What Is AT/RT Cancer?

adult and childhood brain tumors

Mark Kieran, MD, PhD

An atypical teratoid rhabdoid tumor (AT/RT) is a very rare and fast-growing tumor of the central nervous system. AT/RT is part of a larger group of malignant tumors called rhabdoid tumors, which are found outside the brain, in the kidneys, liver and other locations. AT/RT is most commonly found in the cerebellum and brain stem, which control most of the body’s basic functions.

Since AT/RT grows very rapidly, symptoms can develop quickly over days or weeks. Increased head size often occurs in infants due to swelling inside the brain. Other common symptoms of AT/RT include headaches, especially upon waking in the morning, fatigue, lethargy, nausea, vomiting, and trouble with balance and coordination.

Depending on the stage of disease, therapy for AT/RT can be used to either treat the tumor directly or to mitigate complications and side effects. Treatment varies depending the patient’s disease and age, but it can include neurosurgery, radiation therapy, chemotherapy , and stem cell transplant

About 100 new cases of AT/RT occur each year, typically in children under three years of age, though it can also occur in adults. AT/RT represents only 1 to 2 percent of childhood brain tumors and more than 90 percent of AT/RT cases are associated with a genetic mutation in the INI1 gene, which can occur spontaneously or may be inherited.

Despite the fast-growing nature of the disease, treatment is improving with research and clinical trials.  A promising trial led by Mark Kieran, MD, PhD, director of Pediatric Medical Neuro-Oncology at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, showed a treatment for muscle cancers could also be effective against AT/RT.  In addition, it has become clear that a mutation of the gene family that underlies AT/RT is also tied to at least 20 percent of all cancers, including adult cancers of lung, brain, pancreas, ovary, liver, stomach, colon, kidney, and breast cancer.  Recent discoveries by Charles Roberts, MD, PhD, director of Dana-Farber’s Research Program in Solid Tumors, have also identified new drug targets for AT/RT. Currently, Susan Chi, MD, is leading a Phase I clinical trial, testing a Novartis drug for children with AT/RT and adults with forms of leukemia and solid tumors.

Learn more about the latest treatments for AT/RT:

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