Potential of cystamine and cysteamine in the treatment of neurodegenerative diseases

Prog Neuropsychopharmacol Biol Psychiatry. 2011 Mar 30;35(2):380-9. doi: 10.1016/j.pnpbp.2010.11.023. Epub 2010 Nov 24.


Neurodegenerative disorders are a subset of disabling pathologies characterized, in part, by a progressive and specific loss of certain brain cell populations. Current therapeutic approaches for the treatment of these disorders are mainly designed towards symptom management and do not manifestly block their typified neuronal loss. However, research conducted over the past decade has reflected the increasing interest and need to find disease-modifying molecules. Among the several neuroprotective agents emerging from experimental animal work, cystamine, as well as its reduced form cysteamine, have been identified as potential candidate drugs. Given the significant benefits observed in a Huntington's disease (HD) model, cysteamine has recently leaped to clinical trial. Here, we review the beneficial properties of these compounds as reported in animal studies, their mechanistic underpinnings, and their potential implications for the future treatment of patients suffering from neurodegenerative diseases, and more specifically for HD and Parkinson's disease (PD).

Publication types

  • Review

MeSH terms

  • Animals
  • Cystamine / chemistry
  • Cystamine / pharmacology
  • Cystamine / therapeutic use*
  • Cysteamine / chemistry
  • Cysteamine / pharmacology*
  • Cysteamine / therapeutic use*
  • Disease Models, Animal
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacology
  • Enzyme Inhibitors / therapeutic use*
  • Humans
  • Huntington Disease / drug therapy*
  • Huntington Disease / physiopathology
  • Mice
  • Mice, Transgenic
  • Molecular Targeted Therapy
  • Neurodegenerative Diseases / drug therapy*
  • Neurodegenerative Diseases / physiopathology
  • Neuroprotective Agents / therapeutic use
  • Parkinson Disease / drug therapy*
  • Parkinson Disease / physiopathology
  • Radiation-Protective Agents / chemistry
  • Radiation-Protective Agents / pharmacology*


  • Enzyme Inhibitors
  • Neuroprotective Agents
  • Radiation-Protective Agents
  • Cysteamine
  • Cystamine