Table 2. FDA approved or late–stage drugs selected for cell culture screening.
| Compound | Targetsa | Inclusion Criteriab |
|---|---|---|
| LY2157299 | TGF-beta/Smad | 2,3[29] |
| Ganetespib (STA-9090) | HSP (e.g HSP90) | 2,3[30] |
| Panobinostat (LBH589) | HDAC | 2,3[27,31] |
| Vorinostat (SAHA, MK0683) | Autophagy, HDAC | 2,3[27,31] |
| AR-42 | HDAC | 1,2,3 [27,31] |
| Lapatinib (GW-572016) Ditosylate | HER2, EGFR | 1,2,3[24,32] |
| Axitinib | VEGFR, PDGFR, c-Kit | 1,2,3[21,25,28,33] |
| AZD2014 | mTOR | 1,2,3[11,15,23,34,35] |
| OSU-03012 (AR-12) | PDK-1 | 2[21,36] |
| Perifosine (KRX-0401) | AKT | 2,3 [14,36–38] |
| Everolimus (RAD001) | mTOR | 1,2,3[11,15,23,34,35] |
| GSK2126458 (GSK458) | PI3K, mTOR | 2,3[15,36] |
| GDC-0980 (RG7422) | PI3K, mTOR | 2,3 [15,36] |
| CUDC-907 | PI3K, HDAC | 2,3 [13,16,36,39] |
| GDC-0941 | PI3K | 2,3[16,36] |
| Selumetinib (AZD6244) | MEK | 2,3[24] |
| Trametinib (GSK1120212) | MEK | 2,3[24] |
| Vismodegib (GDC-0449) | Hedgehog/Smoothened | 2,3[38,40,41] |
| Bortezomib (PS-341) | Proteasome | 2,3[42] |
aTGF-beta, transforming growth factor beta; HSP, heat shock protein; HDAC, histone deacetylase; HER2, human epidermal growth factor receptor 2; EGFR, epidermal growth factor receptor; VEGFR, vascular endothelial growth factor receptor; PDGFR, platelet-derived growth factor receptor; c-Kit, tyrosine-protein kinase Kit; mTOR, mammalian target of rapamycin; PDK-1, pyruvate dehydrogenase kinase 1; AKT, protein kinase B; PI3K, phosphoinositide 3-kinase; MEK, MAPK/ERK kinase.
b(1) biological activity in clinical trials in NF2 patients, (2) activity against targets in a key pathway regulated by merlin, (3) FDA-approved or in the late stages of clinical development.