A Phase II Study of Everolimus in Patients with Cancers Containing TSC1, TSC2, or MTOR Mutations


Full Title

A Phase II Trial of Everolimus for Cancer Patients with Inactivating Mutations in TSC1 or TSC2 or Activating MTOR Mutations


Everolimus is a drug approved to treat certain cancers, including some types of breast and kidney cancers and neuroendocrine tumors. In this study, researchers are evaluating the safety and effectiveness of everolimus treatment in patients with any type of advanced cancer containing mutations in genes called TSC1, TSC2, or MTOR. These mutations are believed to promote tumor growth, and everolimus blocks the function of these mutated genes.

Everolimus is taken orally (by mouth) once a day.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have a metastatic, inoperable, or recurrent cancer that contains a mutation in TSC1, TSC2, or MTOR.
  • At least 2 weeks must have passed since the completion of any prior chemotherapy and 3 weeks since prior radiation therapy and entry into the study.
  • Prior treatment with a PI3 kinase inhibitor or an MTOR inhibitor is not permitted.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for people age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. Gopa Iyer 646-422-4362.