Volume 27 Issue 10, October 2021

During the COVID-19 pandemic, the public have turned to scientists and doctors for vital information. But science communication is a skill in itself, so we asked the experts to share their tips.

Protein structures predicted using artificial intelligence will aid medical research, but the greatest benefit will come if clinical data can be similarly used to better understand human disease.

Psychedelics have shown great promise in treating mental-health conditions, but their use is severely limited by legal obstacles, which could be overcome.

New strategies based on nucleic acid technologies are being exploited to treat chronic hepatitis B—a pilot clinical study of antisense oligonucleotide treatment shows the potential promise of this approach.

Cytokine blockade can improve the survival rate of patients with COVID-19 who are at risk of respiratory failure, but timing and patient selection are key.

A new spatial analysis calls for a tiered and geographically targeted approach to tackling the vast, ongoing burden of anemia in women within low- and middle-income countries.

A phase 1 trial shows the feasibility of a novel approach combining a powered hip exoskeleton with a passive leg prosthesis to improve the energy efficiency of walking.

In this Perspective, the authors discuss the ethical challenges of individualized therapy (also called n-of-1) trials and argue that, although customized for the patient, these constitute ‘research’ nonetheless.

In an observational cohort of pregnant women in Israel, the BNT162b2 COVID-19 vaccine was found to have effectiveness similar to that seen in the general population.

This case report describes a biomarker-driven closed-loop therapy for depression using implanted electrodes to continually sense brain activity and automatically trigger direct brain electrical stimulation in high-depression states.

Biodistribution analysis of two patients with spinal muscular atrophy shows widespread onasemnogene abeparvovec DNA, mRNA and SMN protein throughout the central nervous system and peripheral organs following intravenous gene therapy administration.

A subdermal implant of the HIV-1 antiretroviral islatravir delivers sustained drug release over 12 weeks in humans.

A single dose of a broadly neutralizing, HIV-specific antibody transiently reduces viral load in plasma, and in some individuals is associated with durable virus suppression in the absence of antiretroviral therapy.

A first-in-human study of an antisense oligonucleotide targeting hepatitis B virus (HBV) RNA provides initial insights into this potential new therapeutic modality for individuals with chronic HBV infection.

Federated learning, a method for training artificial intelligence algorithms that protects data privacy, was used to predict future oxygen requirements of symptomatic patients with COVID-19 using data from 20 different institutes across the globe.

In a large prospective phase 4 trial, vaccination with CoronaVac, an inactivated SARS-CoV-2 vaccine, elicited significantly lower virus-specific IgG antibodies and neutralizing antibodies in patients with autoimmune rheumatic diseases than in age- and sex-matched healthy control trial participants.

The SAVE-MORE phase 3 study demonstrates the efficacy of anakinra, an IL-1α/β inhibitor, in patients with COVID-19 and high serum levels of soluble plasminogen activator receptor.

Geospatial estimates of the prevalence of anemia in women of reproductive age across 82 low-income and middle-income countries reveals considerable heterogeneity and inequality at national and subnational levels, with few countries on track to meet the WHO Global Nutrition Targets by 2030.

In individuals with above-knee amputation, a powered hip exoskeleton, which reduces the metabolic cost of walking by adding energy at the hip joint, greatly improves walking economy and has the potential to improve mobility.

Intralesional injection of an oncolytic virus prior to surgery shows favorable clinical activity over surgery alone in patients with locally advanced, resectable melanoma, supporting further evaluation in the neoadjuvant setting for this population.

Bicistronic CAR T cells targeting CD19 and CD22 exhibit clinical activity and low toxicity in pediatric and young adult patients with B cell acute lymphoblastic leukemia, with relapses associated with limited CAR T cell persistence.

This analysis of a large, clinically annotated cohort of individuals with predisposition to myelodysplastic syndromes reveals insights into the genetic determinants of disease progression and their relationship with clinical manifestations and therapy outcome.

In a randomized phase 2 clinical trial, the angiotensin receptor blocker valsartan improved cardiac structure and function in patients with early-stage hypertrophic cardiomyopathy, a condition for which there are no effective therapies for modifying disease progression.

In a phase 2b study, inhibition of hepatic stearoyl-CoA desaturase in patients with nonalcoholic steatohepatitis was safe and tolerated; although reductions in liver fat were not significant, changes in liver enzymes and histology were observed.

Two phase 2a trials demonstrate the efficacy of a new ACC inhibitor (PF 05221304) for reducing liver fat in patients with NAFLD, with co-administration of a DGAT2 inhibitor (PF-06865571) mitigating ACC inhibitor-mediated increases in serum triglycerides.