Research on treating disease with medication. Covers psychedelics, combination cancer therapy, opioid treatment in pregnancy, and personalized guidelines.
A new study finds that prothrombin concentrate is superior to frozen plasma in controlling bleeding during heart surgery, reducing major bleeding events by nearly 50%. The concentrated product caused fewer adverse effects and saved hospitals resources, suggesting a potential shift in treatment guidelines.
A new class of zwitterionic phospholipids, DOPE-Cx, enhances the functional delivery of mRNA via lipid nanoparticles, overcoming endosomal escape and improving mRNA expression. This breakthrough paves the way for advanced therapeutic applications, including mRNA vaccines, cancer treatment, and protein replacement therapy.
Researchers found that digital PCR for BCR::ABL1 is more sensitive and accurate than conventional real-time quantitative PCR to detect ultralow levels of residual leukemic disease. The technology enabled the detection of stable deep molecular remission in 97% of patients, allowing for safe discontinuation of treatment.
A team of scientists has created a new method to selectively modify specific proteins in complex biological environments. They achieved this using aptamers and deoxyoxanosine, allowing precise conjugation of desired sites on target proteins. This breakthrough technology has the potential to revolutionize cancer diagnosis and treatment.
Researchers at the University of Houston College of Pharmacy have identified a potential therapeutic target to repair injured muscles. They discovered that fibroblast growth factor–inducible 14 (Fn14) plays a crucial role in regulating satellite cell stability and function, which are responsible for muscle growth, repair, and regenerat...
Researchers at UT Health San Antonio have discovered a way to delay or even block recurrence of the deadliest brain cancer after radiation by targeting senescent cells with experimental 'senolytic' drugs. This approach shows promise in preventing tumor growth and improving patient survival.
Researchers analyzed trends in efficacy endpoints for glioblastoma phase II clinical trials, finding a shift towards using time-to-event measures like progression-free survival (PFS) and overall survival (OS). This trend reflects the complexity of GBM treatment evaluation and may indicate evolving clinical trial design.
The INHALE-3 extension study found that a regimen of inhaled technosphere insulin and insulin degludec reduced post-meal hyperglycemia in adults with type 1 diabetes. The treatment showed significant improvements in mean time in range, with 43% of participants expressing interest in continuing the regimen.
A new study found that recombinant adeno-associated virus (rAAV) capsids contain single-stranded DNA impurities derived from plasmid and host cell DNA. The researchers suggest that the adverse effects of these impurities may differ from those of double-stranded DNA, highlighting the need for further evaluation.
Dr. Owen Witte has been awarded the 12th annual Harrington Prize for Innovation in Medicine for his groundbreaking work on leukemia, lymphoma, and other cancers. His discoveries have led to life-saving therapies like Gleevec and ibrutinib, transforming cancer treatment.
Crew-9 astronauts conducted research on protein crystallization studies and a new drug for Alzheimer's, dementia, and Parkinson's. They also supported student-led experiments, contributing to the next generation of explorers.
A Rice University statistician has received a $1 million CPRIT award to advance AI-powered precision medicine for prostate cancer. The project aims to identify lethal forms of prostate cancer earlier and improve treatment selection using AI tools and advanced imaging techniques.
The WIN Consortium is transforming cancer care through personalized medicine, leveraging AI, molecular profiling, and clinical trials. The organization's innovations, including N-of-1 clinical trials and WINTHER and WINGPO trials, are helping clinicians make more precise treatment decisions and improving outcomes for cancer patients.
Researchers at the University of Zurich have developed a technology to test 25 antibodies simultaneously in a single mouse, greatly reducing the number of laboratory animals required. The method uses protein fragments as barcodes for analysis, allowing for high-quality preclinical data on multiple antibody candidates.
Virginia Tech researchers Pamela VandeVord and Gunnar Brolinson received a $2.17 million grant to investigate cranial osteopathic manual manipulation as a potential treatment for traumatic brain injury symptoms, particularly headaches. The noninvasive technique aims to enhance brain fluid motion and balance the autonomic nervous system.
The study found that tunneling nanotube-like structures connect cells in the heart, enabling long-distance intercellular communication essential for heart formation. Disruption of these structures resulted in impaired ventricular wall morphogenesis and defective myocardial growth.
Researchers identified PDGFRA as a promising therapeutic target for pediatric high-grade gliomas. Inhibition of the PDGFRA signaling pathway leads to tumor cell death and has shown potential in laboratory and animal models, as well as initial clinical experience with avapritinib therapy.
Researchers have discovered how severe COVID-19 can destroy immune cells' ability to repair the lungs, leading to lingering effects of long COVID. By enhancing damaged organelles using a FDA-approved drug, they found improved lung healing and reduced inflammation.
Researchers have discovered a new diterpene-based drug that facilitates the repopulation of mature functional neurons in brain regions damaged by traumatic injuries. The study found that these new cells are integrated into neural circuits and develop functional characteristics similar to those eliminated by the injury.
