Atypical Teratoid/Rhabdoid Tumors (ATRT) are aggressive malignant neoplasms that most commonly occur in the Central Nervous System (CNS) in children less than 3 years of age.
While ATRTs most commonly occur in the CNS, biologically and morphologically similar tumors (called rhabdoid tumors) also occur in the kidney and soft tissues.
Despite intensive chemoradiotherapy and surgery, the outcomes for patients with this disease remain dismal, with approximately 75 percent of patients dying from their disease, at a median of 10 months after diagnosis. Furthermore, CNS radiation leads to severe neurocognitive deficits that the majority of young children who do survive are significantly disabled.
Novel treatments are clearly necessary for this disease. Together with our collaborators, Dr. Ted Laetsch and Dr. David Boothman, we are exploring a novel targeted therapy for ATRT. This work is supported by a REACH award from the Alex’s Lemonade Stand Foundation.