HIV Vaccine Breakthrough: Clinical Trials Show Promise for Broadly Neutralizing Antibody Development
• Two phase 1 clinical trials demonstrate successful activation of rare immune cells needed to develop broadly neutralizing antibodies against HIV, marking significant progress in vaccine development.
• The innovative stepwise vaccination strategy using mRNA technology showed 100% success rate in generating VRC01-class antibody responses in participants who received both prime and boost doses.
• Results from trials conducted in North America and Africa showed similar immune responses, supporting the potential for a globally effective HIV vaccine targeting regions most affected by the pandemic.
Breakthrough at UT Health San Antonio: Chemical Endocytic Strategy Could Transform IV Drugs into Oral Treatments
• Researchers at UT Health San Antonio have developed "chemical endocytic medicinal chemistry," a novel approach that could enable large-molecule drugs to be taken orally rather than intravenously.
• The discovery leverages CD36 protein receptors on cell surfaces to facilitate cellular uptake of large and water-soluble drugs, potentially overcoming the blood-brain barrier for treating conditions like brain cancer and Alzheimer's disease.
• This paradigm shift in drug delivery could revolutionize pharmaceutical development, resurrect previously abandoned drug candidates, and enable more personalized medicine based on patients' varying CD36 expression levels.
Novel Radiotherapeutic Rhenium Obisbemeda Doubles Survival Time in Glioblastoma Patients
• A Phase 1 trial led by UT Health San Antonio demonstrated that Rhenium Obisbemeda more than doubled median survival time to 17 months in recurrent glioblastoma patients receiving high doses, compared to the typical 8-month survival.
• The investigational drug uses nanoliposome technology to deliver targeted radiation directly to brain tumors via convection enhanced delivery (CED), bypassing the blood-brain barrier while sparing healthy tissue.
• Plus Therapeutics' Rhenium Obisbemeda recently received FDA Orphan Drug Designation for treating leptomeningeal metastases in lung cancer patients, with Phase 2 trials for glioblastoma expected to complete by year-end.
Novel Senolytic Drug Shows Promise for MASLD and Liver Cancer Treatment
• Researchers from UT Health San Antonio and Tulane University have developed a new drug candidate that effectively eliminates senescent "zombie cells" from the liver, showing potential for treating MASLD and liver cancer.
• The drug targets BCL-xl and BCL-2 proteins to induce senescent cell death, demonstrating superior efficacy in reducing liver damage and cancer growth compared to existing senolytics, with fewer side effects.
• The breakthrough discovery addresses a critical healthcare need in San Antonio, where MASLD prevalence is high due to elevated rates of obesity and diabetes in the community.
PharmAla to Develop Novel MDMA Dosage Form for Military PTSD Trial at UT Health San Antonio
• PharmAla Biotech will supply both 40mg and new 20mg LaNeo MDMA capsules for a Department of Defense-funded PTSD treatment trial targeting active-duty military personnel.
• The STRONG STAR Consortium at UT Health San Antonio secured $10M in Defense Appropriations Act funding to conduct groundbreaking research on MDMA-Assisted Therapy for military populations.
• The study represents one of the first investigations into MDMA-Assisted Therapy specifically designed for active-duty military personnel, with trials to be conducted at UT Health San Antonio and Emory University.
BerGenBio's Bemcentinib Enters Clinical Trial for Advanced Lung Adenocarcinoma
• BerGenBio's bemcentinib is being evaluated in a Phase Ib/II clinical trial in combination with pacritinib for advanced lung adenocarcinoma.
• The trial, sponsored by the Mays Cancer Center, aims to address unmet needs in lung cancer treatment by targeting AXL and JAK-STAT3 pathways.
• Bemcentinib, a selective AXL kinase inhibitor, is combined with pacritinib, a JAK2 inhibitor, to combat tumor growth and metastasis.
• The study, involving 44 patients, is funded by the National Cancer Institute and focuses on safety, tolerability, and efficacy.
Smartphone App Augments Medication to Reduce Opioid Use, Improve Retention
• A recent study reveals that a smartphone app, when used with medication for opioid use disorder (MOUD), significantly reduces opioid use among patients.
• The research indicates that patients using the app alongside MOUD reported 35% fewer days of opioid use compared to those receiving MOUD alone.
• The study also found that app users remained in treatment nearly 19% longer, highlighting the app's potential to improve patient retention.
• The WEconnect Health CM app offers contingency management and recovery support, providing a comprehensive approach to opioid use disorder treatment.
Mifepristone Demonstrates Significant HbA1c Reduction in CATALYST Trial for Hypercortisolism and Difficult-to-Control Type 2 Diabetes
• Mifepristone (Korlym) achieved a statistically significant 1.32% placebo-adjusted reduction in HbA1c in patients with hypercortisolism and difficult-to-control type 2 diabetes.
