New class of heart disease drugs may be effective against fatal muscular disorder

February 9th, 2009 - 12:07 pm ICT by ANI - Send to a friend:

London, Feb 9 (ANI): Researchers at Columbia University Medical Centre say that a new class of experimental drugs for heart failure may be effective against fatal muscular disorder.

During a study, the research team identified a leak that weakens skeletal muscle in Duchenne, a disorder that affects boys usually before the age of 6, destroying their muscle cells and cardiac muscle in heart failure.

This leak allows the calcium to slowly seep into the skeletal muscle cells and excess calcium ultimately cause damage.

Similarly in heart failure patients, the calcium leak weakens the force produced by the heart, and turns on a protein-digesting enzyme that damages its muscle fibres.

Lead researcher Dr. Andrew Marks believes that a new class of experimental drugs developed at CUMC, designed to plug the leak in the heart might be effective against Duchenne.

While experimenting on mice with Duchenne, the researchers found these drugs to dramatically improve muscle strength and reduce the number of damaged muscle cells.

“This was extremely exciting to us,” Nature magazine quoted Dr. Marks, chair of the Department of Physiology & Cellular Biophysics and Clyde and Helen Wu Professor of Molecular Cardiology, as saying.

“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,” he added.

The study appears in the journal Nature Medicine. (ANI)


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Newswise — 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.



Columbia University Medical Center provides international leadership in basic, pre-clinical and clinical research, in medical and health sciences education, and in patient care. The medical center trains future leaders and includes the dedicated work of many physicians, scientists, public health professionals, dentists, and nurses at the College of Physicians & Surgeons, the Mailman School of Public Health, the College of Dental Medicine, the School of Nursing, the biomedical departments of the Graduate School of Arts and Sciences, and allied research centers and institutions. Established in 1767, Columbia's College of Physicians & Surgeons was the first institution in the country to grant the M.D. degree and is among the most selective medical schools in the country. Columbia University Medical Center is home to the largest medical research enterprise in New York City and state and one of the largest in the United States. For more information, please visit www.cumc.columbia.edu.


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© 2009 Newswise. All Rights Reserved.

http://www.newswise.com/articles/view/548856/
I was very interested to see this article too. It popped up on my Google Alert on Monday and I have been waiting for discussions that might reveal more details. Anybody know anything more about this?
Hi Donna:

I just sent you a message.

Donna Taylor said:
I was very interested to see this article too. It popped up on my Google Alert on Monday and I have been waiting for discussions that might reveal more details. Anybody know anything more about this?
How awesome will this be. Sounds great, I hope it becomes a reality. A reality... very quickly.
Does anyone know what drugs are they referring to? In one of the articles, they talked about the drug being in phase 1 clinical trials for safety.
See the website www.armgo.com for company info - products, pipeline, etc... The tab for Rycal Science is interesting with an animation & explaination.
They are in very early stages though...not sure if it would benefit THIS generation of boys.

http://www.armgo.com/pipeline.html

Traci Strafuss said:
See the website www.armgo.com for company info - products, pipeline, etc... The tab for Rycal Science is interesting with an animation & explaination.

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