Inspired by a Spider

A drug developed by University at Buffalo scientists from a protein found in spider venom is moving forward as a promising treatment for Duchenne muscular dystrophy, a fatal genetic disease affecting mostly boys, and likely other types of muscular dystrophy as well.

Frederick Sachs, SUNY Distinguished Professor and professor of physiology and biophysics, led a research team that discovered the protein in the 1990s. He had been studying the possibility of using venom from centipedes, scorpions or spiders to counteract the muscle deterioration that characterizes muscular dystrophy. The right one happened to come from a Chilean rose tarantula—the very same species of spider that Sachs’ research team had been keeping as a pet, named Rosie, in their laboratory.

Existing treatments for muscular dystrophy involve stem cell or genetic mechanisms, and can be unpredictable. This drug works in a different way: by targeting tiny tunnels within cells known as mechanosensitive ion channels. Because muscular dystrophy patients do not produce a protein that helps muscle cells keep their shape, the cells buckle, prompting the ion channels to open and calcium to flood in, damaging the muscle.

The key protein from the venom closes the channels and prevents that deterioration. Moreover, it could potentially prevent cardiac failure, one of the most common causes of death in dystrophic boys.

To develop the drug for market, Sachs co-founded the UB spinoff firm Tonus Therapeutics with two colleagues in his department—Thomas Suchyna and Philip Gottlieb—along with Jeff Harvey, a local stockbroker who was motivated to be part of the effort by his young grandson’s diagnosis.

Following positive results in recent animal studies, the therapy is on track for application to the U.S. Food and Drug Administration and is expected to start in human trials within two years.

For Sachs, the end goal is to extend the lives of children with muscular dystrophy, turning a fatal diagnosis into a manageable condition. “To have a kid get out of a wheelchair and walk away would be unbelievably satisfying,” he says.