. Scientific Frontline: Pharmaceutical
Showing posts with label Pharmaceutical. Show all posts
Showing posts with label Pharmaceutical. Show all posts

Wednesday, January 28, 2026

New cancer-killing material developed by Oregon State University nanomedicine researchers

Graphic depicting how new CDT nanoagent works.
Illustration Credit: Parinaz Ghanbari.

Scientific Frontline: "At a Glance" Summary

  • Main Discovery: A novel iron-based metal-organic framework (MOF) nanoagent has been developed to trigger dual chemical reactions within cancer cells, generating oxidative stress via hydroxyl radicals and singlet oxygen to eradicate malignant cells while sparing healthy tissue.
  • Methodology: The researchers designed a chemodynamic therapy (CDT) agent that leverages the acidic and high-hydrogen peroxide microenvironment of tumors to catalyze the simultaneous production of hydroxyl radicals and singlet oxygen.
  • Key Data: In preclinical studies involving mice with human breast cancer, systemic administration of the nanoagent resulted in complete tumor eradication and long-term prevention of recurrence with no observed systemic toxicity or adverse effects on healthy cells.
  • Significance: This advancement overcomes limitations of existing CDT agents that typically generate only one type of reactive oxygen species or lack sufficient catalytic activity, offering a more potent and durable therapeutic benefit for cancer treatment.
  • Future Application: The team plans to evaluate the therapeutic efficacy of this nanoagent in various other cancer types, including aggressive pancreatic cancer, to establish its broad applicability prior to human clinical trials.
  • Branch of Science: Nanomedicine, Oncology, and Pharmaceutical Sciences

Monday, January 26, 2026

Purdue team announces new therapeutic target for breast cancer

Graduate student Addison Young (left) and Kyle Cottrell, assistant professor, both in Purdue’s department of biochemistry. Young and Cottrell have reported discovering a new therapeutic target for triple-negative breast cancer in the journal RNA.
Photo Credit: Courtesy of Purdue University

Scientific Frontline: "At a Glance" Summary

  • Main Discovery: A specific double-stranded RNA (dsRNA)-binding protein called PACT has been identified as a novel therapeutic target for triple-negative breast cancer (TNBC), a deadly form of the disease that currently lacks targeted therapies.
  • Methodology: Researchers utilized the gene-editing tool CRISPR-Cas9 to deplete PACT in various cell lines, allowing them to observe which cellular pathways became activated and to confirm PACT's role as a suppressor of the RNA-activated protein kinase (PKR).
  • Key Data: The study established that PACT functions as a dimer—requiring the fusion of two monomers to operate—and that TNBC cells are particularly sensitive to its depletion, which triggers a "viral mimicry" state that can lead to cancer cell death.
  • Significance: This research resolves a scientific controversy by confirming PACT acts as a suppressor rather than an activator of PKR; blocking PACT allows PKR to sense dsRNA and initiate stress responses that kill cancer cells, offering a strategy to treat TNBC without broad chemotherapy.
  • Future Application: Scientists aim to develop molecules that specifically inhibit PACT dimerization, creating precise drugs for TNBC and potentially other cancer types that depend on this protein for survival.
  • Branch of Science: Biochemistry and Oncology.
  • Additional Detail: Unlike many therapeutic targets which are enzymes, PACT is a structural protein; therefore, treatment strategies must focus on physically preventing the binding of its two monomers rather than blocking enzymatic activity.

Optimized Solvent Design Improves Lymphatic Drug Delivery to Metastatic Lymph Nodes

Overview of Lymphatic Drug Delivery Systems (LDDS) and the Optimal Ranges of Solvent Osmolarity and Viscosity Depending on Therapeutic Strategies.
Illustration Credit: ©Taiki Shimano et al.

