Promoting in vivo remyelination with small molecules: a neuroreparative pharmacological treatment for Multiple Sclerosis

Sci Rep. 2017 Mar 3:7:43545. doi: 10.1038/srep43545.

Abstract

Multiple Sclerosis (MS) is a neurodegenerative disease where immune-driven demyelination occurs with inefficient remyelination, but therapies are limited, especially those to enhance repair. Here, we show that the dual phosphodiesterase (PDE)7- glycogen synthase kinase (GSK)3 inhibitor, VP3.15, a heterocyclic small molecule with good pharmacokinetic properties and safety profile, improves in vivo remyelination in mouse and increases both adult mouse and adult human oligodendrocyte progenitor cell (OPC) differentiation, in addition to its immune regulatory action. The dual inhibition is synergistic, as increasing intracellular levels of cAMP by cyclic nucleotide PDE inhibition both suppresses the immune response and increases remyelination, and in addition, inhibition of GSK3 limits experimental autoimmune encephalomyelitis in mice. This combination of an advantageous effect on the immune response and an enhancement of repair, plus demonstration of its activity on adult human OPCs, leads us to propose dual PDE7-GSK3 inhibition, and specifically VP3.15, as a neuroprotective and neuroreparative disease-modifying treatment for MS.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Differentiation / drug effects
  • Cell Proliferation / drug effects
  • Cell Survival / drug effects
  • Cells, Cultured
  • Cyclic Nucleotide Phosphodiesterases, Type 7 / antagonists & inhibitors
  • Disease Models, Animal
  • Encephalomyelitis, Autoimmune, Experimental
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacology*
  • Enzyme Inhibitors / therapeutic use
  • Glycogen Synthase Kinase 3 / antagonists & inhibitors
  • Humans
  • Male
  • Mice
  • Multiple Sclerosis / drug therapy
  • Multiple Sclerosis / metabolism*
  • Multiple Sclerosis / pathology
  • Myelin Sheath / drug effects*
  • Myelin Sheath / metabolism*
  • Myelin Sheath / pathology
  • Oligodendroglia / cytology
  • Oligodendroglia / drug effects
  • Oligodendroglia / metabolism
  • Remyelination / drug effects*

Substances

  • Enzyme Inhibitors
  • Glycogen Synthase Kinase 3
  • Cyclic Nucleotide Phosphodiesterases, Type 7