Initial in vivo testing of a multitarget kinase inhibitor, regorafenib, by the Pediatric Preclinical Testing Consortium

Pediatr Blood Cancer. 2020 Jun;67(6):e28222. doi: 10.1002/pbc.28222. Epub 2020 Mar 24.


Background: Regorafenib is a small molecule multikinase inhibitor that inhibits multiple kinases including BRAF, KIT, PDGFRB, RAF, RET, and VEGFR1-3.

Procedures: The in vivo anticancer effects of regorafenib were assessed in a panel of six osteosarcoma models, three rhabdomyosarcoma models, and one Ewing sarcoma model.

Results: Regorafenib induced modest inhibition of tumor growth in the models evaluated.

Conclusion: The overall pattern of response to regorafenib appears similar to that of the kinase inhibitor sorafenib, with pronounced slowing of tumor growth in some models, limited to the period of agent administration, being the primary treatment effect.

Keywords: Ewing sarcoma; multitarget kinase inhibitor; osteosarcoma; patient derived xenograft; pediatric preclinical testing consortium; rhabdomyosarcoma.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis
  • Bone Neoplasms / drug therapy*
  • Bone Neoplasms / enzymology
  • Bone Neoplasms / pathology
  • Cell Proliferation
  • Child
  • Female
  • Humans
  • Mice
  • Mice, SCID
  • Osteosarcoma / drug therapy*
  • Osteosarcoma / enzymology
  • Osteosarcoma / pathology
  • Phenylurea Compounds / pharmacology*
  • Protein Kinase Inhibitors / pharmacology*
  • Pyridines / pharmacology*
  • Rhabdomyosarcoma / drug therapy*
  • Rhabdomyosarcoma / enzymology
  • Rhabdomyosarcoma / pathology
  • Sarcoma, Ewing / drug therapy*
  • Sarcoma, Ewing / enzymology
  • Sarcoma, Ewing / pathology
  • Tumor Cells, Cultured
  • Xenograft Model Antitumor Assays


  • Phenylurea Compounds
  • Protein Kinase Inhibitors
  • Pyridines
  • regorafenib