Atypical teratoid rhabdoid tumor (ATRT) is a very rare and fast-growing cancerous tumor of the brain and spinal cord. Most ATRTs are caused by changes in a gene known as INI1 (or SMARCB1). This gene normally makes proteins that can stop tumors from growing, but in ATRTs, INI1 does not function properly and, as a result, tumor growth is uncontrolled. The disease commonly affects children age 3 or younger but can also affect older children and adults. This gene defect may be inherited, and your doctor may discuss genetic testing.

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ATRT Translational Research Program

In collaboration with the Hope4ATRT Foundation, the ATRT Translational Research Program, within Beat Childhood Cancer, will focus on identifying children with ATRT to support them through clinical treatment trials, and provide resources for the affected children and their caregivers. The Out of Zion Award from the Hope4ATRT Foundation will offer assistance for children with ATRT to enroll in The Signature Study: Molecular Analysis of Pediatric Tumors with Establishment of Tumor Models in an Exploratory Biology Study. This study will allow the tumor to undergo complete genomic analysis to better understand the biology of each child’s tumor by identifying the mutations or pathways active in driving the tumor. In addition, the tumor cells will be grown in culture and xenograft models for high throughput drug screening.

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