Volume 30 Issue 7, July 2024

FDA approval of Iovance’s lifileucel for melanoma shows the promise of tumor-infiltrating lymphocytes for the treatment of solid tumors, with trials ongoing for lung cancer, cervical cancer and more.

Simulations in virtual and augmented reality can help patients build confidence and self-esteem, supported by a real-life or virtual therapist.

Fortunate Machingura heads CeSHHAR Zimbabwe’s Climate, Environment, and Health Department and is also a lecturer at the Liverpool School of Tropical Medicine. She describes her pivot from HIV research to another crucial challenge facing Africa: climate change.

Juliane Fonseca de Oliveira is a mathematician at CIDACS in Salvador, Bahia, Brazil. She describes her part in building an epidemiological model to analyze the diverse populations within the 100 Million Brazilians Cohort.

Shivani Misra describes how meeting a misdiagnosed patient early in her career led her to study precision diabetes in underrepresented groups.

Precision health in low- and middle-income countries should focus on disease prevention and implementation in order to be cost-effective and impactful.

For precision genetic medicines to fulfill their potential as treatments for ultra-rare diseases, fresh approaches to academic–industry partnerships and data sharing are needed, together with regulatory change and adaptation of reimbursement models.

A recent workshop brought together global leaders in diabetes to assess existing approaches to disease heterogeneity and to identify research gaps, with a goal of achieving precision diabetology for all patients globally.

The prospect of genomic screening in newborns attracts both hype and criticism, but carefully designed, large-scale, prospective research studies are the only way to generate the data needed for its rigorous evaluation.

Data from a large randomized trial show that a personalized diet can improve cardiometabolic health, providing support for a ‘food as medicine’ concept that, although centuries old, still lacks robust evidence.

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.

Accumulating data support the systemic benefits of semaglutide (and potentially other GLP-1RA-based therapies) in people with obesity, meaning that cardiologists and other clinicians must become familiar with prescribing them — particularly once market competition makes these drugs more accessible.

As diabetes technology evolves, implementation of equitable, sustainable, remote patient monitoring will ensure that this technology is accessible to everyone with type 1 diabetes — but strategies to address the burden of data collection and review are crucial.

Circuitry-based neuroimaging analyses can enhance our understanding of abnormal brain functioning in patients with major depressive disorder and other psychiatric conditions — but their utility for guiding treatment selection is less certain.

To correct the market failure around pediatric cell and gene therapies, the authors propose a new model to lead late-stage development and commercialize these therapies outside traditional routes.

Electronic health records hold immense potential for providing clinically useful insights for populations and individuals; this Review summarizes the opportunities and challenges, with an emphasis on recognizing and reducing bias.

Genomic capacity building is crucial to addressing the unique healthcare challenges on the African continent; this Review delves into the challenges and opportunities, with an emphasis on infrastructure, workforce and policymaking.

Human genetics, pathogen genomics, social and environmental data and AI are transforming public health—enabling more-precise interventions that account for heterogeneity in and across populations.

The BePRECISE (Better Precision-data Reporting of Evidence from Clinical Intervention Studies & Epidemiology) consortium, comprising 23 experts in precision medicine, cardiometabolic diseases, statistics, editorial and lived experience, provides a checklist tool and recommendations for reporting precision medicine research.

In a phase 1 trial in a single patient with hereditary spastic paraplegia type 50, a personalized AAV9-based gene therapy was developed within 3 years from diagnosis and was well tolerated, showing preliminary evidence of disease stabilization.

A randomized controlled trial showed that following a personalized dietary program led to significant improvements in cardiometabolic and gut health as well as reductions in body weight compared to following standard dietary advice according to US Department of Agriculture guidelines.

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 cohort of 281 children with diagnosed or suspected cancer presenting to the NHS, implementing routine whole-genome sequencing provided clinical benefit in 29% of cases and led to change in management in 7% of patients.

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.

Results from this single-arm cohort of the BFAST trial showed that the clinical efficacy of entrectinib in patients with ROS1-positive NSCLC, selected using liquid biopsies, is consistent with that seen in previous reports where patients were selected using tissue-based testing methods.

Exploratory analyses of the DESTINY-Gastric01 trial show that baseline HER2-associated biomarkers in circulating tumor DNA or tissue samples were associated with therapeutic response in patients with HER2-positive tumors, and these analyses identify potential drivers of resistance.

In the COMPASSION-04 trial, first-line treatment with a bispecific antibody targeting CTLA-4 and PD-1 and chemotherapy in patients with locally advanced or metastatic gastric or gastroesophageal junction adenocarcinoma resulted in clinical responses with a manageable safety profile.

A deep learning model is used to classify central nervous system tumors based on their DNA methylation profile directly from histopathology, and showed high accuracy in a large set of external validation cohorts, potentially informing downstream treatment.

A multimodal deep learning prognostic model based on histopathology outperforms current gold standards for identifying patients with endometrial cancer with different outcomes, in multiple external validation cohorts.

Findings from a multicenter study of 197,080 singleton live births in China show maternal hypertension as a mediator of the risks imposed by heat exposure between conception and 20 weeks of gestation on preterm birth and its various clinical subtypes.

By leveraging paired microbiome and human gene expression data from pediatric patients who underwent hematopoietic cell transplantation, distinct lung–immune system–microorganism interactions were identified as important drivers of fatal lung injury.

Data from pediatric populations with neurodevelopmental disorders, obtained through a combinatorial strategy of literature review scoping and in-patient appointments, were used to construct a Phenomics Aggregation Database (PhenomAD-NDD) that can aid clinical diagnosis of comorbidities.

A phase 2 dose-finding study of an extended-release tablet formulation of ketamine in patients with treatment-resistant depression shows that this formulation overcomes many of the limitations associated with the use of intravenous or intranasal ketamine formulations.

Enabled by augmented muscle afferents, a bionic leg under continuous neural control restores biomimetic adaptations to various walking speeds, terrains and perturbations.

In a post-approval study including more than 17,000 patients on the safety of pulsed field ablation, a new method for treatment of atrial fibrillation, the procedure was found to have a low rate of adverse events but was associated with some unexpected rare complications that will need further study.

In a substudy of the RATE-AF trial, which compared heart rate control therapy using digoxin or the beta-blocker bisoprolol, heart rate and physical activity data collected using a wearable device showed equivalent heart rate control by the two drugs and could be used to predict future heart failure functional class as well as standard clinical measurements.

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.

A prespecified analysis of the SELECT trial revealed that patients assigned to once-weekly subcutaneous semaglutide 2.4 mg lost significantly more weight than those receiving placebo and showed improvements in various anthropometric indices.

In a pre-specified secondary analysis of the SELECT trial, once-weekly subcutaneous semaglutide 2.4 mg in patients with obesity was associated with a 22% reduction in the main 5-component kidney composite endpoint compared to patients on placebo.

In a prospective study, a team-based approach combining continuous glucose monitoring with a technology-assisted remote patient monitoring program improved glycemia in a diverse cohort of children, adolescents and young adults with newly diagnosed type 1 diabetes.

Personalized brain circuit measures quantified using a new imaging technology in 801 patients with depression and anxiety identify six biotypes with unique symptoms, behaviors and responses to different types of treatment.