Drug Development

Preclinical study reveals new cream may prevent or slow growth of some common skin cancers

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.

Deep-sea natural compound targets cancer cells through a dual mechanism

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.

AI tool streamlines drug synthesis

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.

Alliance for Clinical Trials in Oncology highlights new and open colorectal cancer trials in March

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.

Contraception without hormones: Goethe University researches alternatives to “the pill”

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.

Chemically ‘stapled’ peptides used to target difficult-to-treat cancers

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.

Acoziborole Winthrop, developed by DNDi and Sanofi, receives European Medicines Agency positive opinion as three-tablet, single-dose treatment for most common form of sleeping sickness

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.

Starving cancer: Nutrient deprivation effects on synovial sarcoma

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.

Gibson Oncology, NIH to begin Phase 2 trials of LMP744 for treatment of first-time recurrent glioblastoma

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.

URochester researchers awarded up to $22M to study a hidden driver of aging

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.

Scientists unlock a massive new ‘color palette’ for biomedical research by synthesizing non-natural amino acids

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.

New method for housane synthesis

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.

Merck and Mayo Clinic announce new research and development collaboration to support AI-enabled drug discovery and precision medicine

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.

Making AI-based scientific predictions more trustworthy

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.

UH researcher joins $3.2 million effort to develop promising new breast cancer treatment

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.

Rhododendron-derived drugs now made by bacteria

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.

New AI model could cut the costs of developing protein drugs

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.

Exploring how the immune system detects drugs coated with ‘stealth’ polymers

HPV cancer vaccine slows tumor growth, extends survival in preclinical model

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.

University of Missouri conducts first in-person clinical trial for nuclear medicine breakthrough device

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.

Technology transfer for Corallopyronin A successfully completed with Phyton Biotech

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.

New boron compounds pave the way for easier drug development

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.

MIT research shows new tissue models could help researchers develop drugs for liver disease

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.

Synthetic compound has the potential to treat malaria and prevent its transmission

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 Therapeutics will initiate clinical development of its caffeine injection

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.

UH College of Pharmacy expands degree plans to offer doctorate and master of science in population health & pharmacoepidemiology

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.

Exploring marine-derived compounds as potential anti-cancer agents: mechanisms and therapeutic implications

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.

Overcoming the solubility crisis: a solvent-free method to enhance drug bioavailability

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.

Purdue team announces new therapeutic target for breast cancer

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).

USC scientists identify promising new target for Alzheimer’s-linked brain inflammation

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.

Exploring mutations that spontaneously switch on a key brain cell receptor

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.

Plant discovery could lead to new ways of producing medicines

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.

New publication details journey of the molecule behind male birth control pill

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.

Researchers uncover how tumors become resistant to promising p53-targeted therapy

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.

New test shows which antibiotics actually work

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.

HiPerGator supercomputer accelerates diabetes drug discovery advances

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.

Creating hallucination-free, psychedelic-like molecules by shining light on life’s basic building blocks

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.

SwRI upgrades nuclear magnetic resonance laboratory for pharmaceutical R&D

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.

Promising innovative combination therapy for Burkitt's lymphoma

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%.

Scientists boost cell "powerhouses" to burn more calories

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.

New ALS drug stabilizes decline with a trend toward improved strength, mobility for some

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.

UMBC-led team to study how HIV and cancer drugs harm the brain

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.

NU-9 halts Alzheimer’s disease in animal model before symptoms begin

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.

Vasoactive drugs and the distribution of crystalloid fluid during acute sepsis in sheep

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.

Blood test may help identify which colon cancer patients benefit from NSAIDs

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.

Cannabis derivatives could provide new ovarian cancer treatments

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.

Study explores why some drug therapies are ineffective for brain tumor patients

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.

UBCO study finds microdosing can temporarily improve mood, creativity

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.

SickKids-led study reveals promising gut-targeted therapy for C. difficile infections

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.

Prognostic tool could help clinicians identify high-risk cancer patients

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...

Tuberculosis: Scientists develop novel drug candidate for combating resistant pathogens

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.

Artificial Intelligence-driven Strategies and Modern Innovations to Surpass Biopharmaceutic Limitations in Traditional Drug Development

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.

OYE Therapeutics closes $5M convertible note round, advancing toward clinical development

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 synthesize a fungal compound that holds promise for treating brain cancer

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.

Strategies to keep drug discovery research alive in the US despite funding cuts from biomedical researchers

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.

UT Health San Antonio leads $4 million study on glucagon hormone’s role in diabetes, obesity

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.

Much-needed new drug approved for deadliest blood cancer

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.

Ginkgo Datapoints launches the Virtual Cell Pharmacology Initiative to build a community-driven data standard for AI drug discovery

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.

Behind the 2025 “shroom boom” hides a bad trip

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 ...

Solving a 70-year-old medical mystery surrounding a vasodialator may yield a novel way to treat brain cancer

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.