular carcinoma and metastatic colorectal cancer.
Phyton Biotech has successfully transferred the manufacturing process for Corallopyronin A (CorA), a novel anti-infective agent with potent activity against filarial infections and bacterial infections. The technology transfer marks an important milestone in the Company's specialty GMP fermentation capabilities.
The MIT research team has designed a new type of tissue model that accurately replicates the physiology of the liver, including blood vessels and immune cells. The model was used to study metabolic dysfunction-associated steatotic liver disease (MASLD) and showed promising results in identifying potential treatments.
Researchers have developed stable boron-fluorine compounds that enable the modification of complex molecules without breaking down medicines. These new compounds make it possible to increase the effect or reduce side effects of drugs at a late stage, reducing waste and improving resource efficiency.
The UH College of Pharmacy is expanding its graduate program to offer a Doctor of Philosophy and Master of Science in Population Health & Pharmacoepidemiology, addressing a growing demand for training in evaluating medication safety and effectiveness.
Researchers develop a synthetic compound that acts on all three stages of the disease cycle, eliminating parasites from human blood and liver, and preventing transmission to mosquitoes. The molecule has shown effectiveness against P. vivax and P. falciparum species.
OYE-101, a novel intravenous caffeine formulation, aims to accelerate recovery from general anesthesia and deep sedation in adults. The FDA-cleared treatment also seeks to address opioid-induced respiratory depression and related overdose situations.
Researchers developed a solvent-free method to enhance drug bioavailability by transitioning from solid to gas phase. The sealed heating (SH) method increases drug solubility without using organic solvents.
Marine-derived compounds exhibit potent anti-tumor effects through diverse mechanisms, including apoptosis induction and targeting critical signaling pathways. Challenges remain, but innovative delivery systems and deeper insights into molecular targets hold promise for harnessing the therapeutic potential of these natural compounds.
A Purdue University team led by Kyle Cottrell has discovered a new therapeutic target for triple-negative breast cancer, a deadly form of breast cancer lacking targeted therapies. The researchers identified dsRNA-binding proteins, specifically PACT, which suppress another protein called RNA-activated protein kinase (PKR).
A multidisciplinary team at USC has developed selective compounds that inhibit an enzyme tied to brain inflammation in people at genetic risk for Alzheimer’s. The inhibitors preserve normal brain function and cross the blood-brain barrier, suggesting a promising therapeutic approach for neurodegenerative diseases.
Studies exploring the role of histamine H3R receptor in the brain found that specific amino acid mutations can increase constitutive activity while compromising stability. The research provides a foundation for a better understanding of brain diseases and the design of safer drugs.
Scientists at the University of York discovered a plant gene that produces a powerful alkaloid, securinine, in a unique process driven by bacterial-like genes. This finding allows for the mass production of valuable compounds in labs, reducing reliance on rare plants and harsh chemicals.
Researchers describe chemical journey of YCT-529, a non-hormonal male birth control pill, and its promising molecular science behind it. The compound effectively suppresses sperm counts, showing promise as a durable and reversible contraceptive option.
A Mass General Brigham study identifies new mutations that emerge in tumor cells following treatment, driving resistance in patients with different types of cancer. The researchers found two main categories of mutations: those impairing p53 function and others disrupting drug binding, highlighting a path forward for overcoming resistance.
Researchers developed a new method to measure how effectively antibiotics kill bacteria, crucial for treating complex infections. The 'antimicrobial single-cell testing' method predicts treatment success by observing individual bacteria under different conditions.
Scientists at The Wertheim UF Scripps Institute have designed potential diabetes drugs that reprogram insulin-resistant cells into a healthier state while limiting side effect risks. The compounds target PPAR gamma, a master regulator of fat cell and insulin metabolism.
Researchers at UC Davis have developed a new method to create psychoactive-like molecules that mimic the brain's serotonin receptors. The study found five compounds with efficacy ranging from 61% to 93%, but none induced hallucinogenic behavior, offering a potential new therapeutic scaffold for treating psychiatric disorders.
Southwest Research Institute has upgraded its nuclear magnetic resonance (NMR) laboratory to provide robust chemical analysis of organic compounds used in drug discovery and development. The new facility enables rapid and cost-effective analysis using qNMR, which can be more efficient than HPLC for certain applications.
Researchers at Kanazawa University have developed a new combination therapy that combines CAR-T cell therapy with a SUMOylation inhibitor, which effectively suppresses MYC activity and slows the growth of Burkitt's lymphoma cells. The approach extends survival rates in mouse models by up to 80%.
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.
Tofersen, a new drug approved by the FDA, has been shown to delay disease progression and stabilize symptoms in people with a genetic form of ALS. Long-term use of tofersen slows ALS disease progression and death in about one-quarter of participants, leading to stabilization or improvement in strength and mobility.
Researchers will investigate how HIV and cancer drugs damage brain cells over time, identifying potential early biomarkers of neurotoxicity. They will use human brain organoids grown in the lab to mimic brain physiology.
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.
Acute sepsis alters fluid kinetics and creates a 'third space' resistant to adrenergic modulation, leading to maldistribution of crystalloid fluid. Vasoactive drugs have limited effect on fluid distribution in septic sheep compared to healthy animals.
A blood test may help doctors identify which patients with colon cancer can benefit from anti-inflammatory medication and chemotherapy after surgery. The test measures circulating tumor DNA levels, and high-risk patients who test positive see improved survival rates when taking celecoxib with chemotherapy.
Lab studies found that a combination of THC and CBD kills ovarian cancer cells without harming healthy cells. The compounds may prevent metastasis and have minimal toxic effects, making them a promising alternative to current treatments.
Researchers at the University of Plymouth investigate why drugs used to treat other tumours are ineffective against NF2-related schwannoma and meningioma tumours. They explore repurposing clinically tested cancer drugs to target MDR mechanisms, which may lead to effective therapies for patients with these tumours.
A UBC Okanagan study found that microdosing psychedelics temporarily improves mood, creativity, and wellbeing, but these effects do not persist on non-dosing days. The study, tracking over 1,435 participants from 49 countries, suggests that microdosing may 'reactivate' or build upon prior effects of larger-dose psychedelic experiences.
A SickKids-led study reveals how bile acids can bind to block C. diff's most dangerous toxin, leading to the development of a new compound that neutralizes the toxin directly in the gut. This approach preserves gut health and targets the toxin with precision, offering hope for safer treatments.
A new study identifies a practical and powerful prognostic marker that can help clinicians tailor treatment strategies to improve survival for patients with aggressive T-cell lymphoma. Researchers found that patients who relapse within 12 months of initial therapy have a dramatically declining chance of survival, making targeted therap...
Researchers at Martin Luther University Halle-Wittenberg have developed a promising new substance that inhibits the ability of tuberculosis bacteria to produce energy and causes them to die. The compound, PRP020, targets the pathogen's ATP synthase but attacks a different site than existing drugs like bedaquiline.
The integration of AI predictive tools and novel formulations, such as 3D printing and biodegradable nanocarriers, is transforming the approach to traditional drug development. These cutting-edge technologies are improving drug absorption and bioavailability by predicting and overcoming biopharmaceutic challenges.