The Pediatric Malignancies (PE) Program focuses on malignancies unique to children (neuroblastoma, retinoblastoma, diffuse midline gliomas, and medulloblastoma), as well as cancers more common in children than adults: acute lymphoblastic leukemia (ALL), bone sarcomas, and low-grade gliomas (LGG). Pediatric solid tumors, often embryonal in origin, provide opportunities to understand the links between normal development and aberrant signaling in childhood malignancies; to discover therapeutic targets; and to integrate these discoveries into innovative clinical trials. Inherited cancer predispositions, common in pediatric cancers, offer opportunities to uncover genes and proteins that regulate normal growth and are deregulated in cancer. These molecular studies also inform interactions between tumors and the tumor microenvironment in cancer development, understanding the late effects of treatment, and improving the outcome for survivors. The overall goals of the PE Program are (1) to understand the biology of pediatric malignancies and uncover links between normal development and cancer to identify therapeutic targets; and (2) to translate laboratory discoveries into clinical trials, and epidemiological and survivorship studies to improve clinical outcomes for children with cancer. These goals are addressed by undertaking research in four disease-specific themes: Theme 1: Acute leukemia Theme 2: Brain tumors Theme 3: Neuroblastoma Theme 4: Sarcoma and other pediatric solid tumors PE Program: Key Metrics Membership (12 departments, 2 schools) 30 Full 23 Associate 7 Cancer-relevant Funding (direct costs as of $14,090,085 05/31/2017) NCI $4,160,966 30% Peer-reviewed $5,888,503 42% Non-peer-reviewed $4,040,617 29% Cancer-relevant Publications (1/2012-7/2017) 506 Inter-programmatic 201 40% Intra-Programmatic 139 27% High-Impact 184 36% Accruals to Clinical Trials (2016) 188 85 Therapeutic 88 67 Other Interventional 2 5 Non-interventional 98 13
| Dvorak, Christopher C; Satwani, Prakash; Stieglitz, Elliot et al. (2018) Disease burden and conditioning regimens in ASCT1221, a randomized phase II trial in children with juvenile myelomonocytic leukemia: A Children's Oncology Group study. Pediatr Blood Cancer 65:e27034 |
| Fan, Qi Wen; Nicolaides, Theodore P; Weiss, William A (2018) Inhibiting 4EBP1 in Glioblastoma. Clin Cancer Res 24:14-21 |
| Sannino, Sara; Guerriero, Christopher J; Sabnis, Amit J et al. (2018) Compensatory increases of select proteostasis networks after Hsp70 inhibition in cancer cells. J Cell Sci 131: |
| Lam, Christine; Ferguson, Ian D; Mariano, Margarette C et al. (2018) Repurposing tofacitinib as an anti-myeloma therapeutic to reverse growth-promoting effects of the bone marrow microenvironment. Haematologica 103:1218-1228 |
| Truillet, Charles; Parker, Matthew F L; Huynh, Loc T et al. (2018) Measuring glucocorticoid receptor expression in vivo with PET. Oncotarget 9:20399-20408 |
| Phillips, Kathryn A; Trosman, Julia R; Deverka, Patricia A et al. (2018) Insurance coverage for genomic tests. Science 360:278-279 |
| Phillips, Kathryn A (2018) Evolving Payer Coverage Policies on Genomic Sequencing Tests: Beginning of the End or End of the Beginning? JAMA 319:2379-2380 |
| Puri, Sapna; Roy, Nilotpal; Russ, Holger A et al. (2018) Replication confers ? cell immaturity. Nat Commun 9:485 |
| An, Zhenyi; Aksoy, Ozlem; Zheng, Tina et al. (2018) Epidermal growth factor receptor and EGFRvIII in glioblastoma: signaling pathways and targeted therapies. Oncogene 37:1561-1575 |
| Behr, Spencer C; Villanueva-Meyer, Javier E; Li, Yan et al. (2018) Targeting iron metabolism in high-grade glioma with 68Ga-citrate PET/MR. JCI Insight 3: |
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