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The bispecific molecule tebotelimab, which blocks both PD-1 and LAG-3, is well tolerated as a monotherapy and in combination with the anti-HER-2 antibody margetuximab and elicits encouraging clinical activity in solid tumors with high LAG-3 levels and/or expression of IFN-γ-regulated genes.
In the phase 3 MAESTRO-NAFLD-1 trial, the liver-targeted thyroid hormone receptor-β selective agonist resmetirom was well tolerated and improved liver biomarkers in adults with nonalcoholic fatty liver disease.
In this pre-specified interim analysis, patients with resected stage IIB/C melanoma who received adjuvant nivolumab had significantly prolonged recurrence-free survival compared to placebo-treated patients, providing another treatment option for this population.
In the SURMOUNT-3 trial, once-weekly treatment with tirzepatide was demonstrated to result in clinically meaningful additional weight loss in adults with overweight or obesity following initial successful weight loss of at least 5% body weight with intensive lifestyle intervention.
In the first stage of the BR.36 adaptive trial in patients with non-small cell lung cancer receiving anti-PD1 immunnotherapy, the primary endpoint of concordance between circulating tumor DNA (ctDNA) molecular response and RECIST response was met. The results will inform the second, ctDNA-directed stage.
In the phase 3 STAR trial, subretinal administration of the AAV2-based gene therapy timrepigene emparvovec in patients with choroideremia did not lead to a significant difference in the primary endpoint of 15-letter Early Treatment Diabetic Retinopathy Study (ETDRS) improvement in visual acuity from baseline compared to untreated controls.
Microglia mediate the early and selective degeneration of corticostriatal synapses and the development of cognitive deficits in presymptomatic Huntington’s disease via the complement cascade.
Results from the DiViD Intervention, a phase 2 randomized, placebo-controlled trial, showed that antiviral treatment with pleconaril and ribavirin for 6 months resulted in higher endogenous insulin production in children and adolescents with new-onset type 1 diabetes.
In the first-in-human trial of elranatamab, patients with refractory or relapsed multiple myeloma who received the bispecific antibody against BCMA and CD3 experienced no dose-limiting toxicities during dose escalation, and the agent showed promising clinical efficacy.
In a phase 2 trial, the combination of gemcitabine, cisplatin and anti-PD-1 led to a clinical complete response in 43% of patients with muscle-invasive bladder cancer, which facilitated bladder sparing and was associated with long-term bladder-intact metastasis-free survival.
Reactivation of latent SIV with an IAP antagonist, coupled with immunotherapeutic approaches, reduced replication-competent SIV in lymph node CD4+ T cells in rhesus macaques. Further exploration targeting these pathways to reduce the HIV viral reservoir is warranted.
New treatments are essential for methicillin-susceptible Staphylococcusaureus bacteremia, but progress is slow. In this phase III–IV trial, cloxacillin plus fosfomycin failed to show superiority over cloxacillin alone, underscoring the challenges to improving patient outcomes.
In a prospective study, an autism screening app administered using a tablet computer to young children showed high diagnostic accuracy and may facilitate a scalable approach to autism screening.
In the LUMINA-1 trial for fibrodysplasia ossificans progressiva, garetosmab, an activin A monoclonal antibody, did not lead to significant changes in heterotopic ossification lesion activity in pre-existing lesions in period 1. Garetosmab prevented the formation of new lesions in both periods 1 and 2.
Analysis using a combination of molecular and genetic epidemiological approaches reveals an interaction between female sex and the genetic variant PNPLA3 p.I148M that might explain why some women have increased susceptibility to fatty liver disease after onset of menopause.
In patients with Crohn’s disease, CD4+ T cells with cytotoxic TH1 cell-like effector functions reactive against dietary and commensal yeasts are increased in blood and inflamed tissue compared with patients with ulcerative colitis and healthy controls.
A model investigating 26 possible malaria treatment policy intervention scenarios showed that introducing multiple first-line antimalarial therapies is the most effective single policy change in slowing the spread of artemisinin-resistant pfkelch R561H mutations in Rwanda over 5 years.
In this compassionate use study, treatment of adult patients with H3K27M-mutant diffuse midline glioma with a long peptide vaccine targeting H3K27M led to vaccine-induced peripheral T cell immune responses and encouraging clinical efficacy in the majority of patients, including a durable complete response.
A low rate of Clostridioides difficile transmission within an intensive care unit supports the effectiveness of prevention practices and highlights the greater risk to carriers of toxigenic C. difficile.
Results from the phase 3 placebo-controlled MAPP2 trial show that MDMA-assisted therapy reduces post-traumatic stress disorder (PTSD) symptoms and functional impairment in a diverse population with moderate to severe PTSD.