Brown University granted Tivorsan Pharmaceuticals, a start-up firm, an exclusive license for the intellectual property to a potential treatment for Duchenne muscular dystrophy (DMD). The compound is based on a protein called biglycan and in mice slowed the kind of muscle damage that is thought to be the cause of muscle deterioration in boys with DMD by 50%, reports Justin Fallon, Ph.D., professor of neuroscience, who has led the research.
"At this point we cannot say how effective biglycan will be in humans, however the results from the mouse studies are encouraging," points out Dr. 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, M.D., one of the Tivorsan founders and acting CEO, says that the company will raise the money and hire the expert staff to see biglycan through drug development, regulatory review, and approval.
"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," notes Dr. Braunstein, a principal at LifeTech Research in Baltimore. "If biglycan proves safe and effective in human testing, the potential exists for providing relief to thousands of boys and their families around the world."
In 2000, a team led by Dr. Fallon began to accumulate evidence that biglycan, which is produced naturally in the body, regulates the production of a protein called utrophin. Utrophin, discovered in 1989, stimulates the body to repair muscle damage, just like dystrophin, the protein that DMD-afflicted boys are genetically unable to produce.
All young children produce utrophin, but they produce much less of it naturally as they get older. "In normal individuals that's fine because dystrophin is there, but in a Duchenne boy there is no hand-off," Dr. Fallon explains. "The baton is dropped." This helps explain why DMD doesn't begin to show up in boys until they are preschoolers and why they still retain enough strength to walk on their own until they are preteens.
Further research by Dr. Fallon and associate Beth McKechnie revealed that biglycan is rare in its class of proteins because the physiologically active form can be manufactured relatively easily using recombinant lab techniques. In the last several years, Dr. Fallon's group has been testing and refining the use of biglycan in DMD mice.
Last year Dr. Fallon won a four-year $5.2 million grant from the NIH to advance the development of biglycan. Dr. Fallon also reportedly won funding from foundations dedicated to fighting DMD, including Parent Project Muscular Dystrophy and Charley's Fund.