Research on discovering and developing new drugs. Covers structure-revealing tools, eye-disease mechanisms, protein screening, and once-daily oral medicines.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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 company has secured $5 million in funding to support Phase 1 and pivotal trials for its therapies aimed at addressing opioid-induced respiratory depression and improving anesthesia recovery. This financing marks a transformative moment for OYE Therapeutics, positioning the company to enter clinical development with confidence.
MIT chemists successfully synthesized verticillin A, a fungal compound that has shown potential as an anticancer agent. The researchers then generated derivatives of the compound and tested them against pediatric brain tumors, finding that some were effective against cancer cells with high levels of EZHIP protein.
Biomedical researchers recommend diversifying funding sources, pursuing earlier licensing and commercialization, and fostering international collaborations. The US drug discovery landscape is at risk due to federal funding cuts, and alternative approaches are needed to ensure continued progress.
A five-year NIH-funded study will explore the role of glucagon hormone in human metabolism, focusing on its impact on insulin secretion, glucose regulation, and energy balance. The research aims to clarify glucagon's function and inform more effective drug development for obesity and Type 2 diabetes treatment.
Researchers at the University of Virginia Health System have developed a new treatment for acute myeloid leukemia, a deadly form of blood cancer. The FDA-approved medication works by disrupting cellular protein interactions that drive leukemia cell growth and survival, offering patients a potential cure.
The article highlights the mismatch between psychedelics and economic drug development principles. Pharmaceutical companies are developing short-acting compounds and neuroplastogens to engineer trips out of the experience altogether. Dr. Sandy Hager's research suggests investors should remain cautious due to weak intellectual property ...
The Virtual Cell Pharmacology Initiative (VCPI) aims to build the first standardized framework for virtual cell modeling in drug discovery. Ginkgo Datapoints is offering free high-throughput RNA profiling via its platform, generating over 12 billion data points and aiming to test at least 100,000 compounds.
A team at the University of Pennsylvania has solved the mechanism of action of hydralazine, revealing its potential to halt the growth of brain cancer cells. By blocking an oxygen-sensing enzyme, hydralazine can reduce intracellular calcium levels, causing blood vessels to relax and tumor cells to enter a dormant state.
Researchers found that CRISPR-Cas9 gene editing persists longer and produces more predictable results in non-dividing neurons. They also discovered new DNA repair genes that can be used to control gene editing outcomes, which could lead to safer and more effective therapies for genetic diseases.
Clinical trial participants are calling for more inclusive and patient-centered cancer research, highlighting the need for transparent designs and improved communication. The authors propose various improvements to trial design and delivery, including expanding eligibility criteria and involving patient advocates in trial design.
A new computational tool called DeepTarget predicts direct and indirect targets of cancer drugs, revealing that small molecules can have different targets and effects depending on the disease and cell type. The study demonstrates the tool's superior performance in real-world scenarios, highlighting its potential to accelerate drug deve...
Researchers at the University of Kansas have discovered a human gene, PARP14, that plays a role in regulating interferon and boosting the immune response. The protein's antiviral activity against multiple viruses suggests it could guide the development of new therapies for viral infections.
A new compound MCH11, a monoacylglycerol lipase inhibitor, shows promising effects in reducing alcohol consumption and motivation to drink in mice. The molecule exhibits anxiolytic and antidepressant properties with sex-dependent efficacy, correcting genetic alterations associated with alcohol use disorder.
Stuart S. Martin, PhD, has been appointed Chair of the Department of Pharmacology & Physiology at UMSOM, bringing over 20 years of success in advancing basic science research efforts and developing new drug therapies. His groundbreaking discovery of microtentacles on cancer cells promises to lead to new life-extending therapies.
Scientists at Wake Forest University School of Medicine have discovered a new way to kill cancer cells by blocking their ability to clean up harmful waste. By inhibiting peroxiredoxin-3, researchers found that toxic levels of hydrogen peroxide overwhelm cancer cells, destroying them.
Advanced molecular dynamics simulations model complex RNA structures with high accuracy, enabling potential applications in RNA-based therapies and drug design. The study successfully simulated the folding of diverse RNA stem loops, revealing a distinct folding pathway for challenging motifs.
A new small molecule drug, RAGE406R, has been developed to disrupt a key cellular pathway responsible for chronic inflammation and associated complications in patients with diabetes. The breakthrough could offer a new therapeutic option for stopping the harmful effects of both type 1 and type 2 diabetes at the source.
Researchers found that AI models predict protein structures despite modifications in amino acid sequences or ligands, indicating a lack of understanding of physical chemistry. The models only recognize patterns they've seen before and struggle with unknown proteins.
AI accelerates discovery and validation of novel therapeutic targets by integrating multi-omics data and advanced computational models, reducing the traditional decade-long research and development cycle to two to three years. AI also enhances drug efficacy, predicts resistance mechanisms and improves clinical translation.
Researchers developed a machine learning-based workflow, SPaDe-CSP, to predict crystal structures of organic molecules. The workflow narrows the search space by predicting probable space groups and crystal densities before computationally intensive relaxation steps.
A new broad-spectrum antivenom developed by DTU researchers covers 17 African snake species and provides better protection against tissue damage, with a lower risk of immune reactions. The antivenom has shown impressive results in laboratory studies and could revolutionize the treatment of venomous snakebites in Africa.
Researchers from The University of Osaka and The University of Tokyo have developed a novel technology that visualizes specific molecules inside living cells using light. The new photo-responsive alkyne tag enables precise visualization without disrupting molecular dynamics.
The FDA has approved elinzanetant for treating hot flashes and night sweats in postmenopausal women. The drug significantly reduces the frequency and severity of symptoms while improving sleep quality and quality of life.
A novel therapeutic candidate has been developed to treat severe cutaneous adverse reactions such as Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN). The compound suppresses disease-specific keratinocyte death, demonstrating its efficacy in preclinical studies.
Researchers at Sanford Burnham Prebys discovered a new mechanism to confer signaling bias in predictable ways, permitting rational design of new drugs. This breakthrough could lead to better therapies for addiction and psychiatric disorders by targeting the neurotensin receptor 1 (NTSR1) with biased modulators.
Researchers at Gladstone Institutes discover a gene called HMGN1 that disrupts DNA packaging and regulation, leading to heart malformations in people with Down syndrome. Removing the extra copy of HMGN1 from mice with Down syndrome prevents heart defects, paving the way for potential treatments.
Researchers have discovered an enzyme called PapB that can create tight rings in therapeutic peptides, enabling the creation of stronger, longer-lasting versions of GLP-1 medications. This enzymatic method offers a simpler alternative to traditional chemical methods and could improve the stability and effectiveness of these drugs.