Researchers discover edonerpic maleate enhances neural plasticity, supporting cortical reorganization and improving motor control in non-human primates with SCI. The compound promotes AMPA receptor movement to areas responsible for motor control, strengthening existing neural connections.
A new study suggests that hospital rehabilitation for older adults might only be the beginning of a longer journey. While therapy is crucial in mitigating functional decline, only a small minority of patients receiving intensive therapy (14+ hours/week) had a greater likelihood of home discharge.
Dr. Ronald Newbold has been appointed as the permanent CEO of Empire Discovery Institute (EDI), a non-profit drug discovery and development accelerator. With over 30 years of business development experience, Dr. Newbold will lead EDI's growth and expansion, including the addition of new programs and partnerships.
Researchers used CRISPR to cut a single gene from cancer cells of head and neck tumors, resulting in the elimination of 50% of the tumors after 84 days. This groundbreaking study demonstrates that some genes are essential for cancer cell survival, making them excellent targets for CRISPR therapy.
The study highlights the role of the Nwd1 gene in liver disease and its potential as a therapeutic target. Mice with Nwd1 gene deletion exhibited liver pathologies mirroring MASH, including excessive lipid accumulation and increased ER stress.
A new study reports that Raman spectroscopy, a noninvasive technique, can distinguish between abnormal FCD type II tissue and healthy brain cells with remarkable accuracy. This method could provide real-time guidance for surgeons to more accurately identify and remove affected tissue during surgery.
Researchers discovered a molecular switch, FLI-1, essential for blood stem cells to enter an activated state. Transiently producing FLI-1 in quiescent adult mobilized bone marrow stem cells activates them, improving their ability to expand and restore the blood cell supply in a new host.
A study published in Human Gene Therapy found that over half of individuals with Niemann-Pick disease type C1 lacked neutralizing antibodies against AAV2 and AAV9. This absence of antibodies may impact the effectiveness of gene therapy treatments for this rare disorder.
Researchers have discovered a family of genes essential for tuberculosis survival during airborne transmission, providing new drug targets. The study sheds light on the bacterium's protective mechanisms, which could prevent infection spread and treat existing cases.
Researchers have developed a next-generation CAR-T cell therapy called ALA-CART that can enhance the effectiveness and longevity of cancer cells against harder-to-treat cancers. The treatment has shown promising results in fighting acute lymphocytic leukemias resistant to traditional CAR-T cells.
A Phase 2 clinical trial found that a neoadjuvant chemo-immunotherapy regimen achieved a 53% deep response rate, exceeding historical data with chemotherapy alone. The treatment approach led to impressive survival outcomes and fewer toxic side effects in patients with advanced HPV-negative head and neck cancer.
A new Northwestern University study found that enhancing the brain's immune cells can clear Alzheimer's plaques and restore a healthier brain environment in immunized patient brains. The findings could reshape the future of Alzheimer's treatments by shifting the focus from removing plaques to harnessing the brain's natural defenses.
Researchers at the University of Missouri have identified a small molecule drug that targets a specific serotonin receptor, showing promise as a treatment for sarcopenia. The study suggests that this neurotherapeutic can improve muscle strength by activating motor neurons to fire more effectively.
Core-shell nanoparticles offer effective drug encapsulation, shielding from degradation, and controlled release. This innovation enables targeted drug delivery, improving treatment outcomes and reducing side effects. The versatility of these nanoparticles allows for tailored materials to suit different therapeutic needs.
The new AI model leverages hypergraphs to quickly and accurately identify therapeutic gene targets for diseases. HIT outperformed existing models in all tested metrics, demonstrating its accuracy in classifying therapeutic gene targets with great precision.
A study found that iron orchestrates the activation of ferroptosis and necroptosis pathways early in ischemic stroke recovery, exacerbating brain injury. Iron disruption fuels neurological damage, highlighting its role in managing reperfusion injury. Iron chelation therapy emerged as an effective approach to mitigate both pathways.
A study published in JAMA Network Open found that a proposed US Drug Enforcement Administration rule could prohibit more than 4,500 tele-buprenorphine initiations for patients with opioid use disorder. The rule requires an in-person visit with the tele-initiating clinician prior to or within 30 days after tele-initiation.
A new study found that people seeking emergency care for hallucinogen use were at a 2.6-fold higher risk of death within 5 years compared to the general population. The increased risk was highest among individuals with medical comorbidities and those from low-income neighborhoods.
The Texas Tech University Health Sciences Center is conducting a randomized controlled trial to investigate the effectiveness of calcium and vasopressin in treating severely injured patients who experience significant blood loss. The study, known as CAVALIER, will enroll approximately 1,050 participants aged 18-90.
Scientists have identified a new target to prevent cold sores by understanding how the herpes virus triggers its own immune response. The discovery has important implications for genital herpes caused by the same virus, with potential treatments in development.