Scientific Frontline: "At a Glance" Summary

  • Main Discovery: The optimization of solvent osmolarity and viscosity in Lymphatic Drug Delivery Systems (LDDS) significantly regulates drug pharmacokinetics and perinodal dynamics to improve treatment of metastatic lymph nodes.
  • Methodology: Researchers injected therapeutic formulations directly into the sentinel lymph nodes of MXH10/Mo/lpr mice—a model featuring human-sized nodes—to monitor real-time changes in lymphatic and vascular flow based on varied solvent properties.
  • Key Data: Increased solvent osmolarity was observed to promote blood inflow and expand lymphatic sinuses (drug pathways), while solvent viscosity acted as the dominant factor determining the duration of drug retention and the extent of delivery.
  • Significance: The study provides critical guidelines for "tailor-made solvent design," directly validating the protocols for ongoing Phase I clinical trials at Iwate Medical University and Tohoku University Hospital.
  • Future Application: Development of next-generation cancer therapies where drug solvent properties are customized to specific clinical goals, such as maximizing retention time or enhancing downstream distribution.
  • Branch of Science: Biomedical Engineering, Oncology, and Pharmacology.
  • Additional Detail: This research represents the first comprehensive demonstration of how fundamental physicochemical properties of solvents independently influence drug behavior during intranodal administration.

Wednesday, January 21, 2026

Lithium study yields insights in the fight against HIV

Ana Luiza Abdalla and Andrew Mouland in front of a flow cytometer at the Lady Davis Institute for Medical Research. The instrument was used to collect key data for the study
Photo Credit: Lucca Jones

Scientific Frontline: "At a Glance" Summary

  • Main Discovery: Lithium treatment effectively prevents the reactivation of HIV in latent infected cells, keeping the virus dormant through a biological mechanism previously unidentified in this context.
  • Methodology: Researchers utilized a novel fluorescence-based assay to distinguish between dormant and active virus in lab-grown human cells, testing lithium's efficacy while simultaneously disrupting the autophagy pathway to isolate the mechanism of action.
  • Key Data: Experiments demonstrated that lithium's ability to suppress viral reactivation persisted even when the cell's autophagy (recycling) system was disabled, directly contradicting the prevailing hypothesis that autophagy was required for this effect.
  • Significance: This finding supports the feasibility of a "functional cure"—strategies that keep the virus permanently dormant rather than eradicating it—and identifies a new biological target for maintaining HIV latency.
  • Future Application: Development of new pharmaceutical agents that mimic lithium's viral suppression properties without causing the psychoactive side effects or toxicity associated with the drug's current clinical use.
  • Branch of Science: Virology and Pharmacology
  • Additional Detail: While lithium is an inexpensive and readily available drug, the authors explicitly warn against its current use by HIV patients due to significant side effects and the lack of human clinical trials for this specific indication.

Study Sheds Light on the Function of a Key Antibiotic-Producing Enzyme

Researchers have successfully replaced a section of the antibiotic-synthesizing enzyme PikAIII-M5, advancing our understanding of its structure and function and moving us closer to the creation of synthetic antibiotics.
Illustration Credit: ©Tohoku University

Scientific Frontline: "At a Glance" Summary

  • Main Discovery: Researchers successfully engineered a chimeric version of the enzyme PikAIII-M5, a key component in pikromycin biosynthesis, by swapping its beta-ketoreductase domain to control the stereochemistry of macrolide chains.
  • Methodology: The team utilized a synthetic substrate evaluation system to physically replace the beta-ketoreductase domain within the PikAIII-M5 enzyme with an alternative domain, subsequently analyzing how these structural modifications altered the enzyme's biochemical output.
  • Key Data: The study validated that the beta-ketoreductase domain acts as an interchangeable module; its successful replacement demonstrated that specific domain swapping can predictably dictate the structural composition of the resulting macrolactone ring.
  • Significance: This research establishes a verified "design guideline" for combinatorial biosynthesis, enabling more accurate predictions of chemical structures from genomic data and facilitating the engineering of complex, non-natural drug molecules.
  • Future Application: The findings will be applied to create novel macrolide antibiotics with structures not found in nature, directly addressing the global crisis of antibiotic resistance and the shrinking pipeline of effective antimicrobial drugs.
  • Branch of Science: Synthetic Biology, Biochemistry, and Pharmaceutical Sciences.
  • Additional Detail: The researchers describe the strategic engineering process as analogous to "swapping interchangeable parts in a machine," emphasizing the high potential for modular manipulation in antibiotic development.

