Supplementary Materials

Supplementary Material for:

A precision therapy against cancers driven by KIT/PDGFRA mutations

Erica K. Evans, Alexandra K. Gardino, Joseph L. Kim, Brian L. Hodous, Adam Shutes, Alison Davis, Xing Julia Zhu, Oleg Schmidt-Kittler, Doug Wilson, Kevin Wilson, Lucian DiPietro, Yulian Zhang, Natasja Brooijmans, Timothy P. LaBranche, Agnieszka Wozniak, Yemarshet K. Gebreyohannes, Patrick Schöffski, Michael C. Heinrich, Daniel J. DeAngelo, Stephen Miller, Beni Wolf, Nancy Kohl, Timothy Guzi, Nicholas Lydon, Andy Boral, Christoph Lengauer*

*Corresponding author. Email: clengauer{at}

Published 1 November 2017, Sci. Transl. Med. 9, eaao1690 (2017)
DOI: 10.1126/scitranslmed.aao1690

This PDF file includes:

  • Fig. S1. Analysis of compound binding reveals broad type I activity across various forms of KIT.
  • Fig. S2. BLU-285 inhibits KIT signaling in a KIT exon 11/17 human cell line.
  • Fig. S3. BLU-285 exhibits differential activity on PDGFRA WT and V561D and D842V mutants.
  • Fig. S4. Pharmacodynamic analysis of KIT signaling in a P815 allograft model of SM demonstrates in vivo target engagement.
  • Fig. S5. BLU-285 is well tolerated in a KIT mutant exon 11/17 PDX model of relapsed GIST.
  • Fig. S6. Histology of GIST PDX KIT mutant exon 11/17 tumors confirms tumor regression.
  • Fig. S7. Histology of GIST PDX KIT mutant exon 11 tumors was correlated with tumor size regression.
  • Fig. S8. BLU-285 is well tolerated in a KIT mutant exon 11 PDX model of primary GIST.
  • Table S1. Human kinases with more than 90% binding by BLU-285, imatinib, sunitinib, regorafenib, crenolanib, and midostaurin are listed.
  • Table S2. BLU-285 has selectivity for KIT D816V over several kinase antitargets.
  • Table S3. Antitumor activity of BLU-285 and type II inhibitors across various KIT mutant–driven models of disease highlights robust activity of BLU-285.

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