CHOP Is a Founding Member of New NIH-Funded Research Consortium to Test New Cancer Treatments for Children

Preclinical Testing Program Draws on CHOP’s Expertise in Neuroblastoma to Streamline Drug Development

Dr. Maris in lab 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, part of the National Institutes of Health.

CHOP is building on its decades-long investigations in the biology and treatment of the childhood cancer neuroblastoma, an often-deadly tumor of the peripheral nervous system. The other centers in the PPTC, selected like CHOP after a highly competitive process, focus on leukemia, brain tumors, osteosarcoma (tumors in bone), and sarcoma and kidney 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. Maris will collaborate on this project with CHOP co-investigators Dr. Edward Attiyeh, Dr. Lori Hart and Mr. 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,” he added. “These preclinical findings will provide stronger evidence for us to engage proactively with drug companies who could partner in developing these drugs.” Maris also envisions that preclinical research will lead to rational drug combination strategies for more effective treatments, rather than a reliance on single agents.

Maris’s laboratory at CHOP has created animal models of neuroblastoma that incorporate genetic material from patient tumor cells. The value of these models, he stressed, is that they allow scientists to design drug tests highly tailored to specific, well characterized subtypes of human neuroblastoma tumors.

Because neuroblastoma is particularly complex and variable, Maris continued, this approach will bring oncologists closer to personalized medicine: using knowledge of the genetics of a patient’s tumor to directly attack a cancer’s vulnerabilities while sparing healthy cells.

Maris published an editorial commentary this month in the Journal of the American Medical Association 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 NCI Pediatric Preclinical Testing Consortium (PPTC): The NCI-supported Pediatric Preclinical Testing Consortium (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 ten 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 (RTI) International (PI, Dr. Diana Severynse-Stevens) and to five research programs responsible for the evaluation of agents against pediatric cancer preclinical models. The research programs are led by Dr. Peter Houghton (Sarcoma and renal tumors, Greehey Children’s Cancer Institute), Dr. John Maris (Neuroblastoma, Children’s Hospital of Philadelphia), Dr. Richard Lock (Leukemia, Children’s Cancer Institute, Sydney, Australia), Dr. Xiao-Nan Li (Brain tumors, Texas Children’s Hospital), and Dr. Richard Gorlick (Osteosarcoma, Albert Einstein College of Medicine). More information about the PPTC is available at its website:

About The Children’s Hospital of Philadelphia: The Children’s Hospital of Philadelphia was founded in 1855 as the nation’s first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals and pioneering major research initiatives, Children’s Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. In addition, its unique family-centered care and public service programs have brought the 535-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit

Contact: The Children’s Hospital of Philadelphia,