Research on discovering and developing new drugs. Covers structure-revealing tools, eye-disease mechanisms, protein screening, and once-daily oral medicines.
A new Center for Protein Design at the University of Copenhagen aims to create artificially designed proteins with tailored properties to tackle diseases, environmental issues, and industrial applications. The centre will drive fundamental research and translate basic findings into concrete solutions.
A phase 3 trial demonstrated the efficacy of selumetinib in reducing tumor size and alleviating pain in adults with neurofibromatosis type 1. The treatment was found to be effective in patients who received it from day one, as well as those who started it later, with significant reductions in tumor size and pain reported.
A study from Waseda University reveals distinct differences between enantiomeric and racemic thalidomide crystals, with asymmetric and uniform thermal responses attributed to dimer symmetry. This research provides insights into chiral compound behavior and supports rational drug design.
Researchers at the University of Pennsylvania School of Engineering and Applied Science have turned a deadly fungus into a potent cancer-fighting compound. The new compound, called asperigimycins, has shown promising results against leukemia cells, rivaling FDA-approved drugs.
Critical Path Institute's Translational Therapeutics Accelerator awards $250,000 to researchers advancing a promising therapeutic pathway for type 1 diabetes. The project aims to address key challenges in T1D treatment, including beta-cell preservation and immune system modulation.
A new study reveals that Rapamycin has a comparable life-extending effect to dietary restriction in eight species of vertebrates. The compound, originally developed as an immunosuppressant, offers similar benefits without the need for caloric restriction.
Researchers developed AI-driven platform IDentif.AI to uncover sex-specific drug combinations for aortic valve stenosis, a serious heart condition. The study found that female-biased combinations were more effective in addressing the disease in female cells, while male-biased combinations were more effective in male cells.
The ISSCR Embryo Models Working Group has released updated recommendations for the oversight and regulation of SCBEMs. The proposed guidelines aim to balance scientific progress with ethical, legal, and social considerations.
Researchers review nanozymes derived from Chinese herbs, including their catalytic properties, biomedical applications, and potential challenges in developing herbzymes for practical use. The review highlights three main types of herbzymes: herb carbon dot enzymes, polyphenol-metal nanozymes, and herb extract nanozymes.
Researchers have identified a critical step in SARS-CoV-2 viral infection: the protective role of ORF3a, which forms dense bodies surrounding the spike protein to prevent it from being cut into smaller components. This finding could lead to new therapeutic targets and vaccine development strategies.
The Phase 1 study of EBC-129 has shown encouraging signs of efficacy in heavily pre-treated pancreatic ductal adenocarcinoma patients, with an overall response rate of 25% and disease control rate of 87.5%. The drug also demonstrated a manageable safety profile, with no major treatment-related adverse events.
A team of researchers used AI and human collaboration to identify six promising drug pairs against breast cancer cells. The combinations, including simvastatin and disulfiram, showed potential for further research in therapeutic repurposing.
The research aims to improve the survivability of wounded soldiers, enhance combat safety, and accelerate postoperative recovery in a multicenter pivotal clinical study. OYE's technology has shown promise in reducing mortality rates, lowering healthcare costs, and improving surgical workflow efficiency.
Scientists have engineered a herpes virus to activate pathways in T cells, enhancing their ability to fight cancer. The approach uses proteins from the herpes virus to recruit enzymes that sustain T cell function in tumors.
The Experimental Drug Development Centre's EBC-129, an antibody-drug conjugate targeting pancreatic cancer, has received FDA fast track designation. The treatment is undergoing Phase 1 clinical trials and aims to provide new treatment options for patients with solid tumours.
Researchers found that an inexpensive HIV drug can improve vision in patients with diabetic macular edema (DME) more effectively and at a lower cost than existing treatments. The drug, lamivudine, is taken orally and may represent a game-changing option for millions of patients worldwide.
Researchers at University of Gothenburg identified a group of nerve cells controlling semaglutide's appetite-suppressing effects without causing nausea. The discovery may lead to improved treatments for obesity and type 2 diabetes.
A new study at the University of Turku identified conditions under which Bexmarilimab activates the immune system against cancer. The drug was shown to trigger B cell-mediated immune responses in healthy tissue adjacent to tumors. This could lead to more accurate patient selection and improved treatment outcomes.
Researchers at UCLA Health identified a candidate small molecule, DDL-357, that increases concentrations of secreted clusterin, reducing toxic protein phospho-tau and improving mitochondrial function. The drug also improved memory in treated mice in maze-based cognitive tests.
The Center for Research Innovation in Biotechnology (CRIB) has developed a comprehensive database of active pharmaceutical ingredients with evidence of clinical testing. The database provides valuable information on drug discovery and development, including pricing, sponsors, and intended clinical applications.
