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Each year, approximately 1000 children in Australia and New Zealand, aged 0–14 years, are diagnosed with cancer. Despite paediatric cancer accounting for less than 1% of all cancer cases, the impact on their families and communities is profound and disproportionate.
There are few options for patients with relapse/refractory B-cell acute lymphoblastic leukemia, thus this is a major area of unmet medical need. Here, we reveal that inclusion of a poison exon in RBM39, which could be induced both by CDK9 or CDK9 independent CMGC (cyclin-dependent kinases, mitogen-activated protein kinases, glycogen synthase kinases, CDC-like kinases) kinase inhibition, is recognized by the nonsense-mediated mRNA decay pathway for degradation.
An in-depth investigation of gene regulation and cell populations at sites of fetal blood-cell production provides clues as to why children with Down’s syndrome are predisposed to developing leukaemia.
T cells engineered to express chimeric-antigen receptors (CAR-T cells) can effectively control relapsed and refractory haematological malignancies in the clinic. However, the successes of CAR-T cell therapy have not been recapitulated in solid tumours due to a range of barriers such as immunosuppression, poor infiltration, and tumour heterogeneity.
Nick Gottardo MBChB FRACP PhD Co-Head, Brain Tumour Research team; Head of Oncology and Haematology, Perth Children’s Hospital nicholas.gottardo@
Citation: Bailey HD. Exploring Exclusive Breastfeeding and Childhood Cancer Using Linked Data. JAMA Netw Open. 2024;7(3):e243075 Keywords:
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.
Citation: Sek K, Chen AXY, Cole T, Armitage JD, Tong J, ……… Waithman J, Parish IA, et al. Tumor site-directed A1R expression enhances CAR T cell
Gliomas are the most common type of malignant primary central nervous system (CNS) tumors, resulting in significant morbidity and mortality in children and adolescent and young adult (AYA) patients. The discovery of mutations in isocitrate dehydrogenase (IDH) genes has dramatically changed the classification and understanding of gliomas. IDH mutant gliomas have distinct clinical, pathological, and molecular features including a favorable prognosis and response to therapy compared to their wildtype counterparts.
Peroxisome proliferator-activated receptor γ (PPARγ) is a prominent ligand-inducible transcription factor involved in adipocyte differentiation, glucose homeostasis, insulin sensitivity, inflammation, and cell proliferation, making it a therapeutic target for diabetes, metabolic syndrome, autoimmune diseases, and cancer.