Overview

The Therapeutically Applicable Research to Generate Effective Treatments (TARGET) initiative employed comprehensive molecular characterization to determine the genetic changes that drive the initiation and progression of hard-to-treat childhood cancers. TARGET makes the data generated available to the research community with a goal to identify therapeutic targets and prognostic markers so that novel, more effective treatment strategies can be developed and applied.

Improved pediatric cancer treatments are needed because:

• Despite increases in overall survival rates, about 20% of pediatric cancer patients do not respond well to therapy and ultimately die from their diseases. 
• The number of children and adolescents diagnosed with cancer is trending slightly upward.
• Current treatments are particularly harsh on growing children. They often cause severe short- and long-term side effects, such as secondary cancers, physical and emotional health issues, developmental delays, and infertility.
• Current treatment protocols are mostly derived from therapeutic regimens that were formulated for adult cancers. Previous genomics studies revealed that childhood cancers can be genetically distinct from their adult counterparts, suggesting the need for alternate treatment approaches.

The TARGET initiative originated with two pilot projects characterizing the genomes and transcriptomes of “high-risk” subtypes of acute lymphoblastic leukemia (ALL) and neuroblastoma (NBL). The success of the two pilot project teams allowed TARGET to expand its efforts by incorporating higher resolution genomics approaches and the study of additional childhood cancers. To date, TARGET researchers have molecularly characterized subtypes of acute myeloid leukemia, osteosarcoma, and select kidney tumors, and additional subtypes of ALL and NBL.

TARGET was built as the collaborative effort of a large, diverse consortium of extramural and NCI investigators. Members of the Children’s Oncology Group (COG), a clinical trials group devoted exclusively to childhood and adolescent cancer research, comprise the majority of TARGET project teams. COG members were able to provide the TARGET teams access to clinical expertise and accrued tissue materials. TARGET researchers continue to work together within and across teams to generate, analyze, integrate, and interpret high quality genomics data. The goal of working with COG in this collaborative team science arena is to accelerate molecular discoveries and facilitate rapid translation of those findings into the clinic.

TARGET data are available to the greater research community for further investigation. For all data shared through TARGET, patient confidentiality and privacy are protected. This data sharing approach allows investigators outside the initiative to use the data, thereby increasing the chance that novel therapeutics will be developed and benefit children with cancer.