Most drugs are simple, small molecules produced through well-defined chemical reactions: aspirin, Lipitor, and Prozac are made this way. Biotech drugs, on the other hand, are lab-made versions of human proteins such as insulin, and they are produced by living cells. This method of production can make it hard to identify the most potent versions of the compounds, because cells don’t produce identical copies. It also hinders researchers wanting to explore unique version of biotech compounds that cells don’t naturally make.
But a study published on Thursday in Science may help scientists overcome some of these limitations. Building on recent advances in peptide synthesis, scientists at the Sloan-Kettering Institute for Cancer Research in New York report that they have used chemical reactions alone to produce a biologic drug called erythropoietin. The work lays the foundation for chemists to create and identify other biologic compounds that could be more stable or potent than conventionally produced versions or have fewer side effects, says Richard DiMarchi, a biochemist at Indiana University in Bloomington.
Erythropoietin, or EPO, boosts production of red blood cells and is used to treat anemia, which is often caused by cancer, chemotherapy, or kidney failure. One of the original and most successful biotech drugs, EPO is a complex protein with chains of sugars on its surface. Chinese hamster ovary cells, a type of mammalian cells often used in biotech manufacturing, are generally used to produce a humanlike version of EPO for pharmaceutical use. Like many other complex biotech drugs, EPO must be produced in mammalian cells because microbes like bacteria and yeast don’t have the cellular machinery to stick the critical sugar chains onto the protein.
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