Frank M. Balis, MD

Professor of Pediatrics, University of Pennsylvania School of Medicine

Director of Clinical Research, Center for Childhood Cancer Research

Contact Frank M. Balis, MD



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The Children's Hospital of Philadelphia 3501 Civic Center Blvd., Room CTRB4024


Solid tumors in children, renal tumors in children, clinical pharmacology of anticancer drugs

Drug development for childhood cancers
Clinical pharmacology of anticancer drugs

Clinical pharmacology of anticancer drugs


Professor of Pediatrics at the Children's Hospital of Philadelphia (2009– present)


M.D., Medicine, Vanderbilt University (1975)
B.S., Zoology, University of North Carolina (1971)

Extended Bio

As director of Clinical Cancer Research for the Division of Oncology at The Children's Hospital of Philadelphia, I facilitate the clinical research of many outstanding and innovative investigators here at CHOP, and I oversee the Division’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 the premier pediatric oncology clinical research program in the country.

My clinical research focuses on the development of new drugs and treatment strategies for childhood cancers and on studying the pharmacology of anticancer drugs in children in order to optimize their effectiveness and minimize their side effects. 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 may not be predictable 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 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 and they hold the promise of being more effective and less toxic. 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 more safer and more effective treatments.


Selected Publications

Fox Elizabeth, Widemann Brigitte C, Chuk Meredith K, Marcus Leigh, Aikin Alberta, Whitcomb Patricia O, Merino Maria J, Lodish Maya, Dombi Eva, Steinberg Seth M, Wells Samuel A, Balis Frank M. Vandetanib in Children and Adolescents with Multiple Endocrine Neoplasia Type 2B Associated Medullary Thyroid Carcinoma. Clin Cancer Res. Vol 19(15) . 2013 Aug:4239-48.
Mossé Yael P, Lim Megan S, Voss Stephan D, Wilner Keith, Ruffner Katherine, Laliberte Julie, Rolland Delphine, Balis Frank M, Maris John M, Weigel Brenda J, Ingle Ashish M, Ahern Charlotte, Adamson Peter C, Blaney Susan M. Safety and activity of crizotinib for paediatric patients with refractory solid tumours or anaplastic large-cell lymphoma: a Children's Oncology Group phase 1 consortium study. Lancet Oncol. Vol 14(6) . 2013 May:472-80.
Widemann BC, Kim A, Fox E, Baruchel S, Adamson PC, Ingle AM, Glade-Bender J, Burke M, Weigel B, Stempak D, Balis FM Blaney SM.. A phase I trial and pharmacokinetic study of sorafenib in children with refractory solid tumors or leukemias: a Children's Oncology Group Phase I Consortium report. Clin Cancer Res. Vol 18(21) . 2013:6011-22.
Kim A, Dombi E, Tepas K, Fox E, Martin S, Wolters P, Balis FM, Jayaprakash N, Turkbey B, Muradyan N, Choyke PL, Korf B, Widemann BC.. Phase I trial and pharmacokinetic study of sorafenib in children with neurofibromatosis type I and plexiform neurofibromas. Pediatr Blood Cancer. Vol 60(3) . 2013:396-401.
Warren KE, Gururangan S, Geyer JR, McLendon RE, Poussaint TY, Wallace D, Balis FM, Berg SL, Packer RJ, Goldman S, Minturn JE, Pollack IF, Boyett JM, Kun LE.. A phase II study of O6-benzylguanine and temozolomide in pediatric patients with recurrent or progressive high grade gliomas and brainstem gliomas: A Pediatric Brain Tumor Consortium Study.. J Neuro-Oncol. Vol 106. 2012:643-9.
Lodish M, Dagalakis U, Chen C, Sinaii N, Whitcomb T, Aikin A, Dombi E, Marcus L, Widemann B, Fox E, Chuk M, Balis FM, Wells S, Jr, Stratakis CA.. 111 In-Octreotide scintigraphy for identification of metastatic medullary thyroid carcinoma in children and adolescents. J Clin Endocrinol Metab. Vol 97. 2012:E207-12.
Murphy RF, Komlodi-Pasztor E, Robey R, Balis FM, Farrell NP, Fojo T.. Retained platinum uptake and indifference to p53 status make novel transplatinum agents active in platinum resistant cells compared to cisplatin and oxaliplatin. Cell Cycle. Vol 11. 2012:963-73.
Chuk MK, Aikin A, Whitcomb T, Widemann BC, Zannikos P, Beyever E, Balis FM, Fox E.. A phase I trial and pharmacokinetic study of a 24-hour infusion of trabectedin (Yondelis®, ET-743) in children and adolescents with relapsed or refractory solid tumors.. Pediatr Blood Cancer. Vol 59. 2012:865-9.
Kim A, McCully C, Cruz R, Cole DE, Fox E, Balis FM, Widemann BC.. The plasma and cerebrospinal fluid pharmacokinetics of sorafenib after intravenous administration in non-human primates. Invest New Drugs. Vol 30. 2012:524-8.
Balis FM, Fox E.. Challenges of developing new drugs for childhood cancers. Clin Investig. Vol 2. 2012:291-300.