Scientists have identified key corneal neurons involved in sensitivity and developed an experimental model to study nerve regeneration. The Ocular Neurobiology laboratory is participating in the RESTORE VISION project, which aims to improve eye health and quality of life for those affected by rare eye diseases.
Researchers at UCSF have discovered that human lung tissue contains hematopoietic stem cells (HSCs) capable of producing red blood cells, platelets, and immune cells. The finding suggests the lungs could be a potent source for life-saving stem cell transplants, particularly for patients with leukemia.
A nasal spray being tested for Alzheimer's disease could also reduce neuroinflammation in traumatic brain injury, according to a Mass General Brigham-led study. The treatment modulates the immune response, reducing anxiety, cognitive decline, and improving motor skills in mouse models of TBI.
The review delves into genetic, epigenetic, and molecular mechanisms driving tumor metastasis, highlighting the roles of chromosomal instability, epigenetic modifications, and tumor microenvironment interactions. It also discusses the impact of specific genetic mutations and RNA modifications on metastatic behavior.
Researchers have discovered that commonly prescribed medications can disrupt sterol biosynthesis in developing brains, potentially affecting neural development and function. The study highlights the need for reevaluating medication safety during pregnancy and early childhood.
Emerging targeted therapies specifically targeting PIK3CA mutations show promise in preclinical and clinical trials, selectively inhibiting mutant PI3Kα without affecting normal PI3K activity. These novel treatments aim to overcome limitations of existing treatments by improving patient outcomes.
Researchers found that cytomegalovirus infection significantly worsens necrotizing enterocolitis in mouse models, triggering inflammation and disrupting energy production. The study provides insight into the underlying mechanisms of NEC and may lead to the development of new treatments for this devastating condition.
Dermatology researchers have identified a novel skin disease in a male patient with erythroderma, a rare and severe inflammation that causes widespread redness and scaling. Targeted treatment with biologic inhibitors reversed symptoms, providing a promising diagnostic tool for precision-medicine.
US outpatient prescriptions for ivermectin and hydroxychloroquine increased significantly during the COVID-19 pandemic, with usage being three times higher among adults aged 65 and older. The study's findings highlight the need for policy reforms to combat misinformation and mistrust in scientific institutions.
Dr. Gordon Keller's groundbreaking research on directed differentiation of human pluripotent stem cells has illuminated the path to transforming human health. His lab's world-first discovery and pioneering efforts have pushed the boundaries of what is possible, offering hope for regenerating heart, liver, and blood cells.
Researchers at UCSF have identified unique, cancer-specific proteins created through mistakes in RNA splicing. These antigens could be used to create potent immunotherapies that recognize and attack hard-to-treat tumors. The discovery offers new hope for glioma patients and expands the number of targets available for cancer therapy.
A new method combines traditional histopathology with spatial transcriptomics data to improve understanding of chronic kidney disease lesions at the cellular and molecular levels. This approach has the potential to identify new biomarkers and therapeutic strategies for patients.
Researchers developed novel antibiotic BTZ-043 to target drug-resistant tuberculosis. The study showed that BTZ-043 effectively penetrates TB lesions and accumulates there in high concentrations, making it a promising treatment option for TB patients.
Researchers at Virginia Tech have developed a method to convert gut bacteria into mini protein factories that produce and release sustained flows of targeted proteins within the lower intestine. This approach eliminates a major roadblock in delivering drugs to this part of the body, offering potential treatment for chronic diseases.
The Lung Cancer Research Foundation and Israel Cancer Research Fund are partnering to test an innovative new treatment for lung cancer. The project aims to explore the effects of a drug that targets IGF2BP1, a protein involved in cancer cell growth.
A preclinical trial has uncovered how beta-glucan can 'reprogram' immune cells to prevent lung inflammation and reduce the risk of illness and death from influenza. The study's findings highlight beta-glucan as a promising therapy for influenza and other emerging viral pathogens.
A team of researchers from the University of Arizona College of Medicine – Tucson found that an FDA-approved osteoporosis treatment, risedronate, can correct a gene mutation and normalize heart function in animal models. The study provides hope for treating other rare diseases using precision treatments tailored to individual mutations.
Researchers created optimized DNA hydrogels with fewer nucleic acids, achieving efficient and sustained drug release. The new hydrogel units showed prolonged persistence of at least 168 hours post-administration in mice, contributing to anti-tumor effects.
The Damon Runyon-St. Jude Pediatric Cancer Research Fellowship aims to address a funding gap for pediatric cancer research. The program supports innovative projects that could significantly impact the diagnosis or treatment of one or more pediatric cancers.
A study published in Nature Cardiovascular Research found that tricaprin improved long-term survival and recovery from heart failure in patients with triglyceride deposit cardiomyovasculopathy. The supplement reversed structural changes and improved heart muscle function, leading to higher survival rates compared to controls.
Researchers found that flagellin from the gut disrupts immune checkpoint treatment in ovarian cancer patients. Inhibiting this pathway may enhance clinical outcomes and save lives. The discovery could lead to novel therapies capable of targeting ovarian tumors.