From Toxins to Therapeutics

Researchers are finding new drugs for chronic pain and autoimmune diseases by modifying animal venom-derived molecules that target the nervous and immune systems.

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A Caribbean sun anemone (Stichodactyla helianthus)FLICKR, OMAR SPENCE PHOTOGRAPHYAnimal venoms are a veritable treasure trove of proteins and peptides fine-tuned by millions of years of evolution to kill or incapacitate both predator and prey. Usually delivered via injection—through an assortment of fangs, barbs, spines, and stingers—venom toxins evade the body’s defenses to seek out target cells, where they prevent blood cells from clotting, for example, or block ion channels on nerve cells to shut down or subvert their function.

Such high molecular specificity and potency has long made venom a promising source of drug candidates. More than 30 years ago, the US Food and Drug Administration approved the first venom-derived drug—a therapy for hypertension, called Capoten, copied from a pit viper venom peptide. A handful of venom-derived drugs have since been approved for cardiovascular disease, and in 2004, a venom-derived painkiller hit the market. Now, thanks to an increasing knowledge of the human nervous and immune systems, the pipeline from fang to pharmacy is expanding even further, with more pain medications and drugs that target autoimmune diseases such as multiple sclerosis and rheumatoid arthritis.

“We’re really at beginning of something exciting,” said Glenn King, a molecular biologist and spider venom researcher at the University ...

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