As CHOP’s Institutional Principal Investigator for the Children’s Oncology Group (COG), I oversee the Cancer Center’s substantial contributions to the national clinical research efforts of the COG. We are striving to be one of the premier pediatric oncology clinical research program in the COG.
My clinical research focuses on characterizing the clinical pharmacology of anticancer drugs in children in order to optimize their effectiveness and minimize their side effects, and on the development of new drugs and treatment strategies for childhood cancers. The dose and administration schedule of a drug, its effectiveness against childhood cancers, and the types and severity of side effects that can occur in children cannot be predicted from the experience with the drug in adults. As a result, we test new drugs in children separately by evaluating side effects at varying dose levels, by measuring the response of the cancer, and by studying their clinical pharmacology by measuring the amount of drug in the body and its rate of elimination.
Anticancer drug development has been revolutionized by our increasing knowledge of the genetic and molecular defects in the various types of cancer that cause a cell to become cancerous. New classes of anticancer drugs specifically target these defects; as a result, they can more selectively block the growth of cancer cells. These new anticancer drugs hold the promise of being more effective and less toxic. This includes biological agents, which are playing an increasingly important role in the treatment of childhood cancers, such as immunotherapy. The applicability of these new classes of drugs to childhood cancers and the need to develop new approaches to study these drugs in children with cancer are new challenges that our drug development program has the experience and expertise to address.
Biomarkers are essential tools in clinical practice and clinical research. Tumor biomarkers are useful for screening or early detection of cancers, for diagnosis and determining prognosis, and for predicting response to treatment, monitoring response and detecting relapse or tumor progression. Research applications of tumor biomarkers parallel their clinical uses. We have identified GD2 as a biomarker for high-risk neuroblastoma. GD2 may be useful as a diagnostic and prognostic tumor biomarker, and we are studying it in a prospective national clinical trial. We are also studying biomarkers of normal tissue or organ damage to more sensitively detect and measure drug toxicity.
Education and Training
MD - Vanderbilt Medical School, Nashville, TN
Pediatrics - Vanderbilt Medical School, Nashville, TN
Pediatric Hematology/Oncology - The Children's Hospital and Medical Center, Seattle, WA
Pediatric Hematology-Oncology – American Board of PediatricsPediatrics – American Board of Pediatrics
Titles and Academic Titles
The Louis and Amelia Canuso Family Endowed Chair for Clinical Research in Oncology
Institutional Principal Investigator, Children’s Oncology Group
Professor of Pediatrics, Perelman School of Medicine at the University of Pennsylvania
Departments and Services
Areas of Focus
Laboratories & Research Programs
Fox E, Widemann BC, Pastakia D, Chen CC, Yang SX, Cole D, Balis FM. Pharmacokinetic and pharmacodynamic study of tariquidar (XR9576), a P-glycoprotein inhibitor, in combination with doxorubicin, vinorelbine or docetaxel in children and adolescents with refractory solid tumors. Cancer Chemother Pharmacol 76:1273-83, 2015.
Stieglitz E, Ward AF, Gerbing RB, Alonzo TA, Arceci RJ, Liu YL, Emanuel PD, Widemann BC, Cheng JW, Jayaprakash N, Balis FM, Castleberry RP, Bunin NJ, Loh ML, Cooper TM. Phase II/III trial of a pre-transplant farnesyl transferase inhibitor in juvenile myelomonocytic leukemia: a report from the Children's Oncology Group. Pediatr Blood Cancer. 2015 Apr;62(4):629-36. doi: 10.1002/pbc.25342. Epub 2014 Dec 8. PMID: 25704135.
Desai A, Fox E, Smith M, Lim A, Maris JM, Balis FM. Pharmacokinetics of the chimeric anti-GD2 antibody, ch14.18, in children with high-risk neuroblastoma. Cancer Chemother Pharmacol 74:1047-55, 2014.
Widemann BC, Babovic-Vuksanovic D, Dombi E, Wolters PL, Goldman S, Martin S, Goodwin A, Goodspeed W, Kieran MW, Cohen B, Blaney SM, King A, Solomon J, Patronas N, Balis FM, Fox E, Steinberg SM, Packer RJ. Phase II trial of pirfenidone in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas. Pediatr Blood Cancer. 2014 Sep;61(9):1598-602. doi: 10.1002/pbc.25041. Epub 2014 Apr 22. PMID: 24753394.
Nella AA, Lodish MB, Fox E, Balis FM, Quezado MM, Whitcomb PO, Derdak J, Kebebew E, Widemann BC, Stratakis CA. Vandetanib successfully controls medullary thyroid cancer-related Cushing syndrome in an adolescent patient. J Clin Endocrinol Metab. 2014 Sep;99(9):3055-9. doi: 10.1210/jc.2013-4340. Epub 2014 Mar 11. PMID: 24617713.
Widemann BC, Dombi E, Gillespie A, Wolters P, Belasco J, Goldman S, Korf B, Solomon J, Martin S, Salzer W, Fox E, Patronas N, Kieran M, Perentesis J, Reddy A, Wright J, Kim A, Steinberg S, Balis FM. Phase 2 randomized, flexible crossover, double-blinded, placebo-controlled trial of the farnesyltransferase inhibitor tipifarnib in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas. Neuro-oncol 16:707-18, 2014.
Adamson PC, Blaney SM, Bagatell R Skolnik JM, Balis FM. General principles of chemotherapy. In Pizzo PA, Adamson PC, Poplack DG, editors. Principles and practice of pediatric oncology 6th ed. Philadelphia: Lippincott Williams & Wilkins; 2016.
Editorial and Academic Positions
2000-present, Editorial Board, Cancer Chemotherapy and Pharmacology
1995-2017, Founding, Senior, and CME Editor, The Oncologist
2019-present, Chair, NCCN Wilms Tumor Panel
2009-present, Institutional Principal Investigator, Children’s Oncology Group
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