A physician-researcher from The Children’s Hospital of Philadelphia will lead the first-ever pediatric “Dream Team” solely focused on creating new treatments for the most challenging childhood cancers.
Stand Up to Cancer (SU2C) and the St. Baldrick’s Foundation, along with the American Association for Cancer Research (AACR), SU2C’s scientific partner, announced this Dream Team during a press conference April 7 at the AACR Annual Meeting 2013 in Washington, D.C.
John M. Maris, MD, director of the Center for Childhood Cancer Research at The Children’s Hospital of Philadelphia, and professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania, will lead the pediatric Dream Team, which will have $14.5 million in funding over 4 years, provided by SUC2 and St. Baldrick’s. Crystal L. Mackall, MD, chief of the Pediatric Oncology Branch of the National Cancer Institute (NCI), is the co-leader of the Dream Team.
The title of the research project, “Immunogenomics to Create New Therapies for High-Risk Childhood Cancers,” reflects the melding of two powerful disciplines that have historically functioned independently: immunotherapeutics and genomics. The goal is to rapidly translate promising basic research into transformative, targeted treatments that will improve cure rates in children’s cancer.
Immunotherapeutics focuses on developing treatments that harness the body’s own immune system to fight disease. Genomics, the field that analyzes the broad landscape and fine details of the genetic code in DNA, reveals potential targets for disease treatments.
“The motivation for the creation of this collaborative research project is the realization that completely new strategies are needed if we are to have curative therapies for all childhood cancers,” said Dr. Maris. “Our Team hopes to rapidly develop more precise and effective treatments based on the unique characteristics of each child’s tumor, here focusing on the genetic changes that make the cancer cells different from the rest of the child’s body.”
At the press conference, Emily (Emma) Whitehead and her parents Kari and Tom provided dramatic evidence of the power of these scientific tools to treat cancer. Emily, age 7, had a dramatic recovery from a relapsed form of childhood leukemia following an experimental treatment at Children’s Hospital that used her own immune cells. Researchers from CHOP and the University of Pennsylvania genetically engineered Emily’s T cells to find and destroy leukemia cells. Emily, who was featured in news stories last December, remains healthy and cancer-free, nearly a year after receiving the highly innovative therapy.
The Dream Team will focus on the four most deadly pediatric cancers: malignant brain tumors, high-risk leukemias (such as the type of acute lymphoblastic leukemia that Emily Whitehead had), neuroblastomas (which affect the peripheral nervous system) and sarcomas (other tumors of bone and other tissue).
In transforming this scientific knowledge into treatments, the Dream Team members will take a three-step approach. First, they will discover cell-surface molecules offering promising targets in high-risk children’s cancers. Secondly, they will create immunotherapy-based proteins to attack those molecules. The third step is to carry out multi-institutional clinical trials of these treatments in children.
“The success of the program will ultimately be judged by the number of lives saved through our efforts,” concluded Dr. Maris.
For more information on the Dream Team, visit the CHOP Research Institute’s website.