NATIONAL INSTITUTES OF HEALTH

A phase 2 trial of a hormonal gel for male contraception showed 86% sperm suppression in 222 men, achieving effective contraception in eight weeks. The gel combines testosterone and Nestorone, acting faster with less testosterone. Interest in male contraceptives is rising, with surveys indicating willingness among men to use new methods.

FDA advisers unanimously recommend approving Alzheimer’s drug donanemab, highlighting its effectiveness in early stages and benefits outweighing risks. It could allow halting treatment if amyloid plaques are removed, potentially within a year. Concerns include brain swelling/bleeding risks, especially for APOE4 gene carriers, but advocates stress the need for aggressive treatment options.

NIH researchers developed an AI tool, LORIS, predicting cancer response to immune checkpoint inhibitors using routine clinical data. It evaluates age, cancer type, therapy history, blood albumin, neutrophil-to-lymphocyte ratio, and tumor mutational burden. The model, tested on 2,881 patients, accurately predicts treatment response and survival, identifying effective immunotherapy candidates beyond current biomarkers.

In 2022, over 100,000 US deaths were due to drug overdoses, highlighting the urgent need for new solutions like medical devices to combat the opioid crisis. A 2022 FDA and NIDA workshop emphasized the importance of novel monitoring and diagnostic devices, patient engagement, and ecosystem-wide partnerships to develop effective solutions.

The first patient has been dosed in the ONKORAS-101 trial for BBO-8520, a novel KRASG12C inhibitor for non-small cell lung cancer. BBO-8520 targets both ON and OFF states of KRASG12C, aiming to overcome resistance to current treatments and improve patient outcomes. The trial spans the US, Australia, Canada, and the EU.

Uproleselan showed significant improvement in median overall survival (31.2 months) for primary refractory AML patients compared to placebo (10.1 months). Adverse events aligned with chemotherapy profiles. GlycoMimetics plans further studies with NCI and Alliance for Clinical Trials in Oncology, focusing on frontline AML in older adults.

NIH researchers developed an AI tool, LORIS, using routine clinical data to predict cancer response to immune checkpoint inhibitors. The model, based on five clinical features and tumor mutational burden, accurately predicts treatment response and survival, identifying effective immunotherapy candidates beyond current biomarkers. Published in Nature Cancer, it's available for public use.

The Shaw Prize in Life Science and Medicine 2024 is awarded to Swee Lay Thein and Stuart Orkin for their groundbreaking work on the fetal-to-adult hemoglobin switch, enabling a genome-editing therapy for sickle cell anemia and β thalassemia, diseases affecting millions globally. Their research offers hope for effective treatments against these devastating blood disorders.

A new AI algorithm, EXPRESSO, developed by Penn State researchers, enhances predictions and therapies for autoimmune diseases by analyzing genetic data more accurately. It identified 26% more gene-trait associations, offering insights into cell-type specific effects and potential drug repurposing for diseases like lupus and Crohn's.

Using a supercomputing platform, researchers analyzed protein structures to identify novel drug-binding sites, leading to the discovery of Dxr2-017, a compound effective against melanoma with minimal toxicity. This approach leverages unique 3D protein motifs for drug discovery, demonstrating the potential of protein structure as primary data for therapeutic development.