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While focusing on heart disease, researchers discover new tactic against fatal muscular dystrophy
February 09, 2009Based on a striking similarity between heart disease and Duchenne muscular dystrophy, researchers at Columbia University Medical Center have discovered that a new class of experimental drugs for heart failure may also help treat the fatal muscular disorder.
At first glance, heart failure and the muscle-wasting Duchenne disease couldn't appear more dissimilar. Duchenne affects boys usually before the age of 6, destroying their muscle cells. The boys become progressively weaker through their teens and usually die in their twenties. In people without Duchenne, heart failure typically starts much later in life, robbing the heart's pumping ability in the 7th, 8th or 9th decade of life.
But the new study found that the muscle cells affected in both diseases have sprung the same microscopic leak that ultimately weakens skeletal muscle in Duchenne and cardiac muscle in heart failure. The leak lets calcium slowly seep into the skeletal muscle cells, which are damaged from the excess calcium in Duchenne. In people with chronic heart failure, a similar calcium leak continuously weakens the force produced by the heart and also turns on a protein-digesting enzyme that damages its muscle fibers.
Andrew Marks, M.D., the study's leader, hypothesized that a new class of experimental drugs developed at CUMC - which he had designed to plug the leak in the heart - could also work for Duchenne.
The drugs, when given to mice with Duchenne, dramatically improved muscle strength and reduced the number of damaged muscle cells.
"This was extremely exciting to us," says Dr. Marks, chair of the Department of Physiology & Cellular Biophysics and Clyde and Helen Wu Professor of Molecular Cardiology. "If it works in people, our drug won't be a cure, but it could slow the pace of muscle degeneration and extend the lives of people with Duchenne."
The study was published online Feb. 8 in Nature Medicine. Though the new drugs are not FDA-approved or currently available for Duchenne patients, a similar drug that was used in the Duchenne study is undergoing Phase I safety trials, and later this year trials will begin for heart failure.
Columbia University Medical Center
Andrew Marks, M.D., the study's leader, hypothesized that a new class of experimental drugs developed at CUMC - which he had designed to plug the leak in the heart - could also work for Duchenne.
The drugs, when given to mice with Duchenne, dramatically improved muscle strength and reduced the number of damaged muscle cells.
"This was extremely exciting to us," says Dr. Marks, chair of the Department of Physiology & Cellular Biophysics and Clyde and Helen Wu Professor of Molecular Cardiology. "If it works in people, our drug won't be a cure, but it could slow the pace of muscle degeneration and extend the lives of people with Duchenne."
The study was published online Feb. 8 in Nature Medicine. Though the new drugs are not FDA-approved or currently available for Duchenne patients, a similar drug that was used in the Duchenne study is undergoing Phase I safety trials, and later this year trials will begin for heart failure.
Columbia University Medical Center
Muscular Dystrophy Current Events and Muscular Dystrophy News RSSTreatment to improve degenerating muscle gains strength
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Possible help in fight against muscle-wasting disease
A compound already used to treat pneumonia could become a new therapy for an inherited muscular wasting disease, according to researchers at the University of Oregon and the University of Rochester School of Medicine and Dentistry in New York.
Exon-skipping drug prevents muscle wasting, maintains muscle function in dystrophin deficient mice
An exon skipping PPMO has demonstrated dramatic effects in the prevention and treatment of severely affected, dystrophin and utrophin-deficient mice, preventing severe deterioration of the treated animals and extending their lifespan.
To regenerate muscle, cellular garbage men must become builders
For scientists at the European Molecular Biology Laboratory (EMBL) in Monterotondo, Italy, what seemed like a disappointing result turned out to be an important discovery.
Zoo volunteers help explain mysteries of the genome
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Small molecule inhibits pathology associated with myotonic dystrophy type 1
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Researchers identify new function for protein missing in Duchenne muscular dystrophy
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Sticky protein helps reinforce fragile muscle membranes
A new study by scientists at the University of Iowa shows why muscle membranes don't rupture when healthy people exercise.
Stem cell surprise for tissue regeneration
Scientists working at the Carnegie Institution's Department of Embryology, with colleagues, have overturned previous research that identified critical genes for making muscle stem cells.
Researchers make progress toward early identification of muscular dystrophy
The saying "Knowing is half the battle" is never more true than when discussing early treatment of disease. Muscular dystrophy is one such disease where patients can benefit from early treatment. Now, new research is moving doctors and scientists closer to disease diagnosis in advance of patient symptoms.
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