Spinal Delivery of Gene Therapy Promising for Neurodegenerative Disease
Gene therapy delivered for the first time via lumbar puncture showed promise of benefit in slowing progression of giant axonal neuropathy (GAN) in a phase I clinical trial. The slope of change in motor function went from -7.17 percentage points per year before treatment to -0.54 percentage points with the lowest dose to an improvement […]
Novel CAR-T for Glioblastoma Induced ‘Dramatic’ Responses in Early Trial
A new approach to chimeric antigen receptor (CAR) T-cell therapy showed promise in treating recurrent glioblastoma, according to a small phase I study. In the first-in-human trial, three patients with recurrent glioblastoma who were treated with CARv3-TEAM-E T cells experienced “dramatic and rapid” tumor regression within days of receiving a single intraventricular infusion, reported Marcela […]
Military cannot rely on AI for strategy or judgment, study suggests
Using artificial intelligence (AI) for warfare has been the promise of science fiction and politicians for years, but new research from the Georgia Institute of Technology argues only so much can be automated and shows the value of human judgment. “All of the hard problems in AI really are judgment and data problems, and the […]
Presepsin a ‘Robust’ Biomarker of Early-Onset Sepsis
Presepsin holds promise as a biomarker for early-onset sepsis (EOS), a systematic review and meta-analysis confirmed. Across a dozen clinical trials, presepsin had a pooled sensitivity of 0.93 for the primary analysis of culture-proven sepsis (95% CI 0.86-0.96), a pooled specificity of 0.91 (95% CI 0.85-0.95), and a pooled diagnostic odds ratio (DOR) of 131.7 […]
Remote BP Monitoring Dips a Toe Into Post-Acute Care
Remote physiologic monitoring (RPM) of blood pressure (BP) showed promise for post-acute patients transitioning to outpatient care, a small pilot study found. Twenty hypertension patients were discharged from Tennessee post-acute care facilities (e.g., long-term care hospitals, inpatient rehabilitation facilities, and skilled nursing facilities) and given a Bluetooth- or cellular-connected BP monitor and trained to use […]
Meds, surgery may help obesity-related high blood pressure if diet, exercise fall short
Weight-loss medications and surgical procedures show promise for reducing the long-term effects of high blood pressure (also called hypertension) in people who are overweight or have obesity, according to a new American Heart Association scientific statement published today in the Association’s journal Hypertension. A scientific statement is an expert analysis of current research and may […]
Second-Gen Drug for HCM Emerges, Nipping at Mavacamten’s Heels
A second-generation cardiac myosin inhibitor showed promise for the treatment of obstructive hypertrophic cardiomyopathy (HCM) in the phase II REDWOOD-HCM trial. In the dose-finding study, 10 weeks of treatment with aficamten brought resting left ventricular outflow tract (LVOT) gradients down significantly compared with placebo before a 2-week washout period returned gradients to baseline, reported Martin […]