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NCI Initiative to Speed Development of Childhood Cancer Therapies

, by NCI Staff

A computer model of a small molecule drug that inhibits its protein target.

Credit: Kevin Gardner, M.D., Ph.D., NCI Center for Cancer Research

NCI has awarded grants to five research teams to participate in its Pediatric Preclinical Testing Consortium (PPTC).

The PPTC is designed to address an important barrier to developing new drugs for the treatment of childhood cancers: producing reliable data from studies involving laboratory and animal models—often referred to as preclinical models—that can help to prioritize which agents to pursue in human clinical trials.

“Effective prioritization of potential drug candidates is critical,” stressed Malcolm Smith, M.D., Ph.D., associate branch chief of Pediatrics in NCI’s Cancer Therapy Evaluation Program. “There is a large universe of anticancer agents being developed for adult cancers, but because of the relatively small number of children with specific cancers, only a limited number of these agents can be studied in pediatric clinical trials.”

The five research teams were selected to conduct preclinical testing for specific childhood cancers based on their experience and on the preclinical models that they have previously developed, Dr. Smith explained.

Each team’s models have undergone comprehensive genomic analyses to demonstrate that they closely resemble the genetic alterations seen in the respective human cancers for which they were selected.

The principal investigators of each research team include:

  • Richard Gorlick, M.D., Albert Einstein College of Medicine (New York, NY): Osteosarcoma
  • Peter Houghton, Ph.D., Greehey Children’s Cancer Research Institute (San Antonio, TX): Sarcoma and renal cancers
  • Xiao-Nan Li, M.D., Ph.D., Baylor College of Medicine (Houston, TX): Brain cancers
  • John Maris, M.D., Children’s Hospital of Philadelphia: Neuroblastoma
  • Richard Lock, Ph.D., Children’s Cancer Institute (Sydney, Australia): Leukemia

The foundation for the PPTC, Dr. Smith continued, was the Pediatric Preclinical Testing Program (PPTP), a decade-long initiative in which NCI worked with more than 50 pharmaceutical companies to test novel agents in PPTP-provided preclinical models.

One of the most important findings from the PPTP was that many agents that have shown efficacy against adult cancers had limited activity in pediatric preclinical models, he said.

Some investigational drugs did show substantial activity in several models, however, including the MEK inhibitor selumetinib in gliomas with mutations in the BRAF gene and the PARP inhibitor talazoparib (in combination with low-dose temozolomide) for Ewing sarcoma. Both of these agents are now being tested in ongoing pediatric clinical trials.

The PPTP and other research teams have also shown that preclinical testing, when combined with knowledge about the relative drug exposures that can be tolerated by mice and humans, “provides powerful insight into the likely clinical utility of experimental agents,” Dr. Smith said.

As the NCI representatives on the PPTC steering committee, Dr. Smith and Beverly Teicher, Ph.D., of NCI’s Developmental Therapeutics Program, will work with the PPTC research teams to promote collaborations with pharmaceutical companies and academic laboratories for the testing of promising experimental agents against well-characterized childhood cancer preclinical models, he added.

Research Triangle Institute is the coordinating center for the PPTC. More information about the PPTC is available on its website or by contacting the PPTC at

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