Scientists have developed a sugar-coated nanotherapy that effectively traps misfolded proteins, neutralizing their toxic effects on neurons. The treatment significantly boosts the survival of lab-grown human neurons under stress from disease-causing proteins.
FibroFind's patented Precision Cut Tissue Slice (PCTS) platform preserves the architecture and function of both healthy and diseased human tissue, providing unparalleled translational accuracy. The acquisition is expected to scale FibroFind's reach across therapeutic areas and deepen its impact in pre-clinical drug development.
Researchers have developed a new compound, SHP1705, that selectively attacks glioblastoma stem cells by hijacked circadian clock proteins. The compound was found to be safe and well-tolerated in humans during a phase 1 clinical trial.
A new generative AI model, DiffSMol, has been developed to generate realistic 3D structures of small molecules with promising drug properties. The study achieved a 61.4% success rate in creating novel molecules, outperforming prior research attempts.
University of Oklahoma researchers have developed a method to add a single carbon atom to drug molecules, increasing chemical diversity without compromising sensitive structures. This technique, called skeletal editing, has the potential to revolutionize DNA-encoded library technology and reduce healthcare costs.
Researchers developed a topical antibiotic gel that cured middle ear infections in chinchillas within 24 hours. The gel, containing negatively charged liposomes, was more effective than previous formulations in delivering antibiotics across infected eardrums.
Rasika Dias, a renowned chemist at UTA, has been named a 2025 fellow of the Royal Society of Chemistry for his groundbreaking contributions to chemical sciences. He is the second chemistry faculty member to receive this honor.
Researchers developed promising α-synuclein PET tracers that can visualize protein aggregates in living patients, enabling earlier diagnosis and treatment monitoring. Tracers like [18F]C05-05 and [18F]ACI-12589 showed significant results in distinguishing synucleinopathies from healthy controls.
A clinical trial found that LUT014, a topical BRAF inhibitor, significantly reduced the severity of acne-like skin rashes caused by targeted therapy for colorectal cancer. Patients who received LUT014 had improved quality of life and were able to continue receiving their cancer treatment with reduced side effects.
The treatment demonstrated early signals of efficacy, with 65.7% of patients experiencing lasting stable disease, and was generally well-tolerated, with most adverse events being mild and manageable.
Researchers have developed a highly sensitive method to detect nitazenes – highly potent synthetic opioids – and xylazine, an animal sedative not approved for human use, in Australian wastewater. The method achieved up to 1000-fold enrichment and detected trace levels of these substances, posing significant risks of overdose.
Dr. Richard Pazdur, MD, receives the 2025 AACR Enduring Impact Award for his quarter century of service in cancer science and medicine. He has led an era of progress at the FDA, improving outcomes for patients with cancer through expedited development of novel therapeutics.
Researchers at Memorial Sloan Kettering Cancer Center have made significant breakthroughs in understanding Acute Myeloid Leukemia (AML) and early brain development. They identified a rare quiescent population of stem cells responsible for disease persistence and therapy resistance, as well as shedding light on gene expression patterns ...
University of Oklahoma researchers create bioactive sugars to develop novel antibiotics against multi-drug-resistant infections in cancer patients. By using abundant, inexpensive, iron or metal-free, non-toxic blue light, the team can more easily and rapidly synthesize these important carbohydrates.
Common prescription medications can disrupt sterol biosynthesis, potentially causing developmental disorders. The editorial highlights the need for mandatory sterol biosynthesis screening in clinical practice.
Researchers at MD Anderson Cancer Center have discovered that adding copper-loaded agents to radiotherapy can overcome radioresistance in preclinical models of thoracic cancer. A novel blood-based biomarker, OSMR, has also been identified in patients with acute myeloid leukemia (AML), which shows prognostic potential and can help ident...
Researchers from the International Institute of Molecular and Cell Biology in Warsaw discovered a crucial role of enzyme TENT5A in extending poly(A) tails of therapeutic mRNA molecules, making them more stable and effective. Macrophages play a key role in vaccine effectiveness, capturing and neutralizing 'intruders' after administration.
The University of Missouri is partnering with a consortium to design and license a new research reactor, NextGen MURR, which will produce critical medical isotopes for cancer treatment. The project aims to enhance Missouri's role as a leader in nuclear science and medical research.
A novel cannula delivery system allows repeated, nondisruptive delivery of imaging agents to the mouse brain during long-term multiphoton microscopy. This innovation enhances longitudinal studies on brain function, disease progression, and potential treatments.
A team of experts at Cincinnati Children's reports achieving a milestone in growing human liver organoid tissue that faithfully reproduces key zones, nearly doubling rodent survival rate. This breakthrough paves the way for studying human liver biology and disease, accelerating drug development and potentially leading to repair tissues.
