It mainly targets boys, putting most in wheelchairs by the time they're twelve and it will most likely kill them in their twenties.
Duchenne Muscular Dystrophy leaves families with few treatment options and little hope, but now an eight-legged creature could change that.
Most boys could climb these stairs in mere seconds, but for JB Harvey it takes a lot longer. JB has Duchenne Muscular Dystrophy, a disease that slowly destroys muscles people with it rarely make it to 30. "If you don't stop and think about what we've been told his future is, he's a typical 5-year-old kid."
JB's grandpa, Jeff Harvey, wants to change that future. An internet search led him to work being done by University at Buffalo Researcher Fred Sachs. He's exploring the effects of spider venom on muscles. "One spider showed up with a compound that worked."
The Chilean Rose Tarantula. Its venom contains a protein that could slow muscle deterioration. “I've talked to Big Pharma and the minute I said cell mechanics, their eyes closed, and they nodded out and that was the end of that," says Dr. Sachs.
So the two formed their own company. Now they're developing a drug for DMD. So far, experiments show dystrophic mice given the drug had no toxic reactions, promising results that could make JB's future much brighter. “I want to do anything I possibly can to try to make my grandson live as long as possible."
"Before I retire, I'd like to see a boy get out of a wheelchair and walk away because of this," says Dr. Sachs.
Human trials for the drug could start within a year and a half. The therapy won't cure DMD, but if it works it could add years, maybe even decades to patients' lives.