“Deregulated mTOR in desmoid-type fibromatosis: Identification and validation of a new therapeutic agent.” Year 1 of 1

This is a pilot study examining the role of Rapamycin in the treatment of children and young adults with DT deemed likely to recur following resection. Rapamycin directly targets and inhibits mTOR, a growth promoting pathway that may be critical in the development and growth of DT. The mTOR cell proliferation/survival pathway was shown to be activated and essential for tumorigenesis when the APC gene is mutated (affecting the Beta catenin signaling pathway). It is an oral drug being evaluated as an anti-neoplastic agent in a variety of cancer types. Skapek et al plan to determine how effectively rapamycin blocks mTOR activation in desmoid tumors and whether mTOR inhibition blocks desmoid tumor blood vessel growth. Skapek et al plan to determine how effectively rapamycin blocks mTOR activation in desmoid tumors . If Rapamycin is found to be active, they intend to extend this pilot study to a national collaborative study run through the Children’s Oncology Group (COG).