Skip to main content

Table 3 Novel agents for GIST

From: Recent advances and novel agents for gastrointestinal stromal tumor (GIST)

Study Drug Class of Drug Disease Dosage Clinical Trial No. of patients Response Reference
Everolimus mTOR inhibitor TKI resistant Everolimus 2.5 mg/day with Imatinib Phase 1/2 58 PFS 29% [57]
Everolimus mTOR inhibitor Refractory GIST 10 mg/day Phase 1/2 15 ORR27% [59]
Sirolimus mTOR inhibitor TKI resistant with PDGFRA-D842V. Sirolimus (2–3 mg/day) with a TKI. Retrospective 3 Signs of antitumor activity. [60]
Ganetespib Hsp90 inhibitor Following failure of prior therapy 200 mg/m2 IV qweek for 3 wks of a 28 day cycle. Phase 2 26 SD 52% [61]
Retaspimycin (IPI 504) Hsp90 inhibitor Following failure of TKIs 400 mg/m2 weekly for 2 doses in 21‒day cycles Phase 3 47 Too toxic [62]
Perifosine Akt pathway inhibitor Imatinib-resistant 2 doses of perifosine - 100 mg daily or 900 mg qweekly with daily imatnib Phase 2 41 Minimal activity [63]
  1. Abbreviations: SD stable disease; PFS progression free survival; ORR overall response rate.