Image of creme brulee by Moniza, from http://www.allrecipes.com Researchers at the University of Washington have shown that a trace component in burnt sugar (like the creme brulee above) and Caramel Colour III (used to color dark beers, brown sugar, etc) has been shown to help regenerate muscles inboth fruitfly andmouse models of Duchenne muscular dystrophy. Drs. Morayma Reyes (Professor of Pathology and Laboratory Medicine) and Hannele Ruohola-Baker (Professor of Biochemistry; Associate Director of the Institute for Stem Cell and Regenerative Medicine) studied mouse models that were missing a protein responsible for repairing muscle, called dystrophin. Like the human condition, the mice are unable to repair muscle cells. The inability to repair proteins causes muscles, including the heart and respiratory muscles, to weakenover time resulting in death usually prior to age 30. There is currently no cure or effective treatment to help repair the muscle proteins. Dr. Ruohola-Baker discovered that a bioactive lipid called sphingosine 1-phosphate is responsible for regenerating damaged tissues, converting stem cells into adult cells (embryos cannot develop without it), and preventing the death of cells. Others have shown that muscle levels of sphingosine 1-phosphate are lower in mice with muscular dystrophy and the pathways the lipidnormallyactivates do … Continue reading →

Public release date: 9-Aug-2013 [ | E-mail | Share ] Contact: Gina Bericchia Gina.Bericchia@NationwideChildrens.org 614-355-0495 Nationwide Children's Hospital New research on two promising gene therapies suggests that combining them into one treatment not only repairs muscle damage caused by Duchenne muscular dystrophy, but also prevents future injury from the muscle-wasting disease. The work, led by a team at The Research Institute at Nationwide Children's Hospital, is the first to look at the approach in aged mice, a key step toward clinical trials in patients. The findings were published in July in Human Molecular Genetics. "We're excited about the fact that these are older mice and we're still able to see a sustained functional benefit from this combined therapythis hasn't been shown before," says Louise Rodino-Klapac, PhD, a principle investigator in the Center for Gene Therapy at Nationwide Children's and lead author of the research. Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in children, affecting about one out of every 3,500 to 6,000 male births. Symptoms usually begin between the ages of 3 and 5 years and progress rapidly. Most patients are unable to walk by age 12 and ultimately need a respirator to breathe. Patients … Continue reading →

Aug. 10, 2013 Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production of a protein vital to muscle growth and health. The study, led by a team in The Research Institute at Nationwide Children's Hospital, is the first of its kind to show these results from an exon-skipping drug -- a class of therapeutics that allows cells to skip over missing parts of the gene and produce protein naturally. "I've been doing this for more than 40 years and this is one of the most exciting developments we've seen," says Jerry Mendell, MD, lead author of the study and director of the Center for Gene Therapy at Nationwide Children's. "It offers great hope to patients with Duchenne muscular dystrophy and their families." The research, which appears online Aug. 1 in the journal Annals of Neurology, is the first study from a double-blind controlled randomized trial of an exon-skipping agent to provide conclusive proof based on the standard six-minute walk test used to measure muscle function in patients with Duchenne muscular dystrophy (DMD), the most common form … Continue reading →