Table 2.

Novel therapies for NF2 associated tumors – recently completed or published studies and ongoing clinical trials

Drug (ClinicalTrials. gov identifier)	Mode of action	Trial design	Age [years]	n	Results
Prospective therapeutic studies for NF2 patients with progressive VS
Lapatinib [55]*	Dual EGFR/ErbB2 inhibitor	Phase 2 (two-stage) Single arm Open label	>3	17	Volumetric response 24%, hearing response 31%
Everolimus [56]*	mTORC1 inhibitor	Phase 2 (two-stage) Single arm Open label	>3	9	No objective hearing or volumetric responses
Bevacizumab (NCT01207687)	VEGF-A inhibitor	Phase 2 Single arm Open label	≥12	14	Enrollment completed, results pending
Bevacizumab (NCT01767792)	VEGF-A inhibitor	Phase 2 (two-stage) Single arm Open label	12–30	14–22	Study ongoing
Axitinib (NCT02129647)	Multi-kinase inhibitor (VEGFR2, PDGFR, c-kit)	Phase 2 (two-stage) Single arm Open label	≥18	9–17	Study ongoing
Phase 0 (pharmacokinetic/pharmacodynamic, non-therapeutic) studies for NF2 VS/meningioma
Lapatinib (NCT00863122)	Dual EGFR/ErbB2 inhibitor	Phase 0	≥18		Enrollment completed, results pending
Everolimus (NCT01880749)	mTORC1 inhibitor	Phase 0	≥18		Study ongoing

EGFR = endothelial growth factor receptor; mTORC1 = mammalian target of rapamycin complex; PDGFR = latelet derived growth factor; c-kit = cellular homolog of feline sarcoma viral oncogene v-kit; VEGF = vascular endothelial growth factor; HDAC = histone deacetylase