Table 2.
Soft-tissue sarcoma targets, therapeutics, and clinical status
| Target(s) | Therapeutic | Status | Trial ID | |
|---|---|---|---|---|
| LPS | CDK4/6 | Palbociclib (PD0332991) | Phase II trial completed (2017) | NCT01209598 |
| LPS | VEGFR and PDGFR | Pazopanib | Phase II trial completed (2017) | NCT01506596 |
| LPS (MLS) | NY-ESO1 | CAR T cells | Phase II trial ongoing | NCT02992743 |
| LPS (MLS), SS | Class I MHC expression | Recombinant IFNγ | Pilot study ongoing | NCT01957709 |
| LPS (DDLS), LMS | mTOR and CDK4/6 | Everolimus + ribociclib | Phase II trial ongoing | NCT03114527 |
| LPS, LMS | PDL1 and DNA repair | Avelumab (PDL1 mAb) + trabectedin | Phase II trial ongoing | NCT030743 |
| LPS, ES, AS (UPS) | PD1 and mTOR | Nivolumab (PD1 mAb) + ABI009 (mTOR inhibitor) | Phase II trial ongoing | NCT0319017 |
| AS | VEGF | Bevacizumab (VEGF mAb) | Phase II trial ongoing | NCT00288015 |
| LMS, SS | EGFR | Anlotinib (AL3818) | Phase III trial ongoing | NCT03016819 |
| RMS | NY-ESO1, MAGEA4, PRAME, survivin, and SSX | TAA-specific CTLs | Phase I trial ongoing | NCT02239861 |
| RMS, ES | Immunomodulated lysis | Recombinant vaccinia GM-CSF (JX594) | Phase I trial completed | NCT01169584 |
| RMS, SS | CD56 and tubulin | Lorvotuzumab mertansine | Phase II trial ongoing | NCT02452554 |
| ES | EWS-FLI1 | TK216 | Phase I trial ongoing | NCT02657005 |
| SS | G6PD | DHEA | Phase II trial ongoing | NCT02683148 |
| SS | mTOR, c-Kit, and PDGFR | Everolimus + imatinib mesylate | Phase II trial completed | NCT01281865 |
| SS | NY-ESO1-expressing tumor cells | Autologous dendritic cells loaded with allogeneic tumor lysate expressing NY-ESO1 | Phase I/II ongoing | NCT01883518 |
| Advanced STS postchemotherapy | VEGFR1 and VEGFR2, VEGFR3, PDGFR, and c-Kit | Pazopanib | FDA-approved (2012) | — |
| Several | Immunomodulated lysis | HSV1716 | Phase I trial ongoing | NCT00931931 |
| Several | Tubulin and mitotic spindle | Eribulin | FDA-approved (2015) | — |
| Several | DNA repair | Trabectedin | FDA-approved (2016) | — |
| Several | PD1 and CTLA4 | Nivolumab ± ipilimumab (CTLA4 mAb) | Phase II trial ongoing | NCT02500797 |
| Several | mTOR | Ridaforolimus | Phase II trial completed (2015) | NCT00112372 |
| Several | VEGFR, PDGFR, and DNA replication | Pazopanib + topotecan | Phase II trial ongoing | NCT02357810 |
| Several | Topoisomerase 2 and PDGFR | Dexrazoxane + doxorubicin + olaratumab | Phase II trial ongoing | NCT025843 |
| Several | Raf, VEGFR1, VEGFR2, VEGFR3, PDGFR B, and c-Kit | Sorafenib | Phase II trial completed | NCT00217620 |
| Several | PDL1 and NY-ESO1 | Atezolizumab (PDL1 mAb) + CMB305 | Phase II trial ongoing | NCT026099 |
| Several | Histone-lysine methyltransferase EZH2 | Tazemetostat | Phase II trial ongoing | NCT02601950 |
Abbreviations: LPS, liposarcoma; MLS, myxoid liposarcoma; SS, synovial sarcoma; DDLS, dedifferentiated liposarcoma; LMS, leiomyosarcoma; mAb, monoclonal antibody; ES, Ewing’s sarcoma; AS, angiosarcoma; UPS, undifferentiated pleomorphic sarcoma; RMS, rhabdomyosarcoma.