Articles tagged with Translational Medicine
The FDA has accepted Genentech's supplemental Biologic License Application for atezolizumab, supported by phase III ATOMIC trial results. The trial showed a 50% reduction in cancer recurrence or death and 86.3% disease-free survival rate.
Researchers describe a novel disorder caused by biallelic loss-of-function variants in the TMEM63B gene, resulting in severe lung disease. The disorder presents with early onset respiratory distress, lung abnormalities, and developmental delay but no epilepsy.
Researchers developed a topical gel formulation with 4-aminopyridine to treat burn wounds, achieving near-complete closure in 21 days. The gel delivers the drug directly to the wound site, avoiding systemic risks associated with prolonged use.
Researchers at CNIC identify mitochondrial complex I as a key checkpoint for dendritic cell activation. Restoring its function restores immune responses against viruses and tumors. The study paves the way for new strategies in vaccines and cancer immunotherapies.
The UMass Center for Clinical and Translational Science has received a seven-year, $50 million grant from the NIH. The funding supports collaboration among researchers at multiple UMass campuses and clinical partners.
Odylia Therapeutics is advancing a gene therapy treatment for Usher Syndrome Type 1C (USH1C)-associated vision loss, a rare autosomal recessive disorder. The grant will support the development of an investigational USH1C gene therapy designed to restore or preserve vision.
A new study by the University of Southampton has found that reusable catheters are just as safe for patients as single-use ones and do not increase the risk of urinary tract infections. The study followed hundreds of patients for a year and discovered that those who tested reusable catheters used 35 per cent fewer antibiotics.
The UK-led OpenBind initiative has released its first publicly available dataset and predictive AI model, accelerating the discovery of new medicines using artificial intelligence. The release showcases high-quality, standardized experimental data and a trained predictive model, enabling researchers worldwide to drive the next generati...
A new study published in Eye and Vision finds that one drop of low-dose atropine can produce daylong effects in managing myopia. The researchers found that the drop showed clear changes in pupil size and focusing ability, but no short-term structural effects on the eye.
Researchers at Johns Hopkins Medicine have made progress in transplanting retinal ganglion cells to restore vision in people with optic nerve damage. By disrupting the internal limiting membrane, a thin layer of tissue that blocks the growth and survival of transplanted nerve cells, scientists have seen improved transplantation surviva...
The new consensus statement provides updated guidance on CIED lead management, including extraction and management of traditional devices and newer technologies. The recommendations aim to reduce clinical challenges and morbidity within a rapidly evolving technological landscape, ensuring better quality of life for patients.
Five fellows will receive funding for four years to study hard-to-treat pediatric cancers, reduce treatment toxicity, and develop innovative therapies. Their research focuses on Ewing sarcoma, leukemia, kidney cancer, and immune system vulnerabilities.
A first-in-human trial shows that an infusion of immune cells derived from a donor's blood can prime the recipient's immune system to accept a donor liver, potentially reducing lifelong immunosuppression. Eight out of 13 participants achieved complete withdrawal of immunosuppression and remained off for more than three years.
Scientists at the University of Virginia Health System have developed a suite of AI-powered tools, called YuelDesign, YuelPocket and YuelBond, to transform how new drugs are created. These tools can design drug molecules tailored to fit their protein targets exactly, even accounting for protein flexibility.
Aging is redefined as a systems-level failure rather than molecular defects, highlighting the need for coordinated modulation of biological networks. The International Conference on Targeting Longevity 2026 explores new translational strategies and industrial opportunities by focusing on resilience.
A new study found that the common blood pressure medication telmisartan can significantly enhance the cancer-killing activity of olaparib, potentially expanding its use to many more patients. Telmisartan made tumors more vulnerable to PARP inhibitors, even when they lacked specific DNA repair defects.
Dr. Gobbi challenges the global drug-development system, stating that promising treatments for mental illness are failing to reach patients due to venture capital and profit motives. Her research focuses on cannabis, melatonin, and psychedelics, with a goal of improving public-health decisions and treatment outcomes.
The NRG-LU005 trial found that immunotherapy atezolizumab did not improve survival for patients with limited-stage small cell lung cancer when added to chemoradiation. Twice-daily radiation therapy, however, was associated with improved survival in this population.
The ISSCR has launched a new continuing education course on stem cell therapies for Parkinson’s disease, addressing the need for clinicians to provide accurate information on emerging treatments. The course features expert faculty and patient perspectives, and is available through a free and paid pathway.
