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Frank M. Balis, MD
Frank M. Balis
Director, Cancer Clinical Research

Dr. Balis's research focuses on the clinical pharmacology of anticancer drugs, new drug development, and clinical trial design and endpoints. He studies the pharmacokinetics and pharmacodynamics of anticancer drugs and has applied pharmacological principles to clinical trial design and clinical drug development. He is developing new biomarkers to serve as surrogate endpoints of drug toxicity or efficacy in clinical trials.



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As CHOP's institutional principal investigator for the Children's Oncology Group (COG), Dr. Balis oversees the Cancer Center's substantial contributions to the national clinical research efforts of the COG while striving to be one of the premier pediatric oncology clinical research program in Group.

Dr. Balis's clinical research focuses on characterizing the clinical pharmacology of anticancer drugs in children 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, COG researchers 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 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, 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 the 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. The COG has identified GD2 as a biomarker for high-risk neuroblastoma. GD2 may be useful as a diagnostic and prognostic tumor biomarker, and COG researchers are studying it in a prospective national clinical trial. They are also studying biomarkers of normal tissue or organ damage to more sensitively detect and measure drug toxicity.

Education and Training

BS, University of North Carolina, Chapel Hill (Zoology), 1971

MD, Vanderbilt University, 1975

Titles and Academic Titles

Director, Cancer Clinical Research

Institutional Principal Investigator, Children's Oncology Group

Attending Physician

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

Professional Memberships

Cancer Chemotherapy and Pharmacology, Editorial Board, 2009-

The Oncologist, Founding, Senior, and CME Editor, 1995-2017

National Comprehensive Cancer Network, Wilms Tumor Panel, 2019-

Professional Awards

Amos Christie Award in Pediatrics, 1975

Public Health Service Unit Commendation, 1990

Division of Clinical Science Director's Award, 1998

Public Health Service Outstanding Service Medal, 2000

NCI Mentor of Merit Award, 2001

NIH Award of Merit for Outstanding Contributions as Clinical Director, 2003

NIH Award of Merit for Successful Implementation of a Clinical Trial Management System, 2009

NIH Award of Merit for Successful Clinical Development of Glucarpidase, 2009

Chai Lifeline Community Service Award, 2017

Publication Highlights