PROVIDENCE, R.I. [Brown University] Development of a potential treatment for the most common form of muscular dystrophy is moving into a new phase at Brown University. Brown has granted an exclusive license for the intellectual property to Tivorsan Pharmaceuticals, a startup firm that will strive to see it through human trials.
Officials from Tivorsan and Brown will meet on campus today (Monday, Sept. 20, 2010) to mark the start of this next phase of the potential treatment's development.
In mice, the treatment, based on a protein called biglycan, has slowed by 50 percent the kind of muscle damage that is thought to be the cause of muscle deterioration in boys with Duchenne muscular dystrophy (DMD), said Justin Fallon, professor of neuroscience, who has led the research. In DMD, which affects one in every 3,500 boys, sufferers are consigned to an incurable fate in which scar tissue and fat replace almost every skeletal muscle in their body. Patients inevitably die, typically by the time they are in their 20s.
"At this point we cannot say how effective biglycan will be in humans, however the results from the mouse studies are encouraging," said Fallon, who is affiliated with the Brown Institute for Brain Science. "We are seeking an effective treatment that gives boys a better quality of life for years. We hope to prolong the time they can walk without assistance and perform the activities they enjoy doing most."
Joel Braunstein, one of the Tivorsan founders and acting CEO, said the company will raise the money and hire the expert staff to see biglycan through the through the drug development and government regulatory review and approval processes.
"We have a challenging road ahead of us but we are firmly committed to working toward testing the safety and efficacy of biglycan in humans," said Braunstein, a physician and principal at LifeTech Research in Baltimore. "We deeply acknowledge the need and urgency for a ne
|Contact: David Orenstein|