• The CATALYST trial revealed that approximately 23.8% of patients with difficult-to-control type 2 diabetes also have hypercortisolism.
• The safety profile of mifepristone in the CATALYST trial was consistent with its known safety profile, with no new adverse events identified.
• Complete results from the CATALYST trial will be presented at an upcoming medical conference, offering further insights into mifepristone's efficacy and safety.
PSMD Neuroimaging Marker Shows Promise in Identifying Dementia Risk
• A new neuroimaging marker, peak-width of skeletonized mean diffusivity (PSMD), correlates with general cognition and may identify individuals at risk of dementia.
• The study found that higher PSMD values were associated with lower general cognitive function, independent of age, sex, education and intracranial volume.
• PSMD demonstrates excellent instrumental properties and biological validation, making it a potential biomarker for clinical trials related to vascular contributions to cognitive impairment and dementia (VCID).
• Researchers suggest PSMD's non-invasive, automated, and reliable nature makes it ideal for assessing cerebral small vessel diseases in dementia studies.
FDA Grants Orphan Drug Designation to Kind Pharmaceutical's AND017 for Sickle Cell Disease
• The FDA has granted Orphan Drug Designation to AND017, a drug developed by Kind Pharmaceutical, for the treatment of Sickle Cell Disease (SCD).
• AND017 is a first-in-class hemoglobin elevating agent (HbEA) targeting multiple stages of the red blood cell life cycle.
• The ODD provides Kind Pharmaceutical with incentives, including tax credits and potential market exclusivity for AND017 in SCD treatment.
• AND017 is also being developed for anemia associated with chronic kidney disease, cancer, myelodysplastic syndromes and β-thalassemia.
UT Health San Antonio Investigates Gut-Organ Miscommunication in Obesity and Diabetes
• Dr. Marzieh Salehi challenges the myth that obesity is solely caused by calorie imbalance, emphasizing the role of gut-organ communication.
• A clinical trial will study semaglutide's impact on blood glucose regulation in spinal cord injury patients with type 2 diabetes.
• Research aims to understand how semaglutide affects gut-organ communication, potentially leading to precision medicine approaches for metabolic conditions.
• The NIH-funded study seeks to uncover specific communication issues in high-risk populations, like those with spinal cord injuries.
Ohtuvayre (Ensifentrine) Shows Efficacy Across COPD Subgroups in Phase 3 ENHANCE Analyses
• Verona Pharma's Ohtuvayre (ensifentrine) demonstrates consistent efficacy in improving lung function, symptoms, and quality of life for COPD patients, regardless of disease severity.
• Analyses from the Phase 3 ENHANCE trials reveal that ensifentrine reduces exacerbation rates and improves lung function in both current and former smokers with COPD.
• The studies also show Ohtuvayre's benefits extend to COPD patients with or without chronic bronchitis, suggesting broad applicability in managing COPD.
• These findings, presented at CHEST 2024, support Ohtuvayre as a valuable addition to COPD therapy, offering bronchodilation and anti-inflammatory effects.
Dupixent Approved by FDA as First Biologic for COPD with Eosinophilic Phenotype
• The FDA has approved Dupixent (dupilumab) as an add-on maintenance treatment for adults with inadequately controlled COPD and an eosinophilic phenotype.
• Approval was based on Phase 3 trials showing significant reductions in COPD exacerbations and improvements in lung function and quality of life compared to placebo.
• Dupixent targets the IL-4 and IL-13 pathways, offering a novel approach for COPD patients with type 2 inflammation and elevated blood eosinophils.
• Sanofi and Regeneron's Dupixent is now the first and only approved add-on biologic medicine for inadequately controlled COPD in the US.
Semaglutide Investigated for Diabetes, Obesity in Spinal Cord Injury Patients
• A clinical trial is underway to investigate the mechanisms behind diabetes and obesity in individuals with spinal cord injuries.
• The study will assess the safety and efficacy of semaglutide, a GLP-1 receptor agonist, in treating these metabolic conditions in this population.
• Researchers aim to understand how semaglutide impacts glucose tolerance, insulin action, and body composition in patients with spinal cord injury.
• The trial seeks to address the lack of research and treatment guidelines for diabetes and obesity in this high-risk population.
Therapeutic Trials for Long COVID-19: A Call to Action
The article discusses the ongoing challenges and research efforts in understanding and treating Long COVID, also known as Post-Acute Sequelae of SARS-CoV-2 infection (PASC). It highlights the need for therapeutic trials to address the persistent symptoms affecting a significant number of individuals post-acute SARS-CoV-2 infection. The National Institutes of Health's RECOVER initiative is mentioned as a key effort in this area, focusing on understanding the biological mechanisms behind Long COVID and exploring potential treatments.
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