Friday, January 16, 2026

Purdue mRNA therapy delivery system proves to be shelf-stable, storable

The Proceedings of the National Academy of Sciences has published research about a Purdue University virus-mimicking platform technology that targets bladder cancer cells with mRNA therapies. The LENN platform scientists include, from left, Christina Ferreira, Saloni Darji, Bennett Elzey, Joydeep Rakshit, Feng Qu and David Thompson.
Photo Credit: Purdue University /Ali Harmeson

Scientific Frontline: "At a Glance" Summary

  • Main Discovery: The LENN (Layer-by-layer Elastin-like Polypeptide Nucleic Acid Nanoparticle) platform successfully delivers mRNA therapies to bladder cancer cells while retaining full biological activity after being freeze-dried into a shelf-stable powder.
  • Methodology: Researchers engineered a virus-mimicking dual-layer nanoparticle to condense and protect nucleic acids, then subjected the formulation to lyophilization (freeze-drying) and storage at -20°C to validate its stability and rehydration properties.
  • Key Data: The lyophilized samples maintained complete structural integrity and functionality after three days of storage, successfully targeting upregulated receptors on tumor cells without triggering an immune response.
  • Significance: This technology overcomes the severe cold-chain limitations of current lipid nanoparticle systems—which often require storage below -45°C—by providing a biomanufacturable, storable powder form that facilitates easier global distribution.
  • Future Application: The team is upscaling the system for preclinical evaluation and initiating efficacy and safety studies in mouse models of bladder cancer.
  • Branch of Science: Nanomedicine, Pharmaceutical Chemistry, and Oncology.
  • Additional Detail: Multiple reaction monitoring (MRM) profiling confirmed that the system utilizes natural entry pathways and avoids immune detection, potentially solving the "redosing" clearance issues associated with traditional viral vectors.

Thursday, January 1, 2026

What Is: Psychedelic Renaissance

The current "Psychedelic Renaissance" is not a new discovery but a recovery of lost knowledge.
Image Credit: Scientific Frontline

The Fourth Wave of Psychiatry

The field of psychiatry is currently undergoing its most significant paradigm shift since the introduction of the first psychopharmaceuticals in the mid-20th century. For decades, the standard of care for mental health disorders has been dominated by the monoamine hypothesis—the idea that regulating neurotransmitters like serotonin, dopamine, and norepinephrine through daily maintenance medication can rectify chemical imbalances. However, a growing body of evidence, accumulated largely over the last two decades and culminating in the pivotal events of 2024 and 2025, suggests that this model is incomplete. We are witnessing the rise of a "fourth wave" of psychiatry, characterized by the use of psychedelics: compounds that do not merely suppress symptoms but appear to catalyze profound, rapid, and durable healing through mechanisms of neuroplasticity and network reorganization.

This report serves as an exhaustive analysis of the current state of psychedelic medicine as of late 2025. It moves beyond the simplistic "shroom boom" narratives to dissect the complex neurobiology, the rigorous clinical trials, and the volatile regulatory landscape that defines this sector. The subject matter encompasses "classic" psychedelics like psilocybin and lysergic acid diethylamide (LSD), which primarily target the serotonin 2A receptor, as well as "atypical" psychedelics or entactogens like 3,4-methylenedioxymethamphetamine (MDMA).

Monday, December 29, 2025

Researchers find breast cancer drug boosts leukemia treatment

Jeffrey Tyner, Ph.D., and Melissa Stewart, Ph.D., led a team at OHSU that discovered a new drug combination that may help people with acute myeloid leukemia.
Photo Credit: OHSU/Christine Torres Hicks

A research team at Oregon Health & Science University has discovered a promising new drug combination that may help people with acute myeloid leukemia overcome resistance to one of the most common frontline therapies.

In a study published in Cell Reports Medicine, researchers analyzed more than 300 acute myeloid leukemia, or AML, patient samples and found that pairing venetoclax, a standard AML drug, with palbociclib, a cell-cycle inhibitor currently approved for breast cancer, produced significantly stronger and more durable anti-leukemia activity than venetoclax alone. The findings were confirmed in human tissue samples as well as in mouse models carrying human leukemia cells.

