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University of Pittsburgh

Research/Cost AccountingP.O. Box 371220
Pittsburgh, PA 15213
United States

Ewing sarcoma is a cancer most often diagnosed in teenagers. We know that some changes in the make-up of Ewing tumors make them more likely to be aggressive and spread to other parts of the body. When Ewing sarcoma spreads to other parts of the body, like the lung, it is very hard to cure. Radiation is one therapy used to treat Ewing sarcoma in the lungs. Radiation can directly damage tumor cells and can also "call" immune cells into the tumor to help attack cancer cells.

Hepatoblastoma (HB) is the most common pediatric liver cancer and can be classified into several different types. In mice, HBs can be generated by overexpressing in their livers any pairwise or triple combination of three genes that are commonly deregulated in human HBs: ?-catenin (B), YAP (Y) and NRF2 (N). The 4 resulting tumor types (BY, BN, YN and BYN) share many features with human tumors and are currently the best animal surrogates for human HB.

Medulloblastoma (MB) arises in the cerebellum. Group 3, one of four MB subgroups (WNT, SHH, Group 3, and Group 4), is the most aggressive and malignant type in children. Group 3 is characterized by frequent metastasis at diagnosis and the worst prognosis. Although surgical resection, radiotherapy, and chemotherapy are effective at eliminating some forms, patients with Group 3 cannot be cured with conventional therapies and also face a paucity of molecularly targeted therapies.

Background

This project focuses on Ewing’s Sarcoma, a cancer of the bone or soft tissue that predominately affects adolescents. Although patients with localized disease are often cured, the prognosis for patients with metastatic disease is dismal, with survival estimates as low as 10%. We do not yet understand why some Ewing’s Sarcoma cells metastasize, and patient outcomes will not improve until we fill this critical knowledge gap.

Children's Hospital of Pittsburgh (CHP) has a long tradition of participation in pediatric oncology research. CHP is one of 21 institutions in the Children's Oncology Group (COG) Phase 1 Consortium and 1 of 10 in the Pediatric Brain Tumor Consortium (PBTC). We enrolled the largest number of patients to PBTC trials and are the third highest enroller to COG Phase I studies this year.

Children's Hospital of Pittsburgh of UPMC (CHP), has a long-standing and solid tradition of contributing to early drug development for children with cancer. Our institution is a member of the Children's Oncology Group (COG) Phase 1 Consortium and a charter member of the Pediatric Brain Tumor Consortium (PBTC). We also participate in pharmaceutical studies as well as our own institutional new drug protocols and innovative pilot studies.


In simple terms, what is your research project?

Support for a new pediatric cancer drug therapy at Children's Hospital of Pittsburgh of UPMC.

What type of childhood cancer does your project focus on?