Featured Research

from universities, journals, and other organizations

Common drug reverses common effect of becker muscular dystrophy

Date:
November 28, 2012
Source:
Cedars-Sinai Medical Center
Summary:
Researchers have found in an initial clinical trial that a drug typically prescribed for erectile dysfunction or pulmonary hypertension restores blood flow to oxygen-starved muscles in patients with a type of muscular dystrophy that affects males, typically starting in childhood or adolescence.

Cedars-Sinai Heart Institute researchers have found in an initial clinical trial that a drug typically prescribed for erectile dysfunction or pulmonary hypertension restores blood flow to oxygen-starved muscles in patients with a type of muscular dystrophy that affects males, typically starting in childhood or adolescence.

Related Articles


Tadalafil, commonly known by brand names Cialis and Adcirca, reversed the effects of a biochemical chain of events that in Becker muscular dystrophy deprives muscles of an important chemical, nitric oxide, which normally tells blood vessels to relax during exercise, increasing blood flow and oxygenation.

With a single dose, the drug, which works downstream from nitric oxide, fully restored proper blood flow in eight of nine patients in the study, and the effects were "both marked and immediate," according to an article in the journal Science Translational Medicine.

"There is no treatment for this progressive muscle-wasting disease and there has been little research. Previous studies in laboratory mice suggested that drugs such as tadalafil could restore proper blood flow, but this is the first study showing that the drug may offer a therapeutic strategy in humans," said Ronald G. Victor, MD, director of the Cedars-Sinai Hypertension Center of Excellence and associate director of the Cedars-Sinai Heart Institute and the Burns and Allen Chair in Cardiology Research. He is the article's senior author.

Becker muscular dystrophy results from a genetic defect that reduces the amount of a protein called dystrophin in the membrane of muscle cells. With insufficient and poorly functioning dystrophin, patients lose muscle strength and have increased risk of heart failure. Affecting 1 in 19, 000 male births, Becker muscular dystrophy is less common and more slowly progressive -- but still debilitating -- form of Duchenne muscular dystrophy which eliminates all dystrophin, affects 1 in 3,500 male babies born, and usually appears in early childhood.

Victor led the research team that previously discovered the blood flow abnormality from loss of nitric oxide in the muscles of children with Duchenne muscular dystrophy. The new study shows the same blood flow problem is common in adult patients with Becker muscular dystrophy, thereby offering improved blood circulation as a potential avenue for treatment.

The tadalafil study was a double-blind, randomized cross-over trial in which researchers measured forearm muscle oxygenation levels while patients performed handgrip exercise. Patients were evaluated after receiving either a single 20 mg capsule of tadalafil or a placebo. After a two-week break to be sure all drug was out of the patients' systems, the groups were switched. The dose used in this muscular dystrophy study is only one-half the dose approved by the Food and Drug Administration for daily use for adult patients with pulmonary hypertension.

Victor said the blood flow effects in this single-dose trial were dramatic and encouraging but more research is needed before recommending tadalafil for patients with Becker muscular dystrophy.

"Cedars-Sinai is planning longer term studies to determine if correcting muscle blood flow leads to a clinically meaningful outcome," Victor said. "This is not a cure, but it could be the first step toward identifying potential treatments for Becker muscular dystrophy. Providing adequate oxygenation to muscles already weakened by abnormal dystrophin may be a strategy to slow the course of the disease. Tadalafil already is a well-studied and well-known medication that enjoys a very favorable side-effect profile. This class of drugs has been a major advance even though not a cure for patients with pulmonary hypertension, a serious illness related to insufficient blood flow to the lungs. Since no new drug development would be needed, repurposing it for muscular dystrophy could quickly transform clinical practice."

The study was funded by research grants from the Muscular Dystrophy Association and the National Center for Research Resources (UL1RR033176, which is now at the National Center for Advancing Translational Sciences, UL1TR000124). The planning of future studies for patients with Duchenne muscular dystrophy is being supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (U34AR062893