“Of the 25 drug combinations tested, venetoclax plus palbociclib was the most effective. That really motivated us to dig deeper into why it works so well, and why it appears to overcome resistance seen with current therapy,” said Melissa Stewart, Ph.D., research assistant professor in the OHSU School of Medicine and Knight Cancer Institute and lead author of the study.

More than 20,000 Americans are diagnosed with AML each year, making it one of the most common types of leukemia — and one of the most aggressive.

Saturday, December 27, 2025

Pharmaceutical: In-Depth Description

Image Credit: Scientific Frontline / AI generated

Pharmaceutical science is the multidisciplinary field concerned with the discovery, development, manufacturing, and regulation of medications. It acts as the critical bridge between the chemical and biological sciences, focusing on the complex process of turning a chemical entity or biologic agent into a safe and effective therapeutic product. Its primary goals are to understand how drugs interact with biological systems, to design optimal delivery mechanisms for these drugs, and to ensure their safety and efficacy for the prevention and treatment of human and animal diseases.

Tuesday, December 16, 2025

A platform to test new cancer treatments

Differentiated hepatic cells growing in a flask re-gain the appearance of cells present in liver.
Image Credit: © FAMOL, UNIGE

Overcoming acquired treatment resistance is one of the major challenges in the fight against cancer. While combination therapies hold promise, their toxicity to healthy tissue remains a major hurdle. To anticipate these risks, researchers at the University of Geneva (UNIGE) have developed in vitro models of the kidneys, liver, and heart – three organs particularly sensitive to such therapies. This fast, animal-free approach paves the way for safer evaluation of new treatments. The findings are published in Biomedicine & Pharmacotherapy

Recent advances in immunotherapy, targeted therapies, and gene therapies have significantly improved survival rates for patients with cancer. However, over time, many tumors develop resistance to these treatments, undermining their effectiveness. This phenomenon, known as ‘acquired resistance’, has become one of the major challenges in oncology. 

Monday, December 15, 2025

Reproduced human neural circuits show the crucial role of the thalamus in shaping the cortical circuit

Assembloid [3D fluorescent staining] Axons in the thalamus (pink) extended toward the cortex, while those in the cortex (green) extended toward the thalamus at 14 days post-fusion.
Image Credit: Fumitaka Osakada

A Japanese research team has successfully reproduced the human neural circuit in vitro using multi-region miniature organs known as assembloids, which are derived from induced pluripotent stem (iPS) cells. With this circuit, the team demonstrated that the thalamus plays a crucial role in shaping cell type-specific neural circuits in the human cerebral cortex.

These findings were published in the journal Proceedings of the National Academy of Sciences of the United States of America.

Our brain’s cerebral cortex contains various types of neurons, and effective communication among these neurons and other brain regions is crucial for activating functions like perception and cognition.

Patients with neurodevelopmental disorders, such as autism spectrum disorder (ASD), exhibit disruptions in the structure and function of neural circuits in the cerebral cortex. Therefore, understanding the principles of these circuits is essential to uncovering the causes of these disorders and developing new medications.

Thursday, November 27, 2025

Possible therapeutic approach to treat diabetic nerve damage discovered

Longitudinal sections of two injured nerves with regenerating nerve fibers. Both specimens are from diabetic animals; in the lower image, the animal was treated with a peptide. Regeneration can be seen in the green-stained nerve fibers.
Image Credit: Dietmar Fischer / University of Cologne

Researchers have decoded the signaling pathway that inhibits nerve regeneration in diabetes and have developed a therapeutic peptide that could transform the treatment—and possibly even the prevention—of diabetic nerve damage. 

Nerve damage is one of the most common and burdensome complications of diabetes. Millions of patients worldwide suffer from pain, numbness, and restricted movement, largely because damaged nerve fibers do not regenerate sufficiently. The reasons for this are unclear. A research team led by Professor Dr Dietmar Fischer, Professor of Pharmacology at the University of Cologne’s Faculty of Medicine, and Director of the Center for Pharmacology at University Hospital Cologne, has now identified a central mechanism that explains limited regeneration in diabetes. Building on this, the researchers have developed a promising therapeutic approach that can be used to increase regeneration. Their findings were published in the ‘Science Translational Medicine’ journal under the title ‘Failure of nerve regeneration in mouse models of diabetes is caused by p35-mediated CDK5 hyperactivity’.

