Frank M. Balis

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.