? Pediatric Oncology (PO) Program The Pediatric Oncology (PO) Program of the Abramson Cancer Center (ACC) discovers, develops and translates novel therapeutic approaches to improve cure rates and reduce acute and long-term toxicities in children with cancer. Established in 1992, the Program has the overall goal to change the standard of care for children with cancer in the US and across the world. Scientific aims are: 1) Characterize molecular mechanisms of childhood cancers and predisposition for risk evaluation and precision therapies; 2) Further develop innovative cell therapies for hematologic malignancies to improve cure rates and minimize the use of allogeneic stem cell transplantation; and 3) Identify targets in high risk and relapsed solid tumors and develop new therapies for these challenging tumors. The Program is led by Stephan Grupp, MD, PhD, Professor of Pediatrics, Chief of the Cell Therapy and Transplant Section and Medical Director of the Cell and Gene Therapy Laboratory, and Kai Tan, PhD, Associate Professor of Pediatrics. Dr. Grupp is a world leader in cellular therapy, immunotherapy and translational research. Dr. Tan is a leading researcher in cancer genomics and systems biology. They provide translational and transdisciplinary leadership for 35 Program members, who form a dynamic community of investigators from three Departments in the Perelman School of Medicine. The Program has integrated basic, translational, and clinical research components, with a diverse group of investigators who have expertise in cancer genomics, cell biology and signal transduction, tumor immunology and immunotherapy, drug development, clinical pharmacology, epidemiology, clinical research, cancer control, survivorship, and behavioral oncology. Members collaborate within Pediatric Oncology, and with the Cancer Control, Cancer Therapeutics, Tumor Biology, Hematologic Malignancies, and Immunobiology Programs. Clinical investigations are robust, with 1,156 accruals to interventional trials and 2,946 to non- interventional trials in the current funding period. Members engage in research that is relevant to major pediatric cancer issues within our catchment area, and are highly engaged in educational activities across Penn and beyond. Most notably in the current funding period, PO members worked collaboratively with members of other Programs to develop CD19-targeted CAR-T cells (tisagenlecleucel), taking the first CAR-T therapy to FDA approval for children and young adults with ALL. Other accomplishments include advances for targeted therapy of pediatric leukemia, discovery of novel cancer pathways in neuroblastoma and gliomas, and two new FDA approved drugs for pediatric solid tumors. Program members also play major leadership roles in the national pediatric cooperative groups. Program members have $15.1M in annual grant funding (direct), of which $9M is peer-reviewed and $6.3M is NCI-funded. The Program holds 21 R01 equivalents. There were 570 cancer-related publications from the Program since 2015. Of these, 30% are intra-Programmatic, 18% resulted from inter-Programmatic collaboration, and 83% are multi-institutional.

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National Cancer Institute (NCI)
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Subcommittee I - Transistion to Independence (NCI)
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Hinderer, Christian; Katz, Nathan; Buza, Elizabeth L et al. (2018) Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN. Hum Gene Ther 29:285-298
Li, Jinyang; Byrne, Katelyn T; Yan, Fangxue et al. (2018) Tumor Cell-Intrinsic Factors Underlie Heterogeneity of Immune Cell Infiltration and Response to Immunotherapy. Immunity 49:178-193.e7
Raghunathan, Nirupa Jaya; Korenstein, Deborah; Li, Qing S et al. (2018) Determinants of mobile technology use and smartphone application interest in cancer patients. Cancer Med 7:5812-5819
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Safo, Sandra E; Li, Shuzhao; Long, Qi (2018) Integrative analysis of transcriptomic and metabolomic data via sparse canonical correlation analysis with incorporation of biological information. Biometrics 74:300-312
Kall, Stefanie L; Delikatny, Edward J; Lavie, Arnon (2018) Identification of a Unique Inhibitor-Binding Site on Choline Kinase ?. Biochemistry 57:1316-1325

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