Frank M. Balis, MD

Frank M. Balis, MD, is Director of Clinical Cancer Research in the Center for Childhood Cancer Research and holds the Louis and Amelia Canuso Family Endowed Chair for Clinical Research in Oncology. He has particular expertise in clinical pharmacology of anti-cancer drugs and the care of children with renal tumors.

Areas of Expertise: Clinical pharmacology, Renal tumors


As director of Clinical Cancer Research for the Center for Childhood Cancer Research, I facilitate the clinical research programs of many outstanding and innovative clinical investigators here at CHOP, and I oversee the Cancer Center’s substantial contributions to the national clinical research efforts of The Children’s Oncology Group and other smaller cooperative groups. We are striving to be one of the premier pediatric oncology clinical research program in the country.

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 can not 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; and, 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.

New drugs are initially used to treat cancers that have relapsed or not responded to conventional treatments, and they are usually only available through participation on a clinical trial. This allows us to learn whether the new drug is tolerable in children, define the best dose for children and assess whether it is effective for childhood cancers. Our clinical trials of new drugs are also designed to minimize the risk to patients receiving the new drug because the side effects and safe dose for children are usually not known and cannot always be accurately predicted from the experience with the use of the drug in adults. The commitment of our clinical research team and our patients and their families to learn as much as we can about the cancer and about new drugs and new treatment approaches while we provide optimal care aimed at controlling or curing the cancer advances our knowledge and leads to safer and more effective treatments.

Education and Training

Medical School

MD - Vanderbilt Medical School, Nashville, TN


Pediatrics - Vanderbilt Medical School, Nashville, TN


Pediatric Hematology/Oncology - The Children's Hospital and Medical Center, Seattle, WA

Board Certification

Pediatric Hematology/Oncology

Titles and Academic Titles

Director of Clinical Research, Center for Childhood Cancer Research

Co-program Director for Pediatric Oncology, Abramson Cancer Center, University of Pennsylvania

The Louis and Amelia Canuso Family Endowed Chair for Clinical Research in Oncology

Professor of Pediatrics, Perelman School of Medicine at the University of Pennsylvania

Departments and Services

Areas of Focus

Laboratories & Research Programs

Research Topics




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. Read the abstract.

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. Read the abstract.

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. Read the abstract.

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. Read the abstract.

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. Read the abstract



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

Editorial positions

2000-present, Editorial Board, Cancer Chemotherapy and Pharmacology
1995-present, Founding, Senior, and CME Editor, The Oncologist

Academic positions

2009-present, Institutional Principal Investigator, Children’s Oncology Group
2009-present, CCCR Clinical Research Quality Assurance Committee
2009-present, Chair, CCCR Pediatric Protocol Review Committee