Small Molecule Fills Hormone's Shoes

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Science  10 Jul 1998:
Vol. 281, Issue 5374, pp. 149-150
DOI: 10.1126/science.281.5374.149b

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Drug companies have long sought small compounds that mimic the effects of protein drugs yet evade breakdown in the digestive tract, but the small chemicals, which might be just 1/50 of a protein's size, seemed too puny to turn on the proteins' surface receptors. Now, on page 257, researchers report their discovery of a small molecule that activates the receptor for granulocyte-colony-stimulating factor, a cytokine that is commonly used to boost patients' immune systems after chemotherapy. Although the new compound works in mice but not humans, it is being heralded as evidence that the right small molecule can indeed fill a protein hormone's shoes.