In a phase 2 trial of patients with untreated, recurrent and progressive brain metastases treated with an anti-programmed cell death protein 1 inhibitor, the overall intracranial benefit rate was 42.1%, which met the prespecified primary endpoint.

Genomic and cell-free DNA sequencing clarify the clinical diagnosis and inform treatment initiation in a cohort of 356 patients with vascular anomalies.

The US Food and Drug Administration has reduced requirements for preclinical animal testing, leading to a surge of interest in organoids, tissue chips and in silico testing.

Artificial intelligence tools are beginning to upend the drug discovery pipeline, with several new compounds entering clinical trials.

Analysis of tumor samples from patients with nasopharyngeal carcinoma shows that gemcitabine plus cisplatin chemotherapy activates a unique population of innate-like B cells, which enhance the cytotoxic CD8⁺ T cell response and are associated with better clinical outcomes.

In patients with DNA damage response-deficient solid tumors, the oral ATR kinase inhibitor camonsertib was well tolerated; a recommended phase 2 dose was identified; and encouraging anti-tumor activity was observed, with the highest response rate observed in patients with ovarian cancer.

In a phase 2 trial of patients with untreated, recurrent and progressive brain metastases treated with an anti-programmed cell death protein 1 inhibitor, the overall intracranial benefit rate was 42.1%, which met the prespecified primary endpoint.

Genomic and cell-free DNA sequencing clarify the clinical diagnosis and inform treatment initiation in a cohort of 356 patients with vascular anomalies.

Analyses of large population-based cohorts and clinical trials show that using polygenic scores to account for variability in PSA levels improves detection of prostate cancer, suggesting an approach for enhancing screening accuracy.

A multi-ancestry genetic meta-analysis identifies 12 new loci associated with preeclampsia and gestational hypertension and proposes the integration of polygenic scores and clinical factors for disease prediction

Observational studies provide crucial information early during epidemics and pandemics, but they often suffer from methodological shortcomings, which can be resolved.

Nature Medicine explores the latest translational and clinical research news, with a delayed FDA decision on Sarepta’s adeno-associated virus gene therapy SRP-9001.

The US Food and Drug Administration has reduced requirements for preclinical animal testing, leading to a surge of interest in organoids, tissue chips and in silico testing.

Research software engineering is central to data-driven biomedical research, but its role is often undervalued and poorly understood.

Artificial intelligence tools are beginning to upend the drug discovery pipeline, with several new compounds entering clinical trials.