Volume 30 Issue 4, April 2024

Nature Medicine explores the latest translational and clinical research news, with FDA approval of an AI-assisted optical reader to help in the diagnosis of skin cancer.

Growing evidence shows that dietary interventions can be effective at treating or delaying some diseases, but further trials are needed for wider adoption.

Diversity in clinical trials must be accompanied by justice and equity, including benefits for underrepresented participants, in order to boost population health.

Community screening could be used for the monitoring, early detection and prevention of for depression, and to maximize the effects of the policies to reduce its burden.

Steatotic liver disease is part of a revised nomenclature to replace the term fatty liver disease, but this should also drive forward innovation in research, diagnostics and treatments.

Women’s health can be improved by increasing the representation of women in leadership roles in science, medicine and public health.

A small, prospective clinical study shows that ex vivo drug screening of pediatric cancer samples can identify effective therapeutic options. If validated, these findings could herald a new approach to precision medicine in this setting.

Evaluation of a clinical summarization method based on GPT-4 suggests that such models might reduce the documentation burden on clinicians — but prospective evaluation with high-priority tasks will be the true test of its potential.

AI models for tasks such as pathology and dermatology struggle to generalize to new patient groups or hospitals that they were not trained on; learning more robust features from unlabeled data could prevent overfitting to the training distribution and thereby increase fairness.

Telehealth provision of medication abortion is safe and effective, but ensuring equitable access is challenging in the USA — and further compounded by an upcoming Supreme Court case.

Chronic pain is common, with more than one in five adult Americans reporting having pain daily or on most days. A multi-ancestry genomic analysis in 598,339 military veterans in the USA identifies 126 genetic variants associated with pain intensity, highlights shared genetic risk with substance use and psychiatric disorders, and reveals enrichment in GABAergic neurons as a key molecular contributor to experiencing pain.

Most suspected cholera cases are not tested for Vibrio cholerae. Integrating systematic testing into cholera surveillance systems, even with imperfect rapid diagnostic tests, could yield large gains in efficiency and cost savings in the geographic targeting of mass oral cholera vaccination campaigns.

The implementation of PCR tests of pooled saliva samples for universal screening of congenital cytomegalovirus infection was assessed in 15,805 neonates over 13 months. This extensive analysis revealed the high feasibility and empirical efficiency of the pooled testing approach, which had a clinically insignificant loss of sensitivity.

In a difference-in-differences analysis among Medicare beneficiaries in the USA, billion-dollar weather disasters were associated with higher rates of emergency department visits and deaths in the weeks after the disaster. Observed changes were more pronounced among counties that experienced the most loss and damage compared to all affected counties.

Clinical disease trajectories that describe neuropsychiatric symptoms were identified using natural language processing for 3,042 brain donors diagnosed with various neurodegenerative disorders. Trajectories revealed distinct temporal patterns that result in the identification of new clinical subtypes, and a subset of misdiagnosed donors.

Causal machine learning methods could be used to predict treatment outcomes for subgroups and even individual patients; this Perspective outlines the potential benefits and limitations of the approach, offering practical guidance for appropriate clinical use.

Recent developments in bioengineering and organic chemistry have enabled targeting of the previously ‘undruggable’ KRAS; this review summarizes the successes, challenges and future of KRAS therapeutics in the clinic.

Profiling of a case of secondary T cell lymphoma following anti-CD19 CAR T cell therapy suggests that it was not caused by CAR insertional mutagenesis, with single-center analysis indicating that secondary T cell lymphoma risk after commercial CAR T cell treatment is low.

In an observational study evaluating functional precision medicine in children and adolescents with relapsed or refractory solid and hematologic malignancies, it was feasible to provide personalized treatment recommendations to treating physicians on the basis of genomic profiling and ex vivo drug sensitivity testing within 4 weeks.

In a large trial of patients with recurrent high-grade gliomas, IL-13Rα2-targeting CAR-T cells were feasible to manufacture and well tolerated when delivered via intratumoral and/or intraventricular routes.

