PDGFRA is commonly altered in pediatric HGG. Mayr et al. report that the PDGFRA/KIT inhibitor avapritinib demonstrates on-target activity, brain penetrance, and survival benefit in pre-clinical H3K27M...

PURPOSEUKALL 2011 randomly assigned children and young adults (younger than 25 years) with ALL or lymphoblastic lymphoma. The aims were to reduce induction toxicity (randomization 1 [R1]), CNS relapse...

This decision analytical model study estimates the lifetime risks of 8 treatment-related cancers and cardiovascular conditions among childhood cancer survivors and compares them with the general popul...

Point-of-care manufactured CAR T-cell therapy demonstrates a higher complete remission rate and presents a promising therapeutic option for low-middle-income countries like India. The total cost of CA...

Key PointsALK inhibitor or brentuximab monotherapy achieve excellent responses in pediatric patients with R/R ALK-positive ALCL.Optimal targeted therapy, d

Acute lymphoblastic leukemia (ALL) is the most common childhood cancer. The translocation t(1;19), encoding the TCF3-PBX1 fusion, is associated with intermediate risk and central nervous system (CNS)...

Key PointsOur study discovered new regulators of ruxolitinib response in Ph-like ALL, including c-MYC as a therapeutically targetable codependency.A progno

As proof of principle, an analysis using a suite of human-aligned immunocompetent mouse models of hepatocellular carcinoma identifies a promising therapeutic candidate, cladribine, which acts in ...

Whole-gene sequencing of microdissected gastric glands from individuals with and without gastric cancer reveals distinct patterns of somatic mutations and provides insights into influen...

Genome sequencing results and single-cell transcriptomics continue to produce findings that challenge the idea that cancer is purely a ‘genetic disease’. This Essay delves into cancer omics data that ...

A patient with newly diagnosed glioblastoma was safely treated with neoadjuvant nivolumab, relatlimab and ipilimumab before maximal resection, with comprehensive immune profiling showing the induction...

Functional, tumour-promoting GABAergic neuron-to-glioma synapses in diffuse midline gliomas are identified.

PURPOSEUrinary catecholamine metabolites are well-known biomarkers for the diagnosis (Dx) of neuroblastoma, but their clinical significance in determining therapy response during treatment is not well...

Ten percent of children with cancer harbour a mutation in a predisposition gene. In children with the kidney cancer, Wilms tumour, the prevalence is as high as 30%. Certain predispositions are associa...

Ongoing chromothripsis shapes genomic evolution in a diverse range of sarcomas and carcinomas. High-grade osteosarcoma is predominantly driven by the mechanism of loss-translocation-amplification chro...

Key PointsBlast-specific ex vivo venetoclax sensitivity correlated strongly with treatment responses and predicted longer survival.VenEx trial showed that

In a case series of five children with treatment-refractory neuroblastoma, it was feasible to manufacture and administer donor-derived GD2-specific CAR T cells and clinical responses were seen in four...

In a phase 1 trial, pediatric or adult patients with T-ALL who received CD5-targeted CAR-T cell therapy using cells from previous or newly matched donors showed an encouraging clinical response rate, ...

Key PointsAmong NCI high-risk patients, 93% with ETV6::RUNX1 and 54% with hyperdiploid B-ALL had excellent outcomes with low-intensity therapy.NCI high-ris

In the final report of a phase 1 trial evaluating intracerebroventricular B7-H3-targeting CAR T cells in children and young adults with diffuse intrinsic pontine glioma, repeated intracranial infusion...

Edited by Associate Editor Hervé Dombret, this How I Treat series highlights the clinical approach to high-risk subgroups of acute lymphoblastic leukemia (

A foundation model learns transcriptional regulatory syntax from chromatin accessibility and sequence data across a range of cell types to predict gene expression and transcription factor interactions...

Trained on unlabelled, unpaired image and text data, the Multimodal transformer with Unified maSKed modeling excelled in outcome prediction, image-to-text retrieval and visual question answering, pote...

Disclosures. Patel:Kronos Bio: Research Funding; Sobi: Honoraria; Bristol Myers Squibb: Honoraria; AbbVie: Honoraria; Sumitomo: Research Funding; Pfizer: R

Disclosures. Aldoss:Jazz Pharmaceuticals: Other: consulting fees; Takeda Pharmaceuticals: Other: consulting fees; Sobi: Other: consulting fees; Kite Pharma

Severe hemophilia A is managed with factor VIII replacement or hemostatic products that stop or prevent bleeding. Data on gene therapy with hematopoietic stem-cell (HSC)–based expression of factor ...

Leukemia - Genomic profiling of circulating tumor DNA for childhood cancers

Disclosures. Schrappe:JazzPharma, Servier, Amgen: Honoraria, Research Funding, Speakers Bureau. Cario:Jazz Pharmaceuticals: Other: travel support. Dalla-Po

Disclosures. Rau:Abbvie: Other: spouse currently employed; Servier: Other: Advisory board participation; Jazz Pharmaceuticals: Consultancy, Honoraria, Othe

Disclosures. Ware:Nova Laboratories: Other: Medical Advisory Board; Theravia: Other: Medical Advisory Board; Merck Pharmaceuticals: Other: Medical Advisory