Table 1.
Recent clinical trials assessing targeted therapy for NF2-associated vestibular schwannomas with intended targets, phases of therapy, endpoints, and references.
| Drug Name | Therapy Target | Phase Trial/Number of Patients Enrolled | Notable Endpoints | Further Studies Planned | References |
|---|---|---|---|---|---|
| Erlotinib | Epidermal Growth Factor Receptor (EGFR) | Phase 2–10 Patients | 3/10 with minimal radiographic response | No | Plotkin et al., 2010 [115] |
| Everolimus | Mammalian Target of Rapamycin (mTOR) | Phase 2–9 Patients | No radiographic or hearing responses. Prolonged time to progression | No | Karajannis et al., 2014 [110]; Goutagny et al., 2015 [111] |
| Lapatinib | EGFR and Erb2 | Phase 2–21 patients (17 evaluable) |
4/17 with >15% size reduction 4/13 with improved hearing |
Unclear | Karajannis et al., 2012 [114] |
| Bevacizumab | Vascular Endothelial Growth Factor Receptor (VEGFR) | Phase 2, multiple studies (>100 patients reported) |
RR in 41% Hearing improvement in 20% TTP improvement |
No | Lu et al., 2019 [116]; Plotkin et al., 2012 [117]; 2019 [118]; Blakeley et al., 2016 [119] |
| Crizotinib/Brigatinib | Focal Adhesion Kinase (FAK1) | Phase 2, ongoing | Volumetric response as primary aim, hearing secondary | Ongoing | N/A |