Books - Chapters

Adamson PC, Balis FM, Bagatell R, Blaney SM. General principles of chemotherapy. In Pizzo PA, Adamson PC and Poplack DG (eds). Principles and Practice of Pediatric Oncology 6th Ed. Philadelphia, Lippincott Williams & Wilkins. 2010.

Fox E, Citrin D, Balis FM. The legacy of cancer therapy in children. J Natl Cancer Inst 101:1105-7, 2009.

Fox E, Balis FM. Drug therapy in newborns and children. In Atkinson AJ, Daniels CE, Dedrick RL Markey SP (eds). Principles of Clinical Pharmacology 2nd Ed. San Diego, Academic Press pp. 359-373, 2007.

Lowe ES, Balis FM. Dose-effect and concentration-effect analysis. In Atkinson AJ, Daniels CE, Dedrick RL, Markey SP (eds). Principles of Clinical Pharmacology 2nd Ed. San Diego, Academic Press, pp. 289-300, 2007

Original Papers

Widemann BC, Balis FM, Kim A, Boron M, Jayaprakash N, Shalabi A, et al. Glucarpidase, leucovorin, and thymidine for high-dose methotrexate-induced renal dysfunction: clinical and pharmacologic factors affecting outcome. J Clin Oncol. 2010 Sep 1;28(25):3979-86. Epub 2010 Aug 2.

Meany H, Balis FM, Aikin A, Whitcomb P, Murphy RF, Steinberg SM, et al. Pediatric phase I trial design using maximum target inhibition as the primary endpoint. J Natl Cancer Inst. 2010 Jun 16;102(12):909-12. Epub 2010 May 11.

Jacobs S, Fox E, Krailo M, Hartley G, Navid F, Wexler L, et al. Phase 2 trial of ixabepilone administered daily for five days in children and young adults with refractory solid tumors: a report from the Children’s Oncology Group. Clin Cancer Res 16:750-4, 2010.

Jacobs S, McCully CL, Bacher J, Balis FM, Fox E. Extracellular fluid concentrations of cisplatin, carboplatin and oxaliplatin in brain, muscle and blood measured using microdialysis in non-human primates. Cancer Chemother Pharmacol 65:817-24, 2010.

Fox E, Widemann BC, Hawkins D, Jayaprakash N, Dagher R, Aikin AA, et al. Randomized trial of pegfilgrastim vs. filgrastim after chemotherapy in children and young adults with newly diagnosed sarcoma. Clin Cancer Res 15:7361-7, 2009.

Meany HJ, Sackett DL, Maris JM, Ward Y, Krivoshik A, Cohn SL, et al. Clinical outcome in children with recurrent neuroblastoma treated with ABT 751 and effect of ABT-751 on proliferation of neuroblastoma cell lines and on tubulin polymerization in. Pediatr Blood Cancer 54:47-54, 2009.

Wojtuszewski Poulin K , Smirnov AV, Hawkins ME, Balis FM, Knutson JR. Conformational heterogeneity and quasi-static self quenching in DNA containing fluorescent guanine analogs 3MI or 6MI. Biochemistry 48:8861-8, 2009.

