Tivorsan Pharmaceuticals? Biglycan Protein Opens Unique Path for Preserving Muscle Function in Duchenne Muscular Dystrophy
Study published in PNAS shows recombinant human biglycan reduces muscle damage and improves function in mouse model of DMD
PROVIDENCE, R.I.--Dec. 28, 2010--(BUSINESS WIRE)--A novel therapy under development by Tivorsan Pharmaceuticals demonstrated reduction of muscle pathology and improved muscle function in a mouse model of Duchenne Muscular Dystrophy (DMD). The company is actively working towards human clinical studies with a proprietary form of the natural biglycan protein, which appears to have a unique mechanism of action that may apply to all genetic forms of DMD.
In this study, injections of recombinant human biglycan (rhBGN) reduced dystrophic pathology and improved muscle function in mice with the same mutation that affects DMD boys. The study was conducted by Tivorsan's founding scientist, Dr. Justin Fallon of Brown University, and colleagues. These findings are reported in the December 27 online version of the Proceeds of the National Academy of Sciences.
Duchenne Muscular Dystrophy (DMD) is an unrelenting muscle wasting disorder that leads to death in early adulthood. Affecting approximately 1 in 3,500 newborn boys, this most common of X-linked diseases is triggered by mutations of the gene encoding the dystrophin protein. In normal adults, dystrophin keeps muscles strong, but DMD mutations switch off dystrophin production so that affected boys gradually and permanently lose muscle strength. No current treatments successfully address the biologic course of the disease.
"Dr. Fallon and his colleagues have shown us that we have a biologically active molecule with an apparently unique mechanism of action that may be applicable to all genetic forms of DMD,” said Dr. Joel Braunstein, co-founding partner at LifeTech Research, member of the board of directors and acting CEO at Tivorsan. “Most importantly, biglycan shows potential to slow muscle wasting and modify the progressive course of the disease. The study also suggests that simple injections may have a durable effect on muscle strength.”
"Multiple lines of evidence make biglycan a compelling candidate for moving into human testing as soon as possible. Our team recognizes a special opportunity to help many children and their families suffering from this disease, and therefore we are working hard to advance an optimized form of the biglycan protein through production and preclinical testing into the clinic."
Biglycan induces the expression in the muscle cell membrane (sarcolemma) of a fetal protein called utrophin that is typically replaced in adults by dystrophin. Thus, utrophin offers an alternative pathway to maintaining the integrity of the muscle cell membrane in DMD boys. Biglycan acts through an apparently unique mechanism of action, distinct from other treatments in development, yet potentially suitable for concurrent use with such treatments. In this study, systemically administered biglycan was well-tolerated and remained effective for approximately three weeks following a one-time dosing. Therapeutic effects persisted out to three months following dosing once every three weeks.
Tivorsan Pharmaceuticals http://www.tivorsan.com is a protein therapeutics company pioneering a unique approach to treating serious neuromuscular disorders, including DMD and Becker Muscular Dystrophy (BMD). This method, using recombinant human biglycan (rhBGN), is based on 24 years of basic science work in the Fallon laboratory at Brown University (http://neuroscience.brown.edu/fallon). Tivorsan was formed by Dr. Fallon in collaboration with colleagues from Old Forge Holdings of Greenwich, CT and LifeTech Research, a Baltimore-based technology research and development firm (http://lifetechresearch.com). Early support for the Tivorsan program originated, in part, from Federal sources, as well as Charley's Fund (http://charleysfund.com) and the Nash Avery Foundation (http://nashaveryfoundation.org), two philanthropies seeking to accelerate the development of cures for DMD.
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Posted: December 2010