Children's Oncology Group

PROJECT SUMMARY
Since the introduction of chemotherapy for the treatment of childhood leukemia more than 60 years ago, the
prognosis of childhood cancer has improved dramatically. The overall 5-year survival rate for childhood cancers,
many of which were uniformly fatal in the pre-chemotherapy era, is now 84%. Progress for a number of childhood
cancers, however, has been limited, with approximately 50% of children with acute myelogenous leukemia, 50%
of children with high-risk neuroblastoma, and more than 90% of children with brainstem glioma, still succumbing
to their disease. In the US, cancer remains the leading cause of death from disease in children greater than one
year of age. Moreover, the late effects of cancer treatment, including permanent organ and tissue damage,
hormonal and reproductive dysfunction and second cancers, are of special concern, with more than 40% of the
500,000 survivors of childhood cancer (estimated as of 2020) experiencing a significant health related quality of
life complication from childhood cancer and its treatment. Thus, despite our advances, development of new
therapeutic approaches must be a priority for childhood cancer basic, translational and clinical researchers. The
Children’s Oncology Group (COG), the world’s largest organization devoted exclusively to childhood and
adolescent cancer research, was founded 20 years ago. The COG’s multidisciplinary research team, comprised
of more than 9,000 members, conducts research at more than 220 leading children’s hospitals, universities, and
cancer centers. This proposal is for COG, as part of the National Cancer Institute’s (NCI) National Clinical Trials
Network (NCTN), to continue its collaborative research work that supports the mission of improving the outcome
for all children with cancer. The COG will design and conduct clinical-translational studies for children with cancer
that builds on an increasing understanding of the molecular basis for pediatric malignancies and has the highest
potential to improve the outcome. Using innovative clinical trial designs suitable for the study of rare diseases,
we will study novel therapeutic approaches including but not limited to targeted small molecule drugs,
immunotherapies and cellular therapies. The COG research portfolio importantly also includes clinical trials
focused on improving the quality of life for children with cancer and childhood cancer survivors. As more than
90% of children diagnosed with cancer in the US are treated at COG member institutions, COG has the ability to
offer a diverse population of children with cancer and their families the opportunity to participate in innovative
research. This research effort includes allowing for collection and annotation of biospecimens from all children
with cancer, providing the foundation for discovery and accelerating the most promising research efforts
conducted in laboratories around the world. The proposal is for support of the COG Network Operations Center,
scientists who develop and conduct research, and for member sites to participate through enrollment of
eligible subjects.