Wednesday, November 5, 2025

“Rotten egg” gas could be the answer to treating nail infections, say scientists

Nearly half of people aged over 70 suffer from nail infections, which are notoriously difficult to treat.
Photo Credit: Wang Yanwei

Hydrogen sulphide, the volcanic gas that smells of rotten eggs, could be used in a new treatment for tricky nail infections that acts faster but with fewer side effects, according to scientists at the University of Bath and King’s College London (KCL).

Nail infections are mostly caused by fungi and occasionally by bacteria. They are very common, affecting between 4-10% of the global population, rising to nearly half those aged 70 or over.

These infections can lead to complications, particularly in vulnerable groups such as diabetics and the elderly, but are notoriously difficult to treat.

Current treatments include oral antifungals taken in pill form, and topical treatments which are applied directly to the nail.

Monday, October 27, 2025

Treating fibrosis with a chemical derived from Lawsonia inermis

Treatment with Lawsone converts a liver with fibrosis into a healthy liver.
Image Credit: Osaka Metropolitan University

Lawsonia inermis is best known for making henna, a versatile dye that is used to change the color of skin and clothes. Now, researchers from Osaka Metropolitan University have found another use for the pigments extracted from the dye: treating liver disease.

Specifically, they could treat liver fibrosis, a disease that causes excess fibrous scar tissue to build up in the liver as a result of chronic liver injury caused by lifestyle choices such as excessive drinking. Patients with liver fibrosis have increased risks of cirrhosis, liver failure, and cancer. Despite 3–4% of the population having the advanced form of the disease, treatment options remain limited.

Monday, October 20, 2025

New AI Model for Drug Design Brings More Physics to Bear in Predictions

This illustration shows the mesh of anchoring points the team obtained by discretizing the manifold, an estimation of the distribution of atoms and the probable locations of electrons in the molecule. This is important because, as the authors note in the new paper, treating atoms as solid points "does not fully reflect the spatial extent that real atoms occupy in three-dimensional space."
Image Credit: Liu et al./PNAS

When machine learning is used to suggest new potential scientific insights or directions, algorithms sometimes offer solutions that are not physically sound. Take for example AlphaFold, the AI system that predicts the complex ways in which amino acid chains will fold into 3D protein structures. The system sometimes suggests "unphysical" folds—configurations that are implausible based on the laws of physics—especially when asked to predict the folds for chains that are significantly different from its training data. To limit this type of unphysical result in the realm of drug design, Anima Anandkumar, Bren Professor of Computing and Mathematical Sciences at Caltech, and her colleagues have introduced a new machine learning model called NucleusDiff, which incorporates a simple physical idea into its training, greatly improving the algorithm's performance.

Friday, October 17, 2025

Broad-Bayer collaboration leads to drug candidate for a hard-to-treat type of lung cancer

Broad Communications Scientists in the Broad-Bayer oncology alliance have developed a drug candidate, sevabertinib, that could be a new lung cancer treatment.
Illustration Credit: Agnieszka Grosso

An alliance of scientists at the Broad Institute and Bayer Pharmaceuticals have developed a drug candidate, sevabertinib, that could be a new treatment for a group of lung cancer patients who have few options today.

In a new study published in Cancer Discovery, the team described their efforts to develop sevabertinib. They tested the compound in various lung cancer models and showed its potential to treat non-small cell lung cancers that harbor certain mutations in the ERBB2 gene, which encodes the HER2 protein. These mutations occur in 2 to 4 percent of patients with non-small cell lung cancer, or roughly 40,000 to 50,000 people diagnosed globally each year. These patients tend to be women, including those who are younger, have never smoked, and have a poor prognosis. 

The study also reported data from two participants in Bayer’s phase 1/2 clinical trial of the compound. Based on these findings and other data from this ongoing clinical trial, the drug candidate is currently under Priority Review at the FDA, an expedited review of therapies that treat serious conditions. If approved, it would be the first FDA-approved cancer drug based on Broad discoveries, and the first new medicine from the Broad-Bayer oncology research alliance. 

