PEGylated derivatives of cystamine as enhanced treatments for nephropathic cystinosis.

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Omran Z, Kay G, Di Salvo A, Knott RM, Cairns D

PEGylated derivatives of cystamine as enhanced treatments for nephropathic cystinosis.

Bioorg Med Chem Lett. 2011 Jan 1;21(1):45-7. doi: 10.1016/j.bmcl.2010.11.085. Epub 2010 Nov 21.

PubMed ID

21147534 [ View in PubMed

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Abstract

The genetic disease, nephropathic cystinosis is characterized by lysosomal accumulation of the amino acid cystine. Crystallization of cystine in affected organs, if untreated, results in mortality of the affected individuals by their middle to late teens. The only approved treatment for cystinosis is administration of cysteamine. However, cysteamine is associated with an offending odor and taste and this, coupled to a rapid first pass metabolism and a 6h dosing regimen, suggest a clear need to improve the therapy. A number of PEGylated derivatives of cystamine, the disulfide counterpart of cysteamine, have been synthesised and evaluated in cultured cystinotic fibroblasts for toxicity and efficacy. All of the tested compounds were non-cytotoxic and displayed a remarkable depletion of intralysosomal cystine.

DrugBank Data that Cites this Article

Drug Targets

Drug	Target	Kind	Organism	Pharmacological Action	Actions
Cysteamine	Cystine	Small molecule	Humans	Unknown	Cleavage	Details