Table 5. Clinical trials for NF2-associated tumors.

Drug	Phase	Target	Age (year)	End point	Status	Results
PTC299	II	VEGF	≥ 18	VS: tumor volume and/or word recognition	Stage 1 completed; study suspended	Some responses; rare but serious toxicity
Lapatinib	0, translational	EGFR/ErBb2	≥ 18	VS: tumor PK, molecular & gene mutation studies	Enrolling	Pending: preliminary evidence of good drug concentration
Lapatinib	II	EGFR/ErBb2	4 - 80	VS: 15% volume reduction	Completed	4/17 patients with > 15% volume reduction [132]
RAD001	II	mTOR	≥ 3	VS and MEN: 15% volume reduction	Stage 1 enrollment (9 patients) completed	Pending
RAD001	II	mTOR	≥ 15	VS: TTP (MRI)	Open in France, and Los Angeles	Pending
RAD001	0, translational	mTOR	≥ 18	VS and MEN: tumor PK, molecular & gene mutation analysis	In development	N/A
BEV	II	VEGF	≥ 12	VS: word recognition score	Completed enrollment	Pending
Nilotinib	II	PDGF, c-Kit	≥ 18	VS: 20% volume reduction or TTP	Enrolling	Pending
Sorafenib & nilotinib	0, translational	PDGF, VEGR, c-Kit	≥ 18	Cutaneous SWN: PK, molecular studies	Enrolling	Pending

BEV, Bevacizumab; c-Kit: Tyrosine-protein kinase Kit; MEN: Multiple endocrine neoplasia; MRI: Magnetic resonance imaging; mTOR: Mammalian target of rapamycin; NF2: Neurofibromatosis type 2; PDGF: Platelet-derived growth factor; PK: Pharmacokinetics; SWN: Schwannomas; TTP: Time to progression; VS: Vestibular schwannomas.