Children’s Hospital of Philadelphia Is a Founding Member of the New Pediatric Preclinical Testing Consortium

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John M. Maris, MD

Addressing the relatively small number of new cancer drugs for children, a selective group of leading research centers is joining a new federally funded research consortium aimed at bringing scientific rigor and a concentrated effort to identifying new drug candidates for pediatric clinical trials.

The Children’s Hospital of Philadelphia (CHOP) and four other high-profile oncology research programs, plus a coordinating center, together form the Pediatric Preclinical Testing Consortium (PPTC) newly launched by the National Cancer Institute.

CHOP is building on its decades-long investigations in the biology and treatment of neuroblastoma. The other centers in the PPTC, selected like CHOP after a highly competitive process, focus on leukemia, brain tumors, osteosarcoma, sarcoma, and renal tumors.

“The primary rationale for this consortium is the fact that there are very few new drugs for pediatric cancer, and many of those drugs that have been introduced have been dependent on the results of clinical trials in adults,” said pediatric oncologist John M. Maris, MD, who leads CHOP’s research program within the PPTC. Dr. Maris will collaborate on this project with CHOP coinvestigators Edward Attiyeh, MD; Lori Hart, PhD; and Matthew Tang.

“Before testing a drug in children, we need a scientific basis for using it, based on deep understanding of the biology involved and supported by promising results in cell and animal models,” Dr. Maris added. “These preclinical findings will provide stronger evidence for us to engage proactively with drug companies who could partner in developing these drugs.” Dr. Maris also envisions that preclinical research will lead to rational drug combination strategies for more effective treatments, rather than a reliance on single agents.

Dr. Maris published an editorial commentary this month in The Journal of the American Medical Association1 in which he praises the great potential of genetic analysis for improving pediatric cancer treatment but calls for major efforts to mount rigorous preclinical testing of candidate drugs in the most appropriate cell and animal models.

About the PPTC

The PPTC is a program to systematically evaluate novel agents against pediatric solid tumor and leukemia models. The primary goal of the PPTC is to develop high-quality preclinical data to help pediatric oncology researchers identify new agents that will show significant activity when clinically evaluated against selected childhood cancers. By supporting a more reliable agent prioritization process, the PPTC can contribute to the goal of identifying more effective treatments for children with cancer. The PPTC builds upon 10 years of experience with the Pediatric Preclinical Testing Program, which collaborated with more than 50 pharmaceutical companies to test novel agents against the program’s pediatric preclinical models. The PPTC is supported through NCI cooperative agreement research grants to the PPTC Coordinating Center at Research Triangle Institute International (Primary Investigator, Diana Severynse-Stevens, PhD) and to five research programs responsible for the evaluation of agents against pediatric cancer preclinical models.

The research programs are led by:

  • Peter Houghton, PhD, Sarcoma and renal tumors, Greehey Children’s Cancer Institute
  • John Maris, MD, Neuroblastoma, Children’s Hospital of Philadelphia
  • Richard Lock, PhD, Leukemia, Children’s Cancer Institute, Sydney, Australia
  • Xiao-Nan Li, MD, PhD, Brain tumors, Texas Children’s Hospital
  • Richard Gorlick, MD, Osteosarcoma, Albert Einstein College of Medicine

More information about the PPTC is available at its website: ■


1. Schnepp RW, Bosse KR, Maris JM: Improving patient outcomes with cancer genomics: unique opportunities and challenges in pediatric oncology. JAMA 9:881-883, 2015.