Cancer Pharmacology Laboratory



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The Cancer Pharmacology Laboratory focuses on optimizing the safety and efficacy of new and conventional anticancer drugs, developing new drugs for childhood cancers, and devising new clinical trial designs and endpoints to expedite the development of new treatments.

The Cancer Pharmacology Laboratory characterizes the clinical pharmacology of anticancer drugs by developing methods to measure the concentration of these drugs in the plasma of children with cancer and analyzing these concentrations using computer modeling and simulation software in order to identify dosing methods that will optimize each drug's therapeutic effects and minimize toxicity.

The lab is identifying and validating new biomarkers to measure the capacity of the kidneys and liver to eliminate drugs. These biomarkers will be used with the knowledge of a drug's clinical pharmacology to devise more accurate, individualized dosing methods. In addition, the lab is developing biomarkers to more sensitively detect and quantify drug toxicity and to accurately measure drug activity and efficacy to serve as early endpoints in clinical trials. Discovery and validation of circulating tumor biomarkers is also a focus of the lab.

The Cancer Pharmacology Laboratory also conducts a variety of clinical trials of new investigational agents for childhood cancers with an emphasis on studying the new classes of drugs that target the genetic defects in cancer cells that contribute to their malignant behavior.

Future directions in the Cancer Pharmacology Laboratory will include characterizing the clinical pharmacology of biological drugs in children. Biologics are agents that come from living organisms, such as antibodies and cellular therapies. This class of drugs will have an increasingly important role in the treatment of childhood cancers.

Project Highlights

  • The ganglioside GD2 has been validated as a circulating tumor biomarker for neuroblastoma. GD2 is selectively elevated in the serum of children with high-risk, high-stage disease. GD2 was not elevated in children with ganglioneuroma and ganglioneuroblastoma-intermixed subtype, and in children with 10 other childhood cancers. GD2 concentrations were significantly higher in serum from children with MYCN amplified tumors, high-risk tumors, stage 4 tumors, and in children who died of their disease.
  • Pre-clinical pharmacology studies have included evaluation of molecularly targeted agents in murine models of childhood cancer to establish drug concentrations necessary for target inhibition. In addition, the lab leads pharmacology objectives of ongoing and future clinical trials including: sorafenib, alisertib, trametinib, methotrexate, carboplatin, vincristine, and etoposide. The goal of these studies is to integrated pre-clinical and clinical pharmacology to optimize drug exposure, reduce toxicity, and improve outcome in infants, children, and adolescents with cancer.
  • The lab is assessing the determinants of drug exposure, including age and organ function, to optimize therapy for childhood cancer by developing biomarkers of renal function prior to administration of nephrotoxic therapy. In addition, the lab is developing standardized dosing of chemotherapy for infants with cancer.
Frank M. Balis

Frank M. Balis, MD

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.