In an interim analysis of a phase 1/2 trial, a heterologous prime boost vaccine comprised of a chimpanzee adenovirus and self-amplifying mRNA that encodes neoantigens derived from common oncogenic driver mutations in combination with immune checkpoint blockade was safe and elicited neoantigen-specific T cell responses in patients with advanced solid tumors.

In a phase Ib trial, neoadjuvant nivolumab or nivolumab/relatlimab prior to chemoradiotherapy were well tolerated and liquid biopsy analyses show that undetectable ctDNA was associated with longer survival.

In a randomized phase 2 trial comparing anti-PD-1 plus anti-VEGF plus an HDAC inhibitor versus anti-PD-1 plus an HDAC inhibitor in patients with MSS/pMMR colorectal cancer, the triplet combination led to significantly better 18-week progression-free survival.

Treatment of patients with advanced hepatocellular carcinoma with a personalized DNA vaccine in combination with anti-PD-1 therapy was safe and led to encouraging clinical efficacy, with immunological analyses confirming the induction of tumor antigen-specific T cell responses.

Implementation of organized low-dose computed tomography screening in over 4,000 individuals with high risk for lung cancer as part of the Ontario Lung Cancer Screening Pilot reported high cancer detection rates, early detection of cancer and low serious harms.

A multi-ancestry analysis in large cohorts of patients with type 2 diabetes (T2D) identified 12 disease genetic clusters with distinct cardiometabolic trait associations, including two lipodystrophy-related clusters that could provide insights on the increased T2D susceptibility in East Asian ancestries at a given body mass index level.

A cross-ancestry meta-analysis identified 126 genetic loci associated with chronic pain intensity, revealing correlations with substance use and psychiatric traits.

The performance of plasma %p-tau217 is clinically equivalent in classification of Aβ PET status and superior in classification of tau PET status compared to clinically used and FDA-approved CSF tests in cognitively impaired participants.

An exploratory analysis of the 1-year clinical trial PASADENA in individuals with early-stage Parkinson’s disease suggests that prasinezumab might reduce motor signs progression to a greater extent in those with more rapidly progressing disease.

A modeling study of 35 cholera-affected countries in Africa showed that introducing cholera surveillance could improve the effectiveness and cost-effectiveness of preventive cholera vaccination campaigns.

Building on a pre-existing pooling pipeline for the efficient detection of SARS-CoV-2, the results of a large-scale implementation study show the feasibility and benefits of saliva sample pooling to enhance neonatal screening for congenital cytomegalovirus.

Analysis of 42 severe weather disasters (floods, storms and cyclones) in the United States between 2011 and 2016 reported associations with increased emergency department utilization and mortality in affected counties for up to 6 weeks.

The PRODIGITAL-D trial in adults aged 60+ years from socioeconomically deprived areas of Brazil showed that a 6-week self-help mobile messaging psychosocial intervention was effective in improving depression recovery at 3 months compared to a single message control intervention.

Comparative performance assessment of large language models identified ChatGPT-4 as the best-adapted model across a diverse set of clinical text summarization tasks, and it outperformed 10 medical experts in a reader study.

Analyzing clinical records from a cohort of 3,042 donors in the Netherlands Brain Bank, natural language processing models unveil the symptomatology and potential misdiagnoses of a broad range of neurodegenerative disorders.

By learning to pair dermatological images and related concepts in a self-supervised manner, a visual-language foundation model is shown to have comparable performance to supervised models for concept annotation and is used to scrutinize model decisions for enhanced interpretability and accountability of medical imaging applications.

By generating synthetic image samples specific to underrepresented groups, diffusion models help medical image classifiers to achieve greater fairness metrics across a variety of medical disciplines and demographic attributes.

In a series of clinically relevant tasks in computational pathology, AI-driven models display marked performance disparities across demographic groups, which can only partially be mitigated by self-supervision on large training datasets and existing debiasing techniques.

In a prospective study across US states where abortion remains legal, telehealth medication abortion, provided primarily without tests, was effective, safe and comparable to previous findings from large US studies investigating in-person medication abortion care.

In an updated systematic review and meta-analysis, fixed-dose combination therapy using a polypill with at least one blood pressure-lowering drug and one lipid-lowering drug was found to reduce both incident cardiovascular disease and all-cause mortality.