New Research Consortium Aims to Identify Drugs for Pediatric Cancers

The Children’s Hospital of Philadelphia and four other high-profile oncology research programs plus a coordinating center joined the new Pediatric Preclinical Testing Consortium (PPTC) launched by the National Cancer Institute (NCI) to help researchers identify drug candidates for pediatric clinical trials. The centers will focus on neuroblastoma, leukemia, brain tumors, osteosarcoma, 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 Edward Attiyeh, MD, Lori Hart, 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.”

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.

Maris’ laboratory at CHOP has created animal models of neuroblastoma that incorporate genetic material from patient tumor cells. These models allow scientists to design drug tests highly tailored to specific, well characterized subtypes of human neuroblastoma tumors. Because neuroblastoma is particularly complex and variable, 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.

In order to keep pace with genomic medicine, Dr. Maris and colleagues discussed in a recent editorial published in JAMA that academic, federal, and industry leaders must develop new models of drug development, as “rare diseases become even more rare” once genetically defined subsets are revealed.

The commentary addressed results from a study conducted by researchers from the University of Michigan, Ann Arbor, appearing in the same issue that included 91 children and young adults with relapsed, refractory, or rare cancer who underwent genetic sequencing and genetic counseling. A troubling concern highlighted by the study was “the number of children with currently incurable refractory cancer and a potentially actionable finding in which the investigators simply could not find a drug that might have provided some measure of antitumor benefit,” the editorial stated.

The PPTC is supported through NCI cooperative agreement research grants to the PPTC Coordinating Center at Research Triangle Institute International. In addition to CHOP, the other research programs participating in the consortium include Greehey Children’s Cancer Institute, San Antonio (sarcoma and renal tumors); Children’s Cancer Institute, Sydney, Australia (leukemia); Texas Children’s Hospital, Houston (brain tumors), and Albert Einstein College of Medicine, New York (osteosarcoma).


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