Neoplasms study on dbGaP (6,363 participants)

phs000218 — National Cancer Institute (NCI) TARGET: Therapeutically Applicable    Research to Generate Effective Treatments

The Therapeutically Applicable Research to Generate Effective Treatments (TARGET) Initiative seeks to accelerate research in novel marker and drug development, along with understanding the molecular basis of pediatric malignancy, through identification of genomic changes associated with the following childhood cancers: <ul> <li>Acute Lymphoblastic Leukemia (ALL) - A fast-growing type of blood cancer in which too many immature white blood cells are found in the blood and bone marrow.</li> <li>Acute Myeloid Leukemia (AML), including AML Induction Failure cases (AML-IF) - Another type of blood cancer marked by too many myeloblasts, an alternate type of immature white blood cell, are found in the blood and bone marrow. Upon diagnosis, AML patients without high-risk genetic markers undergo standard chemotherapy regimen, called the primary induction phase, to eliminate most cancer cells and induce a remission state.</li> <li>Clear Cell Sarcoma of the Kidney (CCSK) - A rare type of kidney cancer, in which the inside of the cells looks clear when viewed under a microscope. Clear cell sarcoma can spread from the kidney to other organs, most commonly the bone, but also including the lungs, brain, and soft tissues of the body.</li> <li>Neuroblastoma (NBL) - Cancer of cells of the sympathetic nervous system.</li> <li>Cancer Model Systems (MDLS), including PPTP - MDLS includes acute leukemia cell lines and xenografts with matched primary and/or relapsed tumor information, NBL cell lines and xenografts with matched controls, kidney tumor cell lines, normal brain tissues, and embryonic stem cell lines that were sequenced for some TARGET disease projects.</li> <li>Osteosarcoma (OS) - A cancer of the bone that primarily affects children and adolescents.</li> <li>Rhabdoid Tumor (RT) - A rare, fast-growing cancer that usually forms in the kidney or central nervous system (the brain and spinal cord) but can also form in soft tissues in other areas of the body. Rhabdoid tumors tend to spread quickly, are hard to treat, and have a poor prognosis.</li> <li>Wilms' Tumor (WT) - A cancer of cells in the kidney that can spread to the liver, lung and lymph nodes.</li> </ul> More information can be found about each TARGET subproject by following the links on the top of this page. Together these cancers account for the majority of the more than 10,000 childhood cancer cases diagnosed in the United States each year. TARGET has employed a set of advanced and complementary genome analysis technologies, including large scale 2nd and 3rd generation genome sequencing, to strategically characterize alterations in both gene expression and in genomic structure (such as deletions and amplification) that are involved in childhood cancers. The goal of this coordinated effort has been to create a comprehensive genomic and transcriptomic profile of each cancer. Integrated analysis of the TARGET data has identified those genes that are either altered in their expression level or mapped to the chromosome regions of deletion/amplification/translocation, as these genes represent strong candidates for therapeutic targeting. To learn more about the TARGET project, which is now completed, visit the website at <a href="http://ocg.cancer.gov/programs/target">https://www.cancer.gov/ccg/research/genome-sequencing/target</a>. TARGET data is accessible at the <a href="https://gdc.cancer.gov/">NCI's Genomic Data Commons (GDC)</a> via the <a href="https://portal.gdc.cancer.gov/">GDC's Data Portal</a> and from each of the <a href="https://gdc.cancer.gov/about-data/publications#/?groups=&#x26;years=&#x26;programs=TARGET&#x26;order=desc">TARGET Publication Pages at the GDC</a> (Please see the "Supplemental Links" section of any TARGET publication's "Publication Information and Associated Data Files page" at the GDC.). Available datasets include raw sequencing data, datasets generated by the original TARGET research teams (fully annotated clinical information and higher-level/analyzed molecular characterization data - gene expression, copy number variation, epigenetics, targeted sequencing), as well as higher level data generated by the GDC. Please see the <a href="https://www.cancer.gov/ccg/research/genome-sequencing/target/using-target-data">TARGET Use and Publication Guidelines</a> for updated details on the sharing of any TARGET substudy data, including <a href="https://www.cancer.gov/ccg/research/genome-sequencing/target/using-target-data/citing">how to cite TARGET</a>.