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Cysteamine: an old drug with new potential

Besouw, M., Masereeuw, R., van den Heuvel, L. & Levtchenko, E., Aug-2013, In : Drug Discovery Today. 18, 15-16, p. 785-792 8 p.

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  • Cysteamine: an old drug with new potential

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DOI

  • Martine Besouw
  • Rosalinde Masereeuw
  • Lambert van den Heuvel
  • Elena Levtchenko

Cysteamine is an amino thiol with the chemical formula HSCH2CH2NH2. Endogenously, cysteamine is derived from coenzyme A degradation, although its plasma concentrations are low. Most experience with cysteamine as a drug originates from the field of the orphan disease cystinosis, in which cysteamine is prescribed to decrease intralysosomal cystine accumulation. However, over the years, the drug has been used for several other applications both in vitro and in vivo. In this article, we review the different applications of cysteamine, ending with an overview of ongoing clinical trials for new indications, such as neurodegenerative disorders and nonalcoholic fatty liver disease (NAFLD). The recent development of an enteric-coated cysteamine formulation makes cysteamine more patient friendly and will extend its applicability for both old and new indications.

Original languageEnglish
Pages (from-to)785-792
Number of pages8
JournalDrug Discovery Today
Volume18
Issue number15-16
Publication statusPublished - Aug-2013

    Keywords

  • FATTY LIVER-DISEASE, NEPHROPATHIC CYSTINOSIS, HUNTINGTONS-DISEASE, INDUCED LUPUS, CYSTAMINE, CHILDREN, THERAPY, TOXICITY, GROWTH, BDNF

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