First pediatric cancer research dream team led by CHOP oncologist
John Maris, MD, of the Cancer Center at The Children's Hospital of Philadelphia, will lead the first ever pediatric “Dream Team” dedicated to creating new treatments for the most challenging childhood cancers. The pediatric Dream Team will have $14.5 million in funding over four years, provided by Stand Up to Cancer and St. Baldrick’s Foundation. Crystal L. Mackall, MD, chief of the Pediatric Oncology Branch of the National Cancer Institute (NCI), is co-leader alongside Dr. Maris.
Combining genomics and immunotherapy to increase cure rates
Members of the Dream Team include world-renowned pediatric cancer researchers and clinicians from two disciplines: genomics, which is the study of genes and their functions; and immunotherapeutics, which are treatments that use the body’s own immune system to fight disease.
Compared to adult cancers, genetic mutations that could inform research and the development of new treatment options are rare in pediatric cancers. This suggests that pediatric oncology research must move beyond traditional methods of identifying treatments in order to substantially improve outcomes for children with cancer, which have not improved in the past 20 years.
Immunotherapeutics targeting cell surface molecules have shown impressive results in pediatric clinical trials, supporting the idea that immunotherapeutics are an important strategy against childhood cancers. Genomics and immunotherapeutics are both highly productive fields of pediatric cancer translational investigation, but for the most part, they have evolved separately from one another. The Stand Up To Cancer-St. Baldrick’s Pediatric Dream Team will change this by forming a collaborative group of scientists from both fields.
The group has developed a three-pronged research approach to focus on the childhood cancers that are most difficult to treat and lead to the most deaths, such as some forms of relapsed leukemia, neuroblastoma, brain tumors, and other types of tumors. The title of the research project is “Immunogenomics to Create New Therapies for High-Risk Childhood Cancers.”
- They will first identify and validate cancer-specific, cell-surface molecules that could serve as potential targets for immunotherapy of high-risk pediatric cancers.
- Armed with this knowledge, they will then develop various immunotherapeutics that recognize and kill cancer cells expressing these molecules.
- Finally, the Dream Team plans to conduct studies with patients with the most promising of these agents.
The ultimate goal: new therapies that have the potential to dramatically improve survival rates for some of the most lethal childhood cancers, and to improve quality of life after treatment by offering less toxic therapies to children.
Patient Emily Whitehead, treated with an experimental immunotherapy at The Children's Hospital of Philadelphia, is just one example of the promise this area of research holds for children with cancer.
Stephan A. Grupp, MD, PhD, and Tom Curran, PhD, will lead the CHOP-based research programs. Grupp has developed the first highly effective childhood cancer immunotherapy, in collaboration with Carl H. June, MD, of the Perelman School of Medicine at the University of Pennsylvania. It was Grupp’s efforts that resulted in Emily Whitehead’s remarkable recovery from her lethal leukemia.
The Dream Team
The researchers represent seven institutions: CHOP, NCI, the Baylor College of Medicine, Seattle Children’s Hospital, the University of British Columbia, The Hospital for Sick Children (SickKids) in Toronto, and the University of Wisconsin.
The Dream Team also includes important patient advocates: Kelly Cotter, a childhood cancer survivor; Jay Scott and Liz Scott, of Alex’s Lemonade Stand Foundation; Patrick Sullivan, of the Team Finn Foundation; and Lisa Tichenor and Mac Tichenor, of the What Would Willy Want Foundation (Quad W Foundation).
Meet CHOP's representatives on the Stand Up to Cancer-St. Baldrick's Pediatric Dream Team
- John Maris, MD, Co-Leader
- Tom Curran, PhD, FRS, Executive Committee Member
- Stephan A. Grupp, MD, PhD, Executive Committee Member
- Richard Aplenc, MD, PhD, MSCE
- Craig Bassing, PhD
- Kristina A. Cole, MD, PhD
- Carolyn A. Felix, MD
- Katherine A. High, MD, Collaborator, Center for Molecular Therapeutics at CHOP
- Michael D. Hogarty, MD
- Susan Rheingold, MD
- Andrei Thomas-Tikhonenko, PhD
- Fraser Wright, PhD, Collaborator, Center for Molecular Therapeutics at CHOP
- Sharon Diskin, PhD
- David Barrett, MD
- Erica Carpenter, PhD
- Yimei Li, PhD
- Adam Resnick, PhD
- Alix Seif, MD, MPH
- Sarah Tasian, MD
- Patrick Viatour, PhD