TARGET: Therapeutically Applicable Research to Generate Effective Treatments

The Therapeutically Applicable Research to Generate Effective Treatment (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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Distribution of MPAL subtypes by age
September 12, 2018

The genetic basis and cell of origin of mixed phenotype acute leukaemia.

Mixed phenotype acute leukaemia (MPAL) is a high-risk subtype of leukaemia with myeloid and lymphoid features, limited genetic characterization, and a lack of consensus regarding appropriate therapy. Here we show that the two principal subtypes of MPAL, T/myeloid (T/M) and B/myeloid (B/M), are...

Image of TFAP4, a synthetic lethal candidate and master regulator of MYCN-amplified neuroblastoma
June 07, 2018

Transcription factor activating protein 4 is synthetically lethal and a master regulator of MYCN-amplified neuroblastoma.

Despite the identification of MYCN amplification as an adverse prognostic marker in neuroblastoma, MYCN inhibitors have yet to be developed. Here, by integrating evidence from a whole-genome shRNA library screen and the computational inference of master regulator proteins, we identify...

Colorized scanning electron micrograph of a natural killer cell from a human donor.
May 21, 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...

Protein-protein interactions generated using Preppi (v1.0)
May 01, 2018

Cross-Cohort Analysis Identifies a TEAD4-MYCN Positive Feedback Loop as the Core Regulatory Element of High-Risk Neuroblastoma.

High-risk neuroblastomas show a paucity of recurrent somatic mutations at diagnosis. As a result, the molecular basis for this aggressive phenotype remains elusive. Recent progress in regulatory network analysis helped us elucidate disease-driving mechanisms downstream of genomic alterations,...

Figure showing some of the mutated genes by tumor type
March 15, 2018

Pan-cancer genome and transcriptome analyses of 1,699 paediatric leukaemias and solid tumours.

Analysis of molecular aberrations across multiple cancer types, known as pan-cancer analysis, identifies commonalities and differences in key biological processes that are dysregulated in cancer cells from diverse lineages. Pan-cancer analyses have been performed for adult but not paediatric...

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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
Last updated: January 03, 2019

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