Meany HJ, Warren KE, Fox E, Cole DE, Aikin AA, Balis FM. Pharmacokinetics of temozolomide administered in combination with O6-benzylguanine in children and adolescents with refractory solid tumors. Cancer Chemother Pharmacol 65:137-42, 2009.

Abraham J, Edgerly M, Wilson R, Chen C, Rutt A, Bakke S, Robey R, Dwyer A, Goldspiel B, Balis FM, van Tellingen O, Bates S, Fojo A. A Phase I study of the P-glycoprotein (Pgp) antagonist tariquidar (XR9576) in combination with vinorelbine. Clin Cancer Res 15:3574-82, 2009.

Widemann BC, Goodspeed W, Goodwin A, Fojo T, Balis FM, Fox E. Phase I trial and pharmacokinetic study of ixabepilone administered daily for five days in children and adolescents with refractory solid tumors. J Clin Oncol 27:550-6, 2009.

Fox E, Maris JM, Widemann BC, Goodspeed W, Goodwin A, Kromplewski M, Fouts ME, Medina D, Cohn SL, Krivoshik A, Hagey AE, Adamson PC, Balis FM. A phase I study of ABT-751, an orally bioavailable tubulin inhibitor, administered daily for 21 days every 28 days in pediatric patients with solid tumors. Clin Cancer Res 14:1111-5, 2008.

Warren KE, McCully C, Dvinge H, Tjørnelund J, Sehested M, Lichenstein HS, Balis FM. Plasma and cerebrospinal fluid pharmacokinetics of the histone deacetylase inhibitor, belinostat (PXD101), in non-human primates. Cancer Chemother Pharmacol 62:433-7, 2008.

Fox E, Razzouk BI, Widemann BC, Xiao S, O’Brien M, Goodspeed W, et al. Phase I trial and pharmacokinetic study of arsenic trioxide in children and adolescents with refractory or relapsed acute leukemia including acute promyelocytic leukemia or lymphoma. A collaborative study of the Pediatric Oncology Branch, National Cancer Institute and the Children’s Oncology Group. Blood 111:566-73, 2008.

Meany HJ, Fox E, McCully C, Tucker C, Balis FM. The plasma and cerebrospinal fluid pharmacokinetics of erlotinib and its active metabolite (OSI-420) after intravenous administration of erlotinib in non-human primates. Cancer Chemother Pharmacol 62:387-92, 2008.

Bernardi RJ, Bomgaars L, Fox E, Balis FM, Egorin MJ, Lagattuta TF, Aikin A, Whitcomb P, Renbarger J, Lieberman FS, Berg SL, Blaney SM. Phase I clinical Trials of intrathecal gemcitabine in patients with neoplastic meningitis. Cancer Chemother Pharmacol 62:355-61, 2007.

Adamson PC, Matthay KK, O’Brien M, Seeger R, Reaman GH, Sato JK, Balis FM. A phase 2 trial of all-trans-retinoic acid in combination with interferon-alpha-2a in children with recurrent neuroblastoma or wilms tumor: A Pediatric Oncology Branch, NCI and Children’s Oncology Group Study. Pediatr Blood Cancer 49:661-5, 2007.

Jacobs SS, Stork LC, Bostrom BC, Hutchinson R, Holcenberg J, Reaman GH, Erdmann G, Franklin J, Steinberg SM, Balis FM, Adamson PC. A pilot trial of oral and intravenous 6-thioguanine for the treatment of children with standard risk acute lymphoblastic leukemia: A collaborative Children’s Cancer Group/National Cancer Institute Study. Pediatr Blood Cancer 49:250-3, 2007

Dombi E, Solomon J, Gillespie AJ, Fox E, Balis FM, Patronas N, Korf BR, Babovic-Vuksanovic D, Packer RJ, Belasco J, Goldman S, Jakacki R, Kieran M, Steinberg SM, Widemann BC. NF1 plexiform neurofibroma growth rate by volumetric MRI: Relationship to age and body weight. Neurol 68:643-7, 2007.