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A phase 1 trial of PSCA-targeting CAR T cell therapy in patients with PSCA-positive, metastatic castration-resistant prostate cancer demonstrates that the treatment, using a protocol with reduced lymphodepletion, is safe and shows preliminary clinical activity.
This study distinguished age- and metabolism-related gut microbiota signatures and constructed gut microbial age metric using data from a large, prospective cohort. Longitudinal analysis showed that younger microbial age seems to counteract the increased cardiovascular disease risk in metabolically unhealthy older people, indicating an interplay between gut microbiota and host age and metabolism.
We quantified liver, pancreas, heart and kidney fibrosis using MRI T1 mapping in over 40,000 individuals. Using genetic association analyses, we identified a total of 58 loci, 10 of which overlapped across organs. A high burden of fibrosis in three or more organs was associated with an increased risk of mortality.
The largest genome-wide association study of its kind has identified new markers for risk prediction in women of African ancestry — helping to bridge disparities in clinical genomics.
Phase 2 clinical trial results show that retatrutide, a triple agonist of the GIP, GLP-1 and glucagon receptors, results in up to 82% reduction in liver fat.
After early initiation of anti-retroviral therapy in infants infected with HIV-1 in utero, some children sustain undetectable virus levels, despite treatment interruption, and which associated with distinct features of the transmitted virus.
Data from two large longitudinal cohorts in China, in which the participants were clustered into five groups based on their metabolic characteristics, show that a signature of microbiome age modulates the risk of cardiovascular disease in metabolically unhealthy individuals.
An economic evaluation of the E-MOTIVE intervention for postpartum hemorrhage (PPH) compared with usual care in 210,132 women, carried out from a healthcare system perspective, uncovered the cost per case of severe PPH prevented and cost per disability-adjusted life-year averted.
In a single-arm phase 2 trial evaluating intravesical delivery of the oncolytic adenovirus cretostimogene grenadenorepvec with systemic anti-PD-1 in patients with BCG-unresponsive non-muscle-invasive bladder cancer with carcinoma in situ, the complete response rate at 12 months was 57.1%, meeting the primary endpoint.
In the observational ZERO Childhood Cancer Precision Medicine Program PRecISion Medicine for Children with Cancer (PRISM) trial, children with high-risk cancer were treated with molecular tumor board-recommended therapies, resulting in overall clinical response rates that translated into survival benefit after long-term follow-up.
In a randomized, double-blind phase 3 trial, the anti-α4β7 integrin monoclonal antibody vedolizumab plus standard prophylaxis was superior to placebo plus prophylaxis for prevention of lower gastrointestinal acute graft-versus-host disease in patients following allogeneic hematopoietic stem cell transplantation.
Bilateral administration of human OTOF gene therapy appears safe, and preliminary data suggest that it could effectively treat a rare form of hereditary hearing loss by enhancing speech perception, sound source localization and overall auditory experience.
Atrial fibrillation is associated with an increased risk of thromboembolic events and vascular dementia in patients with no previous history of stroke or any conventional risk factors, compared to patients without atrial fibrillation.
An interim analysis of a single-arm trial in 5 children with hereditary deafness shows that binaural AAV gene therapy is safe and leads to hearing improvement up to 13–26 weeks of follow-up.
In a phase 2 trial of dazodalibep, a CD40 ligand, with a crossover stage in two distinct populations of patients with Sjögren’s disease, the compound was well tolerated and led to significantly improved disease activity.
The treaty is a grand social bargain of open sharing of crucial scientific data in real time and equitable allocation of lifesaving medical countermeasures.