mTORC1 inhibition delays growth of neurofibromatosis type 2 schwannoma

Results. We found that treatment with the mTORC1 inhibitor rapamycin reduced the severity of NF2-related Schwann cell tumorigenesis without significant toxicity. Consistent with these results, in an NF2 patient with growing vestibular schwannomas, the rapalog sirolimus induced tumor growth arrest. Conclusions. Taken together, these results constitute definitive evidence that justifies proceeding with clinical trials using mTORC1targeted agents in selected patients with NF2 and in patients with NF2-related sporadic tumors.