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First-in-human results from five cohorts of healthy volunteers and individuals with hepatic steatosis show that RNA interference treatment targeting ANGPTL3 was well tolerated and led to reduction in triglycerides and non-HDL cholesterol.
Treatment of patients with metastatic salivary gland cancer with anti-PD-1 and anti-CTLA-4 led to encouraging clinical benefit in certain histologic subtypes, with translational analyses showing pre-existing T cell clonal expansion in responding tumors.
Analysis of data from the US Department of Veterans Affairs showed that 2 years after severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection, risk for most postacute sequelae remained elevated in people who were hospitalized with COVID-19 but was attenuated in nonhospitalized individuals.
Using extracted images and related labels from pathology-related tweets, a model is trained to associate tissue images and text and approaches state-of-the-art performance in clinically relevant tasks, such as tissue classification.
Patients with stage 4 or unresectable stage 3 melanoma refractory to first-line anti-programmed death protein 1 (PD-1) or anti-programmed cell death 1 ligand 1 have longer progression-free survival when treated with a combination of anti-cytotoxic T-lymphocyte protein 4 (CTLA-4) and anti-PD-1 versus anti-CTLA-4 alone.
This is a post hoc 18-month follow-up analysis of the CRB-401 trial, testing idecabtagene vicleucel (ide-cel, bb2121) in relapsed and refractory multiple myeloma, and reports sustained safety and clinical efficacy, which correlates with T cell phenotypes.
In a pivotal phase 2 trial, elranatamab, a bispecific antibody targeting B-cell maturation antigen (BCMA) and CD3, had a confirmed objective response rate of 61% in patients with relapsed or refractory multiple myeloma who had not previously received BCMA-directed therapy.
A phase I trial of cerebellar deep brain stimulation to enhance chronic post-stroke motor rehabilitation supports the safety and feasibility of the approach while providing encouraging evidence of motor improvements.
In a preclinical study, the delivery of an AAV-based gene therapy encoding GDNF in the brain prevented the return to alcohol use behaviors in a non-human primate model.
Treatment with anti-PDL1 and anti-CTLA4 in combination with mFOLFOX6 chemotherapy for patients with treatment-naive microsatellite stable metastatic colorectal cancer is safe, shows encouraging progression-free survival and induces a tumor-specific immune response.
Machine learning models showed that social disparities, cardiometabolic disease and mental health were the main predictors of aging in Latin American populations, with these factors being more pronounced in low- and middle-income compared to high-income Latin American countries.
In a case series of six patients with refractory lupus nephritis who were treated off-label with an anti-CD38 monoclonal antibody, five patients achieved complete or partial renal responses by 12 months of follow-up.
Proteomic analysis of cerebrospinal fluid from individuals with autosomal dominant Alzheimer’s disease reveals how this complex and chronic disease evolves over many decades.
A newly developed hemodynamic parameter, termed tissue perfusion pressure, can be determined from standard blood pressure monitors and has additive value compared to mean arterial pressure for predicting the risk of mortality and other outcomes for patients in the cardiac intensive care unit.
A machine-learning classifier predicts the origin of cancer of unknown primary based on electronic health records and next-generation sequencing data, showing that patients treated accordingly to model predictions had significantly better outcomes.
Based on a newly developed deep-learning model for predicting the binding affinities of protein–protein interactions, the effects of SARS-CoV-2 spike protein variants on binding to the ACE2 receptor and neutralizing antibodies were analyzed and used to predict immune escape and viral evolution.
In the randomized, double-blind, phase 3 GLOW trial, capecitabine and oxaliplatin combined with zolbetuximab, a monoclonal antibody that targets CLDN18.2, significantly improved progression-free survival and overall survival in patients with CLDN18.2-positive, HER2-negative, untreated, locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma.
T cells containing an anti-mesothelin single-domain antibody fused to a component of the endogenous T cell receptor signaling complex exhibit notable toxicities but encouraging clinical responses in patients with treatment-refractory mesothelioma and ovarian cancer.
Trastuzumab deruxtecan, an anti-HER2–drug conjugate, exhibits the highest objective response rate in patients with HER2-overexpressing metastatic breast cancer, but clinical activity is also observed in patients with HER2-low or non-expressing tumors, potentially pointing to additional determinants of drug efficacy.
Treatment of patients with HER2-mutant non-small-cell lung cancer with the pan-HER inhibitor pyrotinib in a phase 2 trial or compassionate use showed a favorable objective response rate, whereas patients treated in parallel by physician’s choice in a real-world study did not.