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Category: biotech/medical – Page 15

Anti-ANGPTL3 Antibody SHR-1918 for Homozygous Familial Hypercholesterolemia: A Nonrandomized Clinical Trial

Monthly subcutaneous SHR-1918, a fully human monoclonal antibody targeting ANGPTL3, was associated with reduced LDL-C in adults with homozygous familial hypercholesterolemia on stable lipid-lowering therapy.

Question Does SHR-1918, a fully human monoclonal antibody targeting angiopoietinlike 3 (ANGPTL3), lower the low-density lipoprotein cholesterol (LDL-C) level in adults with homozygous familial hypercholesterolemia (HoFH) taking stable lipid-lowering therapy?

Findings In this phase 2 nonrandomized clinical trial of 26 patients, SHR-1918 at 600 mg every 4 weeks was associated with a substantial reduction in LDL-C level exceeding half in adults with HoFH taking stable lipid-lowering therapy and was also associated with lower levels of other lipids, with evidence of a manageable safety profile.

Meaning The promising findings observed in this trial support the launch of a double-blind, placebo-controlled, phase 3 randomized clinical trial to verify the effect and safety of SHR-1918 for HoFH management.

Heart-brain connection via vagus nerve to keep the heart young

The secret to a healthier and “younger” heart lies in the vagus nerve. A recent study published in Science Translational Medicine has shown that preserving bilateral cardiac vagal innervation is an anti-aging factor. In particular, the right cardiac vagus nerve emerges as a true guardian of cardiomyocyte health, helping to preserve the longevity of the heart independently of heart rate.

‘When the integrity of the connection to the vagus nerve is lost, the heart ages more rapidly,’ explains the senior author.

‘Even partial restoration of the connection between the right vagus nerve and the heart is sufficient to counteract the mechanisms of remodelling and preserve effective cardiac contractility,’ adds another author.

‘We have developed an implantable bioabsorbable nerve conduit designed to promote and guide the spontaneous regeneration of the thoracic vagus nerve at the cardiac level,’ explains a co-author.

Treated adult male minipigs displayed improved global circumferential, longitudinal, and radial strains and reduced diastolic dyssynchrony. Histological analysis revealed partial repair with about 20% viable vagal fascicles, restoration of myocardial parasympathetic fibers, normalization of oxidative stress and aging markers, and prevention of interstitial fibrosis.

Dr. Christie M. Ballantyne & Dr. Alexander Tal — Oral PCSK9 Therapy And The Future Of Heart Disease

Oral PCSK9 Therapy And The Future Of Heart Disease — Dr. Christie Ballantyne MD, Director, Center for Cardiometabolic Disease Prevention, Baylor College of Medicine & Dr. Alexander Tal, MD.

Dr. Christie M. Ballantyne, MD is a Cardiologist and is one of the nation’s foremost experts on lipids, atherosclerosis and heart disease prevention. He holds many leadership positions at Baylor College of Medicine (https://www.bcm.edu/people-search/chr… including director of the Center for Cardiometabolic Disease Prevention, co-director of the Lipid Metabolism and Atherosclerosis Clinic, and chief of the Section of Cardiology.

With over 1,000 publications in the area of atherosclerosis, lipids, and inflammation, Dr. Ballantyne’s research on heart disease prevention has led him to become an established investigator for the American Heart Association and the recipient of continuous funding from the National Institutes of Health with a core focus on in basic research of leukocyte–endothelial interactions, translational research in biomarkers, and clinical trials.

Dr. Ballantyne’s many accomplishments have included being elected as Fellow of the American Association for the Advancement of Science, the American Society of Clinical Investigation, and the Association of American Physicians. In 2012, he received the American College of Cardiology Distinguished Scientist Award (Basic Domain).

In 2014 and 2015, Thomson Reuters recognized Dr. Ballantyne as one of “The World’s Most Influential Scientific Minds.” Clarivate Analytics, Web of Science, named Dr. Ballantyne as a “Highly Cited Researcher” 2017–2022 in the top 1% of researchers most cited.

Continue reading “Dr. Christie M. Ballantyne & Dr. Alexander Tal — Oral PCSK9 Therapy And The Future Of Heart Disease” | >

One of the Most Common Diabetes Treatments May Be Making the Disease Worse, Study Warns

A new study suggests that one of the most common diabetes treatments may speed type 2 diabetes progression by causing insulin-producing cells to lose their functional identity. Sulphonylureas have been used to treat type 2 diabetes since the early 1950s and remain among the most frequently prescr

Semaglutide in Schizophrenia Spectrum Disorders

Semaglutide reduced HbA1c and body weight over 26 weeks in individuals with schizophrenia spectrum disorders and early glycemic abnormalities taking clozapine or olanzapine.

Question Can adjunctive semaglutide improve glycemic control and weight outcomes in individuals with early-stage prediabetes or diabetes and schizophrenia spectrum disorders who initiated clozapine or olanzapine within the past 5 years?

Findings In this randomized clinical trial including 73 participants, semaglutide was found to significantly reduce hemoglobin A1c level and body weight over a 26-week period. Approximately one-half of individuals treated with semaglutide achieved low-risk hemoglobin A1c levels, compared with the placebo group.

Meaning This study found that semaglutide can mitigate the early metabolic burden associated with second-generation antipsychotic use in schizophrenia and may support the prevention of long-term cardiometabolic complications when initiated during the early stages of metabolic dysregulation.

Researchers Discover Molecular Difference in Autistic Brains

A study co-led by Richard Carson and colleagues at the Yale School of Medicine has identified a measurable molecular difference in the brains of autistic adults – finding reduced availability of a key glutamate receptor involved in brain signaling balance.

Published in The American Journal of Psychiatry, the research offers new insight into the biological mechanisms of autism and could inform future diagnostic tools and targeted supports.

👉 Read the full story.

Brains of autistic individuals have fewer of a specific kind of glutamate receptor, supporting an idea that autism is driven by a signaling imbalance.

CRISPRi screening in cultured human astrocytes uncovers distal enhancers controlling genes dysregulated in Alzheimer’s disease

2026 (Nature Neuroscience)

• AstroREG, a resource of enhancer–gene interactions in human primary astrocytes, generated by combining CRISPR inhibition (CRISPRi), single-cell RNA-seq and machine learning.

This study reveals how distal DNA ‘switches’ control gene activity in human astrocytes. Using CRISPRi screens and single-cell RNA-seq, we map enhancer–gene links, highlight Alzheimer’s disease-related targets and introduce a model that predicts additional regulatory interactions.

Blood-cleansing method faces its first test as cancer vaccine

Goodrich admits that when his group first proposed the strategy, “everyone said we were crazy.” Researchers have been trying to develop vaccines that contain whole cancer cells for more than 50 years, and although some formulations made it to clinical trials, they produced a poor immune response. None of the vaccines has been approved for humans, although one is available for pets.

The harsh methods previously used to stop cancer cells from reproducing, such as radiation, also caused them to shed their neoantigens, Goodrich says. He argues that the UV-based approach should work better because it preserves these potential immune stimulants.

The new clinical trial, launching this month at City of Hope in California and sponsored by PhotonPharma, aims to recruit eight patients with relapsed ovarian cancer. They will first undergo surgery to remove their tumors. Researchers will then expose the tumor cells to riboflavin and UV light and combine them with an immune-boosting additive known as an adjuvant to produce a custom vaccine. Participants will receive three doses of the vaccine, and researchers will check for side effects and measure immune responses.

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