"As I get older, I would feel really good to have been able to
help out some kids. To have a kid get out of a wheelchair and walk
away would be unbelievably satisfying."
Frederick Sachs, PhD
SUNY Distinguished Professor in physiology and
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
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."