Could Venom Hold the Key to Treating Disease?

For centuries, venom was feared. Today, it’s becoming one of medicine’s most surprising sources of healing. With many nontraditional uses for animal venom, it actually may hold the answers to myriad medical questions.
In the 1980s, Jean-Pierre Raufman, MD, and John Pisano, PhD, conducted studies on the potential of animal venom at the National Institutes of Health, as reported by Kim Tingley in the New York Times Magazine.¹ At that time Pisano had a personal collection of animal venoms that he offered to share with Raufman who was interested in studying the peptides in these compounds. Raufman used a number of these specimens to evaluate their effect on cells taken from different animal organs.

One group was venom taken from Gila monsters. He measured their effect on pancreatic cells harvested from guinea pigs. While some of the initial findings were positive, Raufman set aside his work on the topic. He restarted his research after he moved to the Veterans Affairs Medical Center in the Bronx where he started a collaboration with  John Eng, MD, an endocrinologist. Eng was intrigued by the results of Raufman’s previous work. He was particularly impressed with the Gila monsters’ glacial metabolism and that allowed the animals to survive on a handful of meals per year. Eng’s studies found this was a result of two novel molecules: exendin 3 and exendin 4. He recognized that one of these molecules had potential use for individuals with diabetes. This was because exendin 4 closely resembles the human hormone GLP-1, a natural insulin manager and is important in regulating blood sugar levels. The small intestine releases GLP-1 only when the blood sugar levels are elevated. This slows digestion and results in the individual feeling satiated. The human peptide has this effect for only a few minutes while the Gila monster analog lasts for hours.

Eng went on to patent and license the peptide and later sold the rights to Amylin Pharmaceuticals. The company received approval from the United States Food and Drug Administration for use in the treatment of type 2 diabetes in 2005.

One of the unexpected side effects of this medication was weight loss. Other companies slowly realized how useful this side effect could be and as a result, multiple blockbuster drugs including Ozempic, Wegovy, Mounjaro, and Zepbound followed. The ability of these drugs to transform society is only beginning to be understood, because it appears that some variations could be protective against kidney and heart disease and may reduce inflammation linked to development of Parkinson and Alzheimer diseases.

Research continues on venoms derived from insects, spiders, corals, snakes, lizards, and a even a few mammals. In one important example, David Julius, PhD, a molecular biologist at the University of California, San Francisco, used spider venom to activate cellular receptors for temperature and touch to elucidate the structure and function of these molecules. His work resulted in winning the 2021 Nobel Prize in Physiology or Medicine. Julius feels that venom serves not only as a reservoir of potential pharmacological therapies but also as a powerful tool for basic scientific research. “It is still a great resource, largely untapped, for molecules that if they don’t become drugs can tell us about how to design drugs.“¹

Reference

1. Tingley K. Nature’s ‘Swiss army knife’: what can we learn from venom? New York Times Magazine. November 13, 2004.

From Decisions in Dentistry. July/August 2025;11(4):6.