While focusing on heart disease, researchers discover new tactic against fatal muscular dystrophy

February 8, 2009

Based 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.

Source: Columbia University Medical Center

Explore further: Mouse studies shed light on how protein controls heart failure

Related Stories

Mouse studies shed light on how protein controls heart failure

October 18, 2017
A new study on two specially bred strains of mice has illuminated how abnormal addition of the chemical phosphate to a specific heart muscle protein may sabotage the way the protein behaves in a cell, and may damage the way ...

Saving hearts after heart attacks: Overexpression of a gene enhances repair of dead muscle

October 17, 2017
University of Alabama at Birmingham biomedical engineers report a significant advance in efforts to repair a damaged heart after a heart attack, using grafted heart-muscle cells to create a repair patch. The key was overexpressing ...

Blood cancer gene could be key to preventing heart failure

October 16, 2017
A new study, published today in Circulation, shows that the gene Runx1 increases in damaged heart muscle after a heart attack. An international collaboration led by researchers from the University of Glasgow, found that mice ...

Too little fat is bad for cardiovascular health and leptin therapy may help

October 16, 2017
We know that too much body fat is generally bad for our cardiovascular system, and now scientists are learning more about how too little fat yields some of the same damage.

Scientists reverse advanced heart failure in an animal model

October 4, 2017
Researchers have discovered a previously unrecognized healing capacity of the heart. In a mouse model, they were able to reverse severe heart failure by silencing the activity of Hippo, a signaling pathway that can prevent ...

Tom Petty died from a cardiac arrest – what makes this different from a heart attack and heart failure?

October 6, 2017
Rolling Stone magazine landed in a spot of bother on Monday after publicising news of rock star Tom Petty's death prematurely, while others said it was the result of a heart attack rather than a cardiac arrest. Petty unfortunately ...

Recommended for you

Engineered protein treatment found to reduce obesity in mice, rats and primates

October 19, 2017
(Medical Xpress)—A team of researchers with pharmaceutical company Amgen Inc. report that an engineered version of a protein naturally found in the body caused test mice, rats and cynomolgus monkeys to lose weight. In their ...

New procedure enables cultivation of human brain sections in the petri dish

October 19, 2017
Researchers at the University of Tübingen have become the first to keep human brain tissue alive outside the body for several weeks. The researchers, headed by Dr. Niklas Schwarz, Dr. Henner Koch and Dr. Thomas Wuttke at ...

Cancer drug found to offer promising results in treating sepsis in test mice

October 19, 2017
(Medical Xpress)—A combined team of researchers from China and the U.S. has found that a drug commonly used to treat lung cancer in humans offers a degree of protection against sepsis in test mice. In their paper published ...

Tracing cell death pathway points to drug targets for brain damage, kidney injury, asthma

October 19, 2017
University of Pittsburgh scientists are unlocking the complexities of a recently discovered cell death process that plays a key role in health and disease, and new findings link their discovery to asthma, kidney injury and ...

Study reveals key molecular link in major cell growth pathway

October 19, 2017
A team of scientists led by Whitehead Institute has uncovered a surprising molecular link that connects how cells regulate growth with how they sense and make available the nutrients required for growth. Their work, which ...

Inflammation trains the skin to heal faster

October 18, 2017
Scars may fade, but the skin remembers. New research from The Rockefeller University reveals that wounds or other harmful, inflammation-provoking experiences impart long-lasting memories to stem cells residing in the skin, ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.