Severe COVID-19 and influenza infections can prime the lungs for cancer development, according to new research. Vaccination, however, largely prevents these changes, suggesting a reduced risk of lung cancer.
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.
Researchers have discovered a novel biomarker for fragile X syndrome in both human patients and mouse models, allowing for the comparison of brain wave patterns between species. This breakthrough enables the development of more effective treatments by enabling non-invasive treatment efficacy readouts across species.
A clinical prediction model called the Goliat score can estimate the risk of acute complications in patients with minor and moderate traumatic brain injuries. The model, developed by researchers at UOC and IRBLleida, uses easily accessible variables to predict neurological or cardiorespiratory complications and mortality within 48 hours.
Cancer cells tap into the nervous system's power grid by forming synaptic contacts with nerve cells, promoting tumor growth and spread. Venkataramani's research aims to repurpose the drug perampanel for glioblastoma treatment and develop gene therapy approaches to disconnect tumors from the nervous system.
Researchers developed bioengineered lymphatic tissue (CeLyT) that restored functional lymph nodes in mice with secondary lymphedema. CeLyTs improved lymphedema symptoms by restoring lymphatic flow, filtration capacity, and immune cell populations.
The ISSCR has released a comprehensive roadmap for developing pluripotent stem cell-derived therapies, outlining critical scientific, manufacturing, and regulatory guidance. The Best Practices document provides a risk-based, end-to-end development strategy to support informed decision-making across seven essential areas.
A two-step genome editing method integrates large human genomic fragments into mice, mimicking human regulatory landscapes. This platform enables the creation of physiologically relevant humanized models for therapeutic targets and disease research.
Researchers at the University of Virginia Health System have discovered how the keto diet protects against epilepsy seizures by regulating brain cell activity. The team found that a specific cellular receptor, HCAR2, plays a crucial role in reducing seizures, and may be targeted for new treatments.
Dr. Noritaka Ichinohe challenges the long-held assumption that averaging away individual differences is necessary in psychiatric research. Instead, biological heterogeneity is seen as a phenomenon demanding explanation. The author's work on primate brain mapping and molecular subtyping has identified convergence points between species ...
A new study from Texas A&M University reveals that circadian disruptions change the structure of mammary glands, weaken immune defenses, and fuel aggressive breast cancer. Disabling an immune checkpoint molecule called LILRB4 helps restore the immune system's ability to fight back.
A team of researchers has identified two proteins critical to the destruction of red blood cells by the Oroya fever pathogen. This discovery paves the way for a potential novel therapy against this deadly neglected tropical disease.
The ESMO Immuno-Oncology Congress 2025 will discuss key developments in immuno-oncology, including combined approaches, AI in cancer therapy, and innovative strategies across various cancers. The congress aims to advance the field of immunotherapy and improve patient outcomes.
A new paper from the University of Kansas overturns the idea that gene PTPN22 boosts production of Type 1 interferon, a key antiviral response. Researchers found no difference in interferon production when PTPN22 was knocked out, but discovered its influence on other immune signaling pathways.
Researchers at UC Riverside and City of Hope have developed a novel Pin1 degrading compound that suppresses pancreatic cancer peritoneal metastases. The treatment targets not only cancer cells but also tumor-supporting cells, potentially overcoming treatment resistance.
Researchers at UMC Utrecht are developing a predictive model to accurately predict disease progress in juvenile dermatomyositis. The Interferon Score aims to identify which children can safely reduce medication and which require intensified treatment.
A new AI model uses machine learning to predict drug toxicity in humans by identifying biological differences between cells, mice, and humans. The model improved predictive power over existing state-of-the-art models and demonstrated practicality in predicting market withdrawal due to toxicity.
The International Society for Stem Cell Research invites you to celebrate 20 years of induced pluripotent stem cell (iPSC) discovery and chart its future. The symposium features keynote speakers, featured sessions, and presentations on cutting-edge research.
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 new Alliance trial is exploring the effectiveness of a combination of targeted therapy and immunotherapy for patients with advanced adrenocortical carcinoma, a rare and aggressive cancer. The study aims to improve disease control and quality of life for patients with limited treatment options.
The UC San Diego REACH Center for Translational Science on Whole Person Health aims to advance research through partnerships with complementary and integrative health institutions. The center will support at least 26 new research scholars and fund pilot grants to drive innovation in translational science.
