Everolimus in combination with vandetanib in children, adolescents, and young adults: a phase I study.

ABSTRACT:

Background

Combined use of inhibitors of mammalian target of rapamycin (mTOR) and vascular endothelial growth factor (VEGF-2) receptors is a potential strategy to overcome resistance to either class of drugs when used alone.

Patients and methods

We designed a phase 1 trial to test the drug combination of a multikinase VEGF receptor 2 inhibitor, vandetanib, and an mTOR inhibitor, everolimus, in a pediatric and young adult patient cohort with advanced cancers. Exceptional responders were probed for tumor mutational profile to explore possible molecular mechanisms of response.

Results

Among 21 enrolled patients, clinical benefit was observed in 38% (one patient with partial response and eight patients with stable disease) with a median progression-free survival of 3.3 mo

SUBMITTER: Phadnis S 

PROVIDER: S-EPMC10774869 | biostudies-literature | 2023 Dec

REPOSITORIES: biostudies-literature