Pharmacology
People with cystinosis lack a functioning transporter (cystinosin) which transports cystine from the lysosome to the cytosol. This ultimately leads to buildup of cystine in lysosomes, where it crystallizes and damages cells. Cysteamine enters lysosomes and converts cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome. Biological function
Cysteamine also promotes the transport of L-cysteine into cells, that can be further used to synthesize glutathione, which is one of the most potent intracellular antioxidants.Cysteamine is used as a drug for the treatment of cystinosis; it removes cystine that builds up in cells of people with the disease. History
First evidence regarding the therapeutic effect of cysteamine on cystinosis dates back to 1950s. Cysteamine was first approved as a drug for cystinosis in the US in 1994. An extended release form was approved in 2013. Society and culture
It is approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).In 2013, the regular capsule of cysteamine cost about $8,000 per year; the extended release form that was introduced that year was priced at $250,000 per year.
Glibenclamide, also known as glyburide, is an antidiabetic medication used to treat type 2 diabetes. It is recommended that it be taken together with diet and exercise. It may be used with other antidiabetic medication. It is not recommended for use by itself in type 1 diabetes. It is taken by mouth.Common side effects include nausea and heartburn. Serious side effects may include angioedema and low blood sugar. It is generally not recommended during pregnancy but can be used during breastfeeding. It is in the sulfonylureas class of medications and works by increasing the release of insulin from the pancreas.Glibenclamide was discovered in 1969 and approved for medical use in the United States in 1984. It is available as a generic medication. In 2019, it was the 254th most commonly prescribed medication in the United States, with more than 1 million prescriptions. Medical uses
Glibenclamide is indicated as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes.It is not as good as either metformin or insulin in those who have gestational diabetes. Side effects
Frequently reported side effects include: nausea, heartburn, weight gain, and bloating. The medication is also a major cause of medication-induced hypoglycemia. The risk is greater than with other sulfonylureas.Glibenclamide may be not recommended in those with G6PD deficiency, as it may cause acute hemolysis. Pregnancy and breastfeeding
It is generally not recommended during pregnancy but can be used during breastfeeding.