Rebecca Richards-Kortum has been elected to the National Academy of Medicine for her major contributions to global health. She created low-cost, lifesaving technologies for underserved communities, transforming health systems worldwide through her innovations in cervical cancer diagnostics and neonatal care technologies.
A new study has identified a leptin-sensitive brain circuit in the lateral hypothalamus that helps animals overcome anxiety to perform essential behaviors such as eating, exploring, and limiting maladaptive hyperactivity. Boosting the activity of these neurons can restore adaptive choices even under conditions that cause anxiety.
Scientists found a promising candidate, pleiotrophin, which is essential for brain development and function; restoring it may improve brain circuits in individuals with Down syndrome and other neurological diseases. The study's findings suggest using modified viruses to deliver the protein directly into cells could lead to new treatments.
Researchers developed TAMENDOX to supplement (Z)-endoxifen in patients with CYP2D6 enzyme deficiency, improving drug concentration and effectiveness. The new therapy was well-tolerated, showing promise as an alternative to aromatase inhibitors for premenopausal women.
Researchers at UMass Amherst have developed a nanoparticle-based vaccine that prevents melanoma, pancreatic and triple-negative breast cancer in mice. The vaccine achieved remarkable survival rates, with up to 88% of vaccinated mice remaining tumor-free.
Colin Hill joins C-Path's Board of Directors with experience in AI, drug development, and translational acceleration. He aims to bring precision medicine focus to advance treatments worldwide.
The International Federation of Sports Medicine (FIMS) has partnered with Translational Exercise Biomedicine (TEB), the first such collaboration in Asia. This partnership aims to enhance research publication, host co-branded events, and advance global advocacy for sports medicine.
Dr. Michael Welsh's research on cystic fibrosis paved the way for a triple-drug combination therapy that has transformed CF from a fatal condition into a manageable one for over 90% of patients. His work also revealed the function and 'broken' nature of the CFTR protein, laying the foundation for targeted therapies.
A non-electronic soft robotic device guides a soft tube into the trachea, improving success rates for both expert and non-expert users. The device yields a 100% success rate with highly trained users and an 87% success rate with non-expert providers after just five minutes of training.
Researchers found that frail patients with residual intra-abdominal bacteria or fungi are at a higher risk of developing postoperative ileus and infections. The study suggests that incorporating frailty assessment and prehabilitation programs into surgical planning may help reduce complications.
The study found that increasing Piezo1 activity leads to a coordinated response, resulting in increased skin surface area and thickness. The findings could ultimately help doctors develop noninvasive therapeutic approaches to create new skin for treating burns and other injuries.
Researchers at Institute of Science Tokyo found that basophils suppress lung inflammation during ARDS recovery, suggesting potential treatment strategies. Basophils release interleukin-4, which helps immune cells coordinate responses and alleviate lung inflammation.
Researchers at Lehigh University and the Cleveland Clinic are developing a nonsurgical therapy for pelvic organ prolapse using drug-delivering nanoparticles. The treatment aims to delay or reverse matrix degradation, reducing the severity of POP in patients with earlier stages of the disorder.
Researchers discovered that GLP-2 analogs reshape the intestinal environment by expanding villi and crypts, increasing regulatory T cells, and improving nutrient absorption. This breakthrough offers new hope for patients with short bowel syndrome and potentially benefits immunocompromised individuals.
A wearable robot has been upgraded to provide personalized assistance to ALS and stroke patients. The device uses machine learning and a physics-based model to adapt to an individual user's movements, offering more nuanced help with daily tasks.
A recent study by researchers at the University of Missouri has made a groundbreaking discovery using patient's own cartilage cells to aid in healing after hip surgery. The research team successfully cultured and expanded cartilage cells, or chondrocytes, in the lab, paving the way for future clinical applications.
Scientists have created a detailed map of cell behavior in the small intestine of people with coeliac disease using single-cell RNA sequencing. The study found that immune signals from immune system proteins encourage fibroblasts to support changes in the gut lining, suggesting communication between cells plays a key role in the disease.
Researchers are developing an AI imaging approach to distinguish between tumor progression and treatment effects in glioblastoma patients, promising to improve care and outcomes. The AI approach has already shown accuracy rates of up to 74% in initial testing.
Researchers at Syracuse University have identified a new target for treating obesity and diabetes, reducing nausea and vomiting associated with existing treatments. A molecule called tridecaneuropeptide (TDN) produced by brain support cells suppresses appetite and improves glucose processing without adverse effects.