TARGET: Therapeutically Applicable Research to Generate Effective Treatments

The Therapeutically Applicable Research to Generate Effective Treatments (TARGET) program applies a comprehensive genomic approach to determine molecular changes that drive childhood cancers. The goal of the program is to use data to guide the development of effective, less toxic therapies. TARGET is organized into a collaborative network of disease-specific project teams.

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News & Publications

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Graphical abstract from Chun, et al (2019).

November 19, 2019

Identification and Analyses of Extra-Cranial and Cranial Rhabdoid Tumor Molecular Subgroups Reveal Tumors with Cytotoxic T Cell Infiltration.

Extra-cranial malignant rhabdoid tumors (MRTs) and cranial atypical teratoid RTs (ATRTs) are heterogeneous pediatric cancers driven primarily by SMARCB1 loss. To understand the genome-wide molecular relationships between MRTs and ATRTs, we analyze multi-omics data from 140 MRTs and 161 ATRTs. We...

March 15, 2019

Predicting Complete Remission of Acute Myeloid Leukemia: Machine Learning Applied to Gene Expression.

Machine learning (ML) is a useful tool for advancing our understanding of the patterns and significance of biomedical data. Given the growing trend on the application of ML techniques in precision medicine, here we present an ML technique which predicts the likelihood of complete remission (CR)...

February 13, 2019

Genetic mechanisms of primary chemotherapy resistance in pediatric acute myeloid leukemia.

Acute myeloid leukemias (AML) are characterized by mutations of tumor suppressor and oncogenes, involving distinct genes in adults and children. While certain mutations have been associated with the increased risk of AML relapse, the genomic landscape of primarychemotherapy-resistant AML is not...

December 13, 2018

A unique subset of low-risk Wilms tumors is characterized by loss of function of TRIM28 (KAP1), a gene critical in early renal development: A Children's Oncology Group study.

This study explores the genomic alterations that contribute to the formation of a unique subset of low-risk, epithelial differentiated, favorable histology Wilms tumors (WT), tumors that have been characterized by their expression of post-induction renal developmental genes (Subset1 WT). We...

Colorized scanning electron micrograph of a natural killer cell from a human donor.

November 15, 2018

Clinically Relevant Cytotoxic Immune Cell Signatures and Clonal Expansion of T Cell Receptors in High-risk MYCN-not-amplified Human Neuroblastoma.

Purpose: High-risk neuroblastoma is an aggressive disease. DNA sequencing studies have revealed a paucity of actionable genomic alterations and a low mutation burden, posing challenges to develop effective novel therapies. We used RNA sequencing (RNA-seq) to investigate the...

Projects

Acute Lymphoblastic Leukemia

The TARGET Acute Lymphoblastic Leukemia projects elucidate comprehensive molecular characterization to determine the genetic changes that drive the initiation and progression of hard-to-treat childhood cancers. Acute lymphoblastic leukemia (ALL) is a cancer of white blood cells, the cells in the body that normally fight infection.

View Acute Lymphoblastic Leukemia Project Page

Acute Myeloid Leukemia

The TARGET Acute Myeloid Leukemia projects elucidate comprehensive molecular characterization to determine the genetic changes that drive the initiation and progression of high-risk or hard-to-treat childhood cancers. Acute myeloid leukemia (AML) is a cancer that originates in the bone marrow from immature white blood cells known as myeloblasts. About 25% of all children with leukemia have AML.

View Acute Myeloid Leukemia Project Page

Kidney Tumors

The TARGET Kidney Tumor projects elucidate comprehensive molecular characterization to determine the genetic changes that drive the initiation and progression of high-risk or hard-to-treat childhood cancers. Pediatric kidney tumors fall into four primary categories: Wilms tumors (~85% of all cases), clear cell sarcomas of the kidney (~5%), congenital mesoblastic nephromas (~4%), and rhabdoid tumors of the kidney (~3%). The TARGET initiative is investigating three of these tumor types.

View Kidney Tumors Project Page

Neuroblastoma

The TARGET Neuroblastoma projects elucidate comprehensive molecular characterization to determine the genetic changes that drive the initiation and progression of high-risk or hard-to-treat childhood cancers. Neuroblastoma (NBL) is a cancer that arises in immature nerve cells of the sympathetic nervous system, primarily affecting infants and children.

View Neuroblastoma Project Page

Osteosarcoma

The TARGET Osteosarcoma project elucidates comprehensive molecular characterization to determine the genetic changes that drive the initiation and progression of high-risk or hard-to-treat childhood cancers. Osteosarcoma (OS) is the most common type of bone cancer in children and adolescents.

View Osteosarcoma Project Page

Pan-cancer Model Systems

The TARGET Pan-cancer Model Systems (MDLS) were developed to study the mechanisms of tumor development and response to treatments of high-risk or hard-to-treat childhood cancers. MDLS includes acute leukemia cell lines and xenografts with matched primary and/or relapsed tumor information, neuroblastoma 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.

View Pan-cancer Model Systems Project Page

TARGET Analysis Working Group

The TARGET Analysis Working Group uses comprehensive molecular characterization to determine the genetic changes that drive the initiation and progression of high-risk or hard-to-treat childhood cancers. Analysis of molecular aberrations across multiple cancer types, known as pan-cancer analysis, identifies commonalities and differences in key biological processes dysregulated in cancer cells.

View TARGET Analysis Working Group Project Page

Last updated: July 05, 2019