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Biologic and Clinical Analysis of Childhood Gamma Delta T-ALL Identifies LMO2/STAG2 Rearrangements as Extremely High Risk

Acute lymphoblastic leukemia expressing the gamma delta T-cell receptor (γδ T-ALL) is a poorly understood disease. We studied 200 children with γδ T-ALL from 13 clinical study groups to understand the clinical and genetic features of this disease. We found age and genetic drivers were significantly associated with outcome.

Radiographic and visual response to the type II RAF inhibitor tovorafenib in children with relapsed/refractory optic pathway glioma in the FIREFLY-1 trial

Due to their anatomical locations, optic pathway gliomas (OPGs) can rarely be cured by resection. Given the importance of preserving visual function, we analyzed radiological and visual acuity (VA) outcomes for the type II RAF inhibitor tovorafenib in the OPG subgroup of the phase 2 FIREFLY-1 trial.

SNO-EANO-EURACAN consensus on management of pineal parenchymal tumors

Pineal parenchymal tumors are rare neoplasms for which evidence-based treatment recommendations are lacking. These tumors vary in biology, clinical characteristics, and prognosis, requiring treatment that ranges from surgical resection alone to intensive multimodal antineoplastic therapy.

Comments and Controversies in Oncology: The Tribulations of Trials Developing ONC201

Our international team highlights issues with efficacy reports in several studies on DMG with the new drug ONC201.

Combining CRISPR-Cas9 and TCR exchange to generate a safe and efficient cord blood-derived T cell product for pediatric relapsed AML

Hematopoietic cell transplantation (HCT) is an effective treatment for pediatric patients with high-risk, refractory, or relapsed acute myeloid leukemia (AML). However, a large proportion of transplanted patients eventually die due to relapse. To improve overall survival, we propose a combined strategy based on cord blood (CB)-HCT with the application of AML-specific T cell receptor (TCR)-engineered T cell therapy derived from the same CB graft.

Rare case of spontaneous simultaneous extensive subcutaneous emphysema, bilateral pneumothoraces, pneumomediastinum and pneumorrhachis

Nick Gottardo MBChB FRACP PhD Co-Head, Brain Tumour Research team; Head of Oncology and Haematology, Perth Children’s Hospital nicholas.gottardo@

Diffuse Intrinsic Pontine Glioma

This chapter summarizes recent advances in diffuse intrinsic pontine glioma and potential novel therapies

New therapeutic opportunities from dissecting the pre-B leukemia bone marrow microenvironment

We provide evidence that targeting leukemia-induced bone loss is a therapeutic strategy for pre-B ALL

A CHAF1B-Dependent Molecular Switch in Hematopoiesis and Leukemia Pathogenesis

Here we report that CHAF1B is required for normal hematopoiesis while its overexpression promotes leukemia

PI3K activation in neural stem cells drives tumorigenesis which can be ameliorated by targeting the cAMP response element binding protein

A novel mouse model for glioma demonstrating that the PI3K pathway is important for initiation of tumorigenesis