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A gene therapy product combines astrocyte replacement with stable delivery of a neuroprotective factor; a first-in-human study demonstrated safety and will inform the design of further clinical trials for this neurodegenerative disease.
In patients with large B cell lymphomas, immune features of the tumor microenviroment predict clinical outcomes after CAR T cell therapy; as the number of patients treated with CAR T cells is set to increase, refinement of these and other biomarkers will be crucial.
Incorporating genetic factors into risk models improves the prediction of severe obesity for survivors of childhood cancer, which could promote early interventions and better long-term care.
Infrequently dosed, longer-acting antiretroviral agents are making adherence to medication easier, leading to better outcomes for those living with HIV or at risk of infection.
A study evaluating serial injections of oncolytic virus therapy shows promising outcomes in patients with glioblastoma, and opens the door to longitudinal study designs with the potential to yield rich molecular insights.
In the phase 3 SPR1NT trial, pre-symptomatic gene therapy demonstrated impressive clinical outcomes in infants with a genetic diagnosis of spinal muscular atrophy (SMA); long-term safety follow-up of these patients must now be a key priority.
The concentrations of two key blood biomarkers for Alzheimer’s disease are affected by some medical conditions, which could potentially lead to misdiagnosis.
New technologies allow the noninvasive detection and staging of asymptomatic alcohol-associated liver disease; further refinement of this approach could transform clinical management and improve patient outcomes.
Biopsies from synovial joints — and the abundance of B cells and macrophages therein — may instruct more effective treatment decisions for individuals with rheumatoid arthritis.
Preventing atherosclerotic cardiovascular disease in adults will require innovative public health approaches focused on children and families, to improve risk factor trajectories across the lifespan and prioritize children at highest risk of future disease.
A phase 3 trial highlights the importance of problem-led design, multidisciplinary commitment and patient-reported outcomes — with skilled oncology nurses having a crucial role.
Electronic cigarettes might be superior to nicotine replacement therapy as a quitting aid for pregnant women — but enthusiasm is hampered by low rates of smoking cessation and unknown long-term harm.
The DAWN and SEED trials demonstrate the potential of glucokinase activators for the treatment of type 2 diabetes, but how they fit in the overall treatment algorithm remains to be determined.
Two new studies provide robust epidemiological evidence and a mechanistic link, with potential implications for strategies that target Epstein–Barr virus.
As the pandemic evolves, questions remain but field studies are increasingly difficult; if carefully designed, human challenge models could offer a practical and timely solution.
Antibiotic resistance genes evolve in the environment, in animals, and in humans; strategic action is needed on all three fronts to help understand resistance and to limit its spread.
Armored CAR T cells show early signs of clinical activity in patients with castration-resistant prostate cancer, paving the way for further development and optimization.
New technologies are expanding the reach and accessibility of preimplantation genetic testing of human embryos. But what these advances can deliver is still unclear, and a frank assessment of their profound ethical implications is urgently needed.
