American Association for Cancer Research

Therapeutically Targeting the Epigenome in Aggressive Pediatric Cancers


Malignant rhabdoid tumors and atypical teratoid/rhabdoid tumors (RT) occur primarily in children under 2 years old. Despite intensive treatment, over 80 percent of patients with RT die within about a year of diagnosis. These tumors are unique because unlike most cancers, they have not accumulated alterations in their DNA sequence compared to the patient's normal cells. Instead, this cancer often results from the loss of a specific gene, SNF5, which affects the physical structure or packaging of the DNA. This alteration to the DNA packaging and other epigenetic changes can affect the expression of many genes that cancer cells use to grow. This is significant because epigenetic changes are not permanent and can potentially be reversed with the right therapies. Roberts has developed a model system to examine different types of epigenetic pathways and to determine if there are therapies that can reverse the cancer-causing effects of losing the SNF5 gene. This could lead to improved treatment for RT and could also have much broader implications since epigenetic changes play an important role in causing almost every type of cancer. 


Charles M. Roberts, M.D., Ph.D., assistant professor in the Department of Pediatrics at Harvard Medical School; assistant professor of pediatric oncology at the Dana-Farber Cancer Institute

Updated: Dec. 3, 2009