Gordon Lynch, head of the department of physiology at The University of Melbourne in Victoria, Australia, was awarded an MDA research grant totaling $405,000 over a period of three years to study the potential of heat shock proteins for treatment of Duchenne muscular dystrophy (DMD). Heat shock proteins help cells fold other proteins properly, and deal with stresses from a variety of sources. The new grant complements previous MDA-funded research by Lynch into strategies aimed at improving muscle function in DMD.
“Muscle wasting and weakness are major symptoms of many muscular disorders, including Duchenne muscular dystrophy,” Lynch says. “Although considerable efforts are being directed to the development of gene therapies for DMD, these techniques are far from perfected. In the interim, alternative therapies need to be developed, and research directed to preserving muscle fibers, enhancing muscle regeneration and promoting muscle growth.”
Recently, Lynch discovered that increasing the levels of heat shock proteins in muscle fibers can reduce the damage that occurs in mouse models of DMD.
"There are important and unresolved questions regarding the potential of heat shock proteins for clinical application to DMD and related disorders,” Lynch says. He will be studying whether increasing the levels of these proteins, specifically one called Hsp72, in older dystrophic mice can offer the same benefits as seen in younger ones, and whether beneficial effects are seen in the heart, an important target in the treatment of DMD. He also will study the potential of increasing the levels of these proteins in other forms of muscular dystrophy.
“Elucidating the full therapeutic potential of Hsp72 induction in the skeletal and cardiac muscles of various models of muscular dystrophy will facilitate the development of a novel treatment to improve both skeletal and cardiac muscle function and quality of life for patients with muscular dystrophy,” Lynch says.
Funding for this MDA grant began Feb. 1, 2013.