In vivo structure-activity relationship study of dorsomorphin analogues identifies selective VEGF and BMP inhibitors

Research output: Contribution to journalJournal articleResearchpeer-review

  • Jijun Hao
  • Joshua N Ho
  • Jana A Lewis
  • Kaleh A Karim
  • R Nathan Daniels
  • Patrick R Gentry
  • Corey R Hopkins
  • Craig W Lindsley
  • Charles C Hong

The therapeutic potential of small molecule signaling inhibitors is often limited by off-target effects. Recently, in a screen for compounds that perturb the zebrafish embryonic dorsoventral axis, we identified dorsomorphin, the first selective inhibitor of bone morphogenetic protein (BMP) signaling. Here we show that dorsomorphin has significant "off-target" effects against the VEGF (vascular endothelial growth factor) type-2 receptor (Flk1/KDR) and disrupts zebrafish angiogenesis. Since both BMP and VEGF signals are known to be involved in vascular development, we sought to determine whether dorsomorphin's antiangiogenic effects are due to its impact on the BMP or VEGF signals through the development of analogues that target BMP but not VEGF signaling and vice versa. In a structure-activity relationship (SAR) study of dorsomorphin analogues based primarily on their effects on live zebrafish embryos, we identified highly selective and potent BMP inhibitors as well as selective VEGF inhibitors. One of the BMP inhibitors, DMH1, which exclusively targets the BMP but not the VEGF pathway, dorsalized the embryonic axis without disrupting the angiogenic process, demonstrating that BMP signaling was not involved in the angiogenic process. This is one of the first full-scale SAR studies performed in vertebrates and demonstrates the potential of zebrafish as an attractive complementary platform for drug development that incorporates an assessment of in vivo bioactivity and selectivity in the context of a living organism.

Original languageEnglish
JournalACS chemical biology
Volume5
Issue number2
Pages (from-to)245-53
Number of pages9
ISSN1554-8929
DOIs
Publication statusPublished - 19 Feb 2010
Externally publishedYes

    Research areas

  • Animals, Bone Morphogenetic Proteins/antagonists & inhibitors, Drug Evaluation, Preclinical/methods, Embryo, Nonmammalian/drug effects, Pyrazoles/chemistry, Pyrimidines/chemistry, Quinolines/chemistry, Structure-Activity Relationship, Vascular Endothelial Growth Factor A/antagonists & inhibitors, Vascular Endothelial Growth Factor Receptor-2/antagonists & inhibitors, Zebrafish/embryology

ID: 213627140