期刊
BIOORGANIC & MEDICINAL CHEMISTRY LETTERS
卷 21, 期 1, 页码 45-47出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.bmcl.2010.11.085
关键词
Cystinosis; Cysteamine; Cystamine; Pro-drugs; PEGylation
资金
- Cystinosis Foundation, Ireland
- EPSRC
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 6 h 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. (C) 2010 Elsevier Ltd. All rights reserved.
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