A phase II study of everolimus in patients with advanced solid malignancies with <i>TSC1, TSC2, NF1, NF2</i> or <i>STK11</i> mutations.

ABSTRACT:

Background

Activation of the mTOR pathway has been implicated in the development of several malignancies and alterations in TSC1, TSC2, STK11 and NF1, can lead to the dysregulation of this pathway. Furthermore, mutations in TSC1 and NF2 are known to confer sensitivity to everolimus-an mTOR inhibitor. Based on these data, a single-arm, open label, single-institution phase II basket study was designed to assess the activity of everolimus in patients with solid malignancies whose tumors harbored mutations in TSC1, TSC2, NF1, NF2, or STK11.

Methods

A total of 12 patients with histologically confirmed diagnosis of advanced solid tumors (metastatic, recurrent, or unresectable) with mutations in TSC1, TSC2, NF1, NF2 or STK11 g

SUBMITTER: Devarakonda S 

PROVIDER: S-EPMC8339787 | biostudies-literature | 2021 Jul

REPOSITORIES: biostudies-literature