Jeff Harvey, a stockbroker from Clarence, NY, learned 1½ years ago that his grandson—now 3 years old—had Duchenne muscular dystrophy, a fatal affliction that causes patients' muscles to waste away.

Harvey began reading about Duchenne muscular dystrophy and the limited treatments available for its victims. His research yielded a promising result: Frederick Sachs, a SUNY Distinguished Professor in physiology and biophysics, along with Thomas Suchyna, an assistant professor of physiology and biophysics, had discovered years ago that a peptide found in tarantula venom (GsMTx4) showed promise as a therapy for Duchenne muscular dystrophy.

Within months, the two men launched Rose Pharmaceuticals, which they named after Rosie, Sachs' pet tarantula, and Rose, Sachs' grandmother. In September 2010, the U.S. Food and Drug Administration designated GsMTx4 as an orphan drug for Duchenne muscular dystrophy, which allows a shorter testing period than normal drugs. 

Now, the company is seeking financing for clinical trials of the therapy, which improved the motor ability of dystrophic mice and showed no toxicity in the animal tests.

"We want to beat the disease," Harvey says. "My hope is that my grandson will one day have access to this treatment."

"No one in their right mind would have given spider spit to a kid with dystrophy, so it's only through the basic science that you end up here," Sachs says. "If we can carry it all the way to the clinic, that would be satisfying. You learn new things through basic research and through serendipity. If you keep your eyes open, you see things you would never have seen otherwise."