A new, easier-to-administer sustained-release formulation of flucytosine has entered Phase II clinical trials in Malawi and Tanzania. The trial aims to address the growing concern of cryptococcal meningitis, a major health threat to people with weakened immune systems, particularly those with advanced HIV.
A landmark study found that over 3 million children worldwide lost their lives in 2022 due to antimicrobial resistance (AMR)-related infections. The study data also revealed a sharp rise in the use of Watch and Reserve antibiotics, posing serious long-term risks.
Researchers used CRISPR interference to examine every gene in the human genome and discovered a new set of genes contributing to Parkinson's disease risk. The study identified the Commander complex, which regulates lysosomal function and is implicated in PD risk, offering opportunities for new treatments.
The journal is inviting submissions on AI for engineering, focusing on novel methodologies and applications to solve real-world problems. Submissions can cover various areas such as intelligent manufacturing, energy, transportation, and medicine.
Researchers have identified a potential precision medicine approach to prevent chronic kidney disease progression by targeting type 5 collagen, a minor component of scar tissue. A repurposed anti-cancer drug, Cilengitide, has been shown to slow kidney scarring and disease progression in high-risk individuals.
Scientists at City of Hope have identified a new molecular target for treating pancreatic cancer, using an experimental drug to slow tumor growth and damage tumor cells. The approach showed promise in clinical trials with two patients, resulting in up to a 49% shrinkage in liver metastases.
A new artificial intelligence-based method detects genetic markers of antibiotic resistance in bacteria, potentially leading to faster and more effective treatments. The method, called Group Association Model, uses machine learning to identify key mutations linked to drug resistance, reducing false positives and misdiagnoses.
Researchers at Mayo Clinic developed a personalized deep brain stimulation platform to treat drug-resistant epilepsy. The study found that this approach reduced seizures while also improving memory and sleep in patients with temporal lobe epilepsy.
Neuro-immunologist Stephen Hauser has won the 2025 Breakthrough Prize in Life Sciences for his role in identifying the immune system's primary driver of damage to nerve cells in multiple sclerosis. His B-cell theory has led to the development of therapies that have transformed treatment, reducing relapses and improving prognosis.
Researchers at MIT have developed a method to mass manufacture nanoparticles that target cancer cells, eliminating the need for manual polymer mixing and streamlining production. This approach integrates good manufacturing practice (GMP)-compliant processes, making it suitable for large-scale production of cancer treatments.
A clinical study confirms that early treatment with fosdenopterin/rcPMP significantly reduces the risk of early death and promotes healthy brain development in infants with MoCD type A. The therapy restores the missing molybdenum cofactor, leading to improved developmental milestones.
Scientists at ISTA create a new brain organoid model that incorporates microglia to study viral infections, such as Rubella, and test the effectiveness of ibuprofen. The results show that microglia play a crucial role in inflammatory reactions and that ibuprofen exerts its protective effects by inhibiting two inflammatory enzymes.
A new paper highlights emerging alternatives to traditional diabetes medicines, such as continuous glucose monitors and lifestyle interventions like GEM, which can improve patient adherence and long-term health outcomes.
Researchers identified a new way to fight infections like Lyme and syphilis by disrupting the bacteria's 'motor', preventing it from spreading through the body. The findings could have wide-ranging impacts on the treatment of infections in the future as concern about antibiotic-resistant strains grows.
A novel AI-based method called scNET combines gene expression data with networks of possible gene interactions to identify biological patterns in response to drug treatments. The system reveals complex mechanisms underlying cellular behavior, providing insights for new therapeutic approaches.
A study reveals that most Acinetobacter baumannii infections in a Malaysian hospital belonged to the Global Clone 2 lineage, which is also dominant worldwide. The majority of bacterial samples were resistant to multiple antibiotics, including carbapenems.
Researchers developed new AI models, InstaNovo and InstaNovo+, to vastly improve accuracy and discovery in protein science. These models excel in tasks such as de novo peptide sequencing, identifying microorganisms, and discovering novel peptides, with implications for personalized medicine, cancer immunology, and beyond.
Researchers propose GLDH as a more liver-specific biomarker for detecting liver injury, offering greater specificity than current ALT and AST tests. The test performed similarly to ALT for detecting liver injury but was not impacted by associated muscle injury.
Researchers have identified six promising biomarkers that can detect drug-induced kidney injury earlier than traditional markers, enabling more timely monitoring during drug development. These biomarkers could lead to the development of safer medications and improve patient outcomes.
A new medical database automatically compiles patient records of obese individuals, offering insights for health promotion and drug development. The J-ORBIT database reveals strong links between obesity and various diseases, including diabetes, hypertension, and coronary heart disease.