This is a pilot study examining the role of sirolimus in the treatment of children and young adults with desmoid tumor that is deemed likely to recur following resection. Sirolimus directly targets and inhibits a pathway that may be critical for the development and growth of desmoid tumor. Sirolimus and several related drugs have been used in children and adults to suppress the immune system following transplant and, more recently, as an anti-neoplastic agent in a variety of cancer types. Sirolimus is attractive as a potential drug for desmoid tumor because it is well-tolerated in children and young adults and it can be given orally. Sirolimus acts by blocking a growth promoting pathway called the mTOR pathway. Dr. Weiss will determine whether sirolimus can decrease tumor size, decrease tumor-associated pain, and block the mTOR pathway. The data obtained in this pilot study will be used to leverage additional resources to support a national collaborative study, such as one run through the Children’s Oncology Group (COG), in children and young adults.
*The above grant awards are subject to a mutually satisfactory grant agreement between the parties.