cysteamine

Cysteamine is a medication used to treat cystinosis, a rare genetic disorder that causes the accumulation of cystine within cells. Cysteamine is an amino acid that has been shown to bind to cystine, forming a complex that is excreted in the urine, thereby reducing cystine levels within cells.

Cysteamine was first synthesized in the 1970s and was approved for medical use in 1983 in the United States. It is available in both oral and eye drops formulations. The medication is typically administered 2-3 times daily and its effectiveness is gradually increased over several years of treatment.

Studies have shown that cysteamine is effective in reducing the accumulation of cystine within cells, thereby slowing down the progression of cystinosis. However, the medication does not reverse the damage that has already been done, and its long-term effectiveness in preventing kidney failure and other complications of cystinosis remains uncertain.

Cysteamine has been also used as an antidote for acetaminophen poisoning, as it can help to reduce the formation of N-acetyl-p-benzoquinoneimine (NAPQI), one of the toxic metabolites of acetaminophen.

Side effects of cysteamine may include gastrointestinal disturbances, headache, and changes in liver function. The medication should be used with caution in patients with liver disease, as it may exacerbate liver damage.

Overall, cysteamine is a treatment option for cystinosis and acetaminophen poisoning, but its effectiveness and safety profile should be closely monitored by healthcare professionals.