Development of potential iron chelators for the treatment of Friedreich's ataxia: ligands that mobilize mitochondrial iron

Biochim Biophys Acta. 2001 May 31;1536(2-3):133-40. doi: 10.1016/s0925-4439(01)00041-2.

Abstract

Friedreich's ataxia (FA) is a crippling neurodegenerative disease that is due to iron (Fe) overload within the mitochondrion. One therapeutic intervention may be the development of a chelator that could remove mitochondrial Fe. We have implemented the only well characterized model of mammalian mitochondrial Fe overload to examine the Fe chelation efficacy of novel chelators of the 2-pyridylcarboxaldehyde isonicotinoyl hydrazone (PCIH) class. In this model we utilize reticulocytes treated with the haem synthesis inhibitor succinylacetone which results in mitochondrial Fe-loading. Our experiments demonstrate that in contrast to desferrioxamine, several of the PCIH analogues show very high activity at mobilizing (59)Fe from (59)Fe-loaded reticulocytes. Further studies on these ligands in animals are clearly warranted considering their potential to treat FA.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Drug Design
  • Friedreich Ataxia / chemically induced
  • Friedreich Ataxia / drug therapy*
  • Friedreich Ataxia / metabolism
  • Iron / metabolism
  • Iron / pharmacology
  • Iron Chelating Agents / chemical synthesis*
  • Iron Chelating Agents / pharmacology
  • Iron Radioisotopes
  • Isoniazid / analogs & derivatives*
  • Isoniazid / chemical synthesis*
  • Isoniazid / pharmacology
  • Ligands
  • Mice
  • Mitochondria / drug effects
  • Mitochondria / metabolism
  • Molecular Structure
  • Phenylhydrazines
  • Pyridoxal / analogs & derivatives*
  • Pyridoxal / chemical synthesis*
  • Pyridoxal / pharmacology
  • Reticulocytes / drug effects
  • Reticulocytes / metabolism

Substances

  • Iron Chelating Agents
  • Iron Radioisotopes
  • Ligands
  • Phenylhydrazines
  • phenylhydrazine
  • Pyridoxal
  • pyridoxal isonicotinoyl hydrazone
  • Iron
  • Isoniazid