Authors identified transcription factor activating protein 4 (TFAP4) as a critical effector of MYCN amplification in neuroblastoma and showed that silencing TFAP4 inhibits MYCN-amplified neuroblastoma cell growth.
TARGET study associated RNA signatures of cytotoxic tumor-infiltrating lymphocytes with the presence of activated NK-/T-cells and suggested improved outcomes in newly diagnosed high-risk neuroblastoma patients with MYCN-NA tumor.
Scientists proposed a framework to investigate the genomic alterations in neuroblastoma subtypes and identified TEAD4, a transcription factor, as a novel target for therapy.
This study by TARGET researchers analyzed DNA changes and sequenced the genomes, exomes, and transcriptomes of 1,699 pediatric leukemia and solid tumors. Out of the 142 genes associated with cancer in these pediatric patients, only 45% matched those found in similar studies of adult cancers.
A study of nearly 1,000 pediatric acute myeloid leukemia (AML) cases revealed marked differences between the genomic landscapes of pediatric and adult AML and offered directions for future work.
Molecular assessment of participants from pediatric AML trials showed that recurrent structural alterations and age-specific mutational profiles can be used to stratify subjects in terms of overall and progression-free survival, and it highlighted the need for age-tailored targeted therapies.
Researchers identified abundant expression of miR-106a, a marker for treatment resistance, in relapsed and refractory pediatric AML through a comprehensive miRNA profile to identify potential biomarkers as predictors for improved outcomes.
Authors performed comprehensive analysis of Wilms Tumors and identified convergence of numerous genetic changes on limited developmental pathways resulting in oncogenesis. Findings suggested that targeting common pathways was better for intervention than targeting individual gene mutations.
In this first comprehensive genomic study of T-lineage acute lymphoblastic leukemia (T-ALL),researchers identified a large number of unrecognized driver mutations and associated altered pathways. These results have significant therapeutic weight in children with T-ALL.
TARGET investigators examined whether circulating miRNAs can be used as prognostic biomarkers in osteosarcoma patients. miR-21, miR-221, and miR-106a were found to be expressed significantly higher in cancer samples and were correlated with outcome.