Table 1.
Selected examples of ongoing, or recently completed, trials investigating novel therapies for metastatic ES.
| Drug Class | Example Drug | Number of Patients | Clinical Trial | Results | Current Status | Presented By |
|---|---|---|---|---|---|---|
| EWSR1-FLI1 target agents | TK216 RP2D and expansion |
n = 15 | Phase I/II RP2D and expansion |
DCR 47% | Proceeding in further trials | Ludwig et al., ESMO 2020 |
| IGF-1R inhibitors | Ganitumab (added to backbone chemotherapy, metastatic |
n = 150 | Phase III, AEWS1221 |
No benefit in EFS or OS | Correlative studies | DuBois et al., CTOS 2019 |
| mTOR | Everolimus (Combination with lenvatinib) |
n = 1 | Phase Ib | Phase II ongoing | Dela Cruz, ASCO2020 | |
| Microtubuli inhibitors | Eribulin | Phase II NCT03441360 | Ongoing | |||
| CDK4/6 inhibitors | Palbociclib, abemaciclib |
Are being investigated in early trials | ||||
| PARP inhibitors | Talazoparib (combination with irinotecan and temozolamide) |
n = 22 (both arms) |
Phase I | DCR 73% | Federico et al., EJC 2020 | |
| Multi-targeted tyrosine kinase inhibitors | Cabozantinib | n = 45 | Phase II, CABONE | DCR 60% | Italiano et al., ESMO 2018 | |
| Regorafenib | n = 23 | Phase II, Regobone | DCR 70% | Duffaud et al., ESMO 2020 | ||
| Regorafenib | Phase II, SARC024 | Ongoing | ||||
| Pazopanib (combination with irinotecan and temozolamide) |
n = 7 | PAZIT | SD 57% | Doses investigated not tolerable | Vo et al., ASCO 2020 |
CDK4/6: cyclin-dependent kinase 4/6, DCR: disease control rate, EFS: event-free survival, ES: Ewing sarcoma, IGF-1R: insulin-like growth factor 1 receptor, mTOR: mammalian target of rapamycin, OS: overall Survival, PARP: poly (ADP-ribose) polymerase, RP2D: recommended phase II dose.