Billion Dollar Gila Monster Spit?

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Source.

This venomous lizard, Heloderma suspectum, harbors a billion dollar secret: a special protein in his saliva and tail. That protein, exenatide, is highly effective in treatment for type 2 diabetes.

As American's girth and weight continues to increase, so does type 2 diabetes. It is a vicious cycle:

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Increased obesity leads to more enlarged fat cells that respond less and less to insulin needed for control of blood sugar - a hallmark for type 2 diabetes. This leads to increased appetite and overeating and obesity. And the cycle continues.

The protein isolated from the Gila monster can break this cycle by reducing appetite and enhancing how the pancreas responds to insulin. Like any medicine, there are side effects {a warning was added to the manufacturer's label}, and because it is a protein, it must be injected.

Researchers have searched for years for a way to mimic this protein using a "small" molecule - that is, simpler - because usually such "small" molecules can be taken as a pill. But this goal remains elusive.

So how could the product of gila monster's spit be worth a billion dollars? Consider this:

The medication, Byetta, produced by Amylin/Lilly yielded $710 million in sales in 2010. It's only a matter of time to reach that milestone.

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But there's a broader lesson here: nature has yielded, and is hiding, many secrets that can improve our health outcomes and increase our lifespan. Yes, exercise and good nutrition is tremendously important, but such medications provide a critical boost.

To learn more about these medicines from nature, see a recent article published in Chemical & Engineering News. You will learn about medicinal secrets within the magician's cone snail and the saw-scaled viper!

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It's tough to get metabolic disease clinical trials done due to the number of participants required. Peptides are convenient for HTS discovery, which most pharmaceutical companies and some CROs already have, but the models (DIO mice etc) are not great and often targets that seem really great, like PPAR, turn out to be useless in the clinic.

Problems include short half-life and immunogenicity. Thus far, most pharmaceutical companies, I kid you not, just don't bother trying to figure out immunogenicity because it's considered too complicated a problem to model and a positive result would doom their pipeline--even if you aren't sure how accurate your model is and therefore don't know if it means anything. Hence, synthetic or endogenous peptides tend to be preferable to naturally occurring nonhuman ones.