Thursday, October 16, 2025

Muscle wasting reversed in patients with rheumatoid arthritis

Photo Credit: Roger Vaughan

Patients with rheumatoid arthritis increased their leg muscle volume when treated with an anti-rheumatic drug, offering new hope for improved muscle health.

Publishing in the prestigious journal, The Lancet Rheumatology, the team from Newcastle University and The Newcastle upon Tyne Hospitals NHS Foundation Trust describe how 15 patients were given Tofacitinib, a Janus kinase (JAK) inhibitor used to treat RA, as part of an experimental medicine study. After 6 months their leg muscles increased in size, particularly in the thigh.  

Sarcopenia is a progressive, age-related musculoskeletal disease characterized by the loss of muscle mass, strength, and physical performance, increasing the risk of falls, fractures, physical disability, and mortality. Currently there are no medicines approved to reverse this muscle-wasting disease. It is commonly seen in patients with rheumatoid arthritis where chronic inflammation contributes to the loss of muscle mass and strength.

Tuesday, October 7, 2025

Clinical trial marks key milestone in fight against antibiotic resistance

Infections with antibiotic-resistant bacteria cause a significant burden of disease worldwide.
Photo Credit: Scientific Frontline / AI Generated

An international clinical trial, led by The University of Queensland, has been hailed as a significant step forward in the global challenge of antibiotic resistance.

UQ researchers have led the first randomized trial across 6 countries to examine a new antibiotic, cefiderocol, in the treatment of life-threatening, drug-resistant infections.

Associate Professor Patrick Harris, of UQ’s Centre for Clinical Research, said the drug was found to be effective and safe in treating bloodstream infections.

"The study is the first randomized controlled trial to specifically examine the use of cefiderocol in bloodstream infections and sepsis," he said.

“With increasing global antimicrobial resistance, there is a need for the development of new antibiotics.’’

Monday, October 6, 2025

Antibody discovered that blocks almost all known HIV variants in neutralization assays

Image Credit; Scientific Frontline / AI Generated

 A Cologne-led research team has discovered the antibody 04_A06, which neutralizes the human immunodeficiency virus (HIV) in almost all tested variants in vitro and even overcomes typical resistance mechanisms. The discovery potentially opens up new perspectives for the prevention and treatment of HIV infections.

An international research team led by the University of Cologne has discovered an antibody that could advance the fight against HIV. The newly identified antibody 04_A06 proved to be particularly effective in laboratory tests. It was able to neutralize 98.5 percent of more than 300 different HIV strains, making it one of the broadest antibodies against HIV identified. In experiments with humanized mice – animals whose immune system has been modified to resemble that of humans – 04_A06 permanently reduced the HIV viral load to undetectable levels. Most other HIV antibodies, in contrast, only achieve short-term effects in this animal model, as resistance develops quickly. The study ‘Profiling of HIV-1 elite neutralizer cohort reveals a CD4bs bNAb for HIV-1 prevention and therapy’ was published in Nature Immunology.

Sunday, October 5, 2025

New mechanism revealed: How leukemia cells trick the immune system

Thoas Fioretos, Niklas Landberg, and Carl Sandén are the research team behind the study now being published in Nature Cancer.
Photo Credit: Tove Smeds

A research team at Lund University in Sweden has discovered a mechanism that helps acute myeloid leukemia cells to evade the body’s immune system. By developing an antibody that blocks the mechanism, the researchers could restore the immune system’s ability to kill the cancer cells in laboratory trials and in mice. The discovery is published in Nature Cancer.

Immunotherapy has improved the treatment for many cancers, but progress has been limited in leukemia. Acute myeloid leukemia (AML) is particularly intractable, with a five-year survival rate of just over 30 per cent. The existing treatments are often aggressive and may include both strong chemotherapy and stem cell transplantations.

“We wanted to see if we could find surface proteins unique to leukemia stem cells, and which would therefore act as interesting targets for a targeted treatment. If such proteins were not present on healthy blood stem cells it might be possible to attack the tumor – without harming the healthy blood system,” says Thoas Fioretos, research group leader and professor of clinical genetics at Lund University, and senior consultant at Skåne University Hospital.

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