BUFFALO, N.Y. -- While Spider-Man is capturing the imagination
of theatergoers, real-life spider men in Upstate New York are
working intently to save a young boy's life.
It all began in 2009, when Jeff Harvey, a stockbroker from the
Buffalo suburbs, discovered that his grandson, JB, had Duchenne
muscular dystrophy. The disease is fatal. It strikes only boys,
causing their muscles to waste away.
Hoping to help his grandson, Harvey searched Google for
promising muscular dystrophy treatments and, in a moment of
serendipity, stumbled upon University at Buffalo scientist
Frederick Sachs, PhD.
Sachs was a professor of physiology and biophysics who had been
studying the medical benefits of venom. In the venom of the Chilean
rose tarantula, he and his colleagues discovered a protein that
held promise for keeping muscular dystrophy at bay. Specifically,
the protein helped stop muscle cells from deteriorating.
Within months of getting in touch, Harvey and Sachs co-founded
Tonus Therapeutics,
a pharmaceutical company devoted to developing the protein as a
drug. Though the treatment has yet to be tested in humans, it has
helped dystrophic mice gain strength in preliminary
experiments.
The therapy is not a cure. But if it works in humans, it could
extend the lives of children like JB for years -- maybe even
decades.
Success can't come quickly enough.
JB, now four, can't walk down the stairs alone. When he runs, he
waddles. He receives physical therapy and takes steroids as a
treatment. While playing tee ball one recent day, he confided to
his grandfather, "When I grow up, I want to be a baseball
player."
It was a heartbreaking moment.
"Oh, I would be thrilled if you could be a baseball player,"
Harvey remembers replying. He's doing everything he can to make
sure that JB -- and other boys like him -- can live out their
dreams.
For the complete story and multimedia, visit http://www.buffalo.edu/home/feature_story/good-venom.html.