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Early clinical data on in vivo CAR-T cell therapy for multiple myeloma reveals key mechanistic insights and could mark the beginning of a new era of fast, streamlined and accessible immunotherapies.
In a phase 1 trial, the in vivo generation of anti-BCMA CAR-T cells by lentiviral delivery was feasible and did not lead to dose-limiting toxicities in five patients with relapsed or refractory multiple myeloma.
Results of the phase 2 POLAR trial show that biomarker-guided treatment in patients with metastatic pancreatic cancer based on homologous repair deficiency leads to encouraging clinical response rates in immune cell-infiltrated tumors.
The small molecule rezatapopt binds a unique pocket in Y220C-mutant p53 and restores its tumor-suppressor function; a phase 1 study provides proof of concept of this therapeutic approach.
The first-in-human clinical trial of the LRRK2-targeting antisense oligonucleotide BIIB094 in Parkinson’s disease demonstrates that the treatment is well tolerated and produces dose-dependent reductions in cerebrospinal fluid levels of LRRK2 and phosphorylated Rab10, indicating successful target engagement.
In a large UK-based primary care randomized trial, immediate artificial intelligence (AI)-driven prioritization of chest X-rays did not significantly shorten time to computed tomography or lung cancer diagnosis versus standard workflow, suggesting that AI-based prioritization alone is unlikely to accelerate the lung cancer diagnostic pathway.
The transmission dynamics of the 2023–2024 Oropouche virus (OROV) outbreak in Brazil (Manaus City) revealed a twofold increase in Oropouche seroprevalence in Manaus, and in a separate historical reconstruction, the authors estimate that there were approximately 9.4 million OROV infections during major outbreaks across Latin America and the Caribbean.
Trained and validated on data from 2,344 patients with smoldering multiple myeloma, a new algorithm using longitudinal biomarker dynamics provides accurate prediction of risk of disease progression, outperforming established models.
Analyses of more than 10,000 participants from the Human Phenotype Project revealed distinct, persistent food–microbiome associations that can be used to simulate personalized dietary interventions to improve cardiometabolic health.
Over five years, implementation of the NHS England Lung Cancer Screening Programme achieved high early-stage detection rates and demonstrated that the programme is both feasible and scalable for reaching high-risk and underserved populations.
Coral et al. outline five fundamental tenets to promote clinically meaningful, reproducible, scalable and equitable health outcomes through precision medicine.
A deep learning model using smartwatch data was shown to predict peak oxygen uptake and unplanned healthcare events in the TRUE-HF prospective cohort of patients with heart failure, as well as unplanned healthcare utilization in patients with heart failure in the All of Us Research Program.
In a real-world cohort of 888 patients with solid cancers receiving whole-genome sequencing profiling, actionable biomarkers were identified in 73% of patients, and targeted therapies were associated with clinical benefit in patients without prior systemic therapy.
Increased blood levels of phosphorylated tau (p-tau) are a diagnostic tool in Alzheimer’s disease. In a multicenter, cross-sectional study, we show that serum p-tau could also serve as a biomarker for the most common types of systemic amyloidosis, including the potential differentiation between amyloidosis-related polyneuropathy and polyneuropathy due to other causes.
The health benefits of clean energy transitions are unevenly distributed, even when emissions targets are met. A health-centered global governance framework is urgently needed to ensure that health justice is embedded in climate policy.
A trial emulation study using electronic health record data from 174,678 patients with type 1 diabetes revealed that GLP-1RA initiation is associated with reduced risk of major cardiovascular events and end-stage kidney disease.
