Pharmacologic targeting of a stem/progenitor population in vivo is associated with enhanced bone regeneration in mice

J Clin Invest. 2008 Feb;118(2):491-504. doi: 10.1172/JCI33102.

Abstract

Drug targeting of adult stem cells has been proposed as a strategy for regenerative medicine, but very few drugs are known to target stem cell populations in vivo. Mesenchymal stem/progenitor cells (MSCs) are a multipotent population of cells that can differentiate into muscle, bone, fat, and other cell types in context-specific manners. Bortezomib (Bzb) is a clinically available proteasome inhibitor used in the treatment of multiple myeloma. Here, we show that Bzb induces MSCs to preferentially undergo osteoblastic differentiation, in part by modulation of the bone-specifying transcription factor runt-related transcription factor 2 (Runx-2) in mice. Mice implanted with MSCs showed increased ectopic ossicle and bone formation when recipients received low doses of Bzb. Furthermore, this treatment increased bone formation and rescued bone loss in a mouse model of osteoporosis. Thus, we show that a tissue-resident adult stem cell population in vivo can be pharmacologically modified to promote a regenerative function in adult animals.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Bone Regeneration*
  • Boronic Acids / pharmacology*
  • Bortezomib
  • Cell Differentiation
  • Female
  • Mesenchymal Stem Cell Transplantation
  • Mesenchymal Stem Cells / cytology
  • Mesenchymal Stem Cells / drug effects*
  • Mice
  • Mice, Inbred C57BL
  • Osteoblasts / cytology*
  • Protease Inhibitors / pharmacology*
  • Pyrazines / pharmacology*

Substances

  • Boronic Acids
  • Protease Inhibitors
  • Pyrazines
  • Bortezomib