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Integrative approaches continue to improve diagnostic accuracy for pediatric brain cancers, but much more is needed from researchers, governments and regulators if precision medicine with curative treatments are to become a reality.
The NEOSTAR trial is a key step on route to better outcomes; but the best approach is likely to be an individualized one, reflecting the many factors that influence treatment response.
Epidural spinal cord stimulation improved upper limb function in two participants after stroke, revealing a potentially exciting new tool for stroke recovery.
The widely used artificial sweetener erythritol affects platelet function and is associated with major cardiovascular events — fuelling calls for a re-evaluation of the use and regulation of non-nutritive sweeteners.
High-resolution meta-omics have enabled the discovery of the microbial enzymes that inactivate an ulcerative colitis drug and predict subsequent treatment failure, an approach that could enable more personalized treatment of inflammatory bowel disease.
Pre-symptomatic gene editing in preclinical models of hypertrophic cardiomyopathy shows therapeutic promise; clinical studies are now needed to assess safety and efficacy in humans.
A study prospectively evaluating a stratified approach to selecting treatment heralds a new era of precision medicine for type 2 diabetes, which should incorporate ongoing discovery, social determinants of health and healthcare transformation.
In a phase I trial, the combination of dabrafenib, trametinib and spartalizumab uncovers the crosstalk between the MAPK pathway and the immune system, which offers a promising new strategy to improve clinical outcomes.
As another clinical trial of a mitochondria-targeting cancer therapy faces failure, it calls for a thorough re-evaluation of the strategy; the time has come to go from the bedside back to the bench.
A clinical trial shows the feasibility of a noninvasive imaging approach to diagnose a surgically amenable subtype of primary aldosteronism — a common and often poorly managed form of hypertension.
A phase 1 trial using an allogeneic stem-cell-based therapy in people with progressive multiple sclerosis (MS) shows the feasibility and tolerability of the approach; rigorous evaluation of this and other regenerative strategies for MS is now urgently needed.
Automated insulin delivery systems that are already used in type 1 diabetes show promise for patients with type 2 diabetes, and are poised to have a substantial effect on public health.
Prospective, longitudinal clinical studies incorporating high-throughput, single-cell analyses could identify which bacterial antigens to include in TB vaccines — and which to avoid.
A variant of the IL7 gene predicts the toxicity of checkpoint inhibitors in patients with cancer, via a mechanism shared with autoimmune diseases — which could inform biomarker and treatment strategies in both of these contexts.
Osteoarthritis is highly heterogeneous, so effective therapies will need to target clearly defined molecular endotypes, restore mechanical joint function and reduce pain; thus, a ‘one-size-fits-all’ approach is unlikely to succeed.
Circulating tumor DNA (ctDNA) informs predictive biomarkers in non–small-cell lung cancer, but the presence of ctDNA itself could also be a prognostic indicator.
In a clinical trial, non-nutritive sweeteners — which are supposedly inert — were shown to disrupt the gut microbiome of healthy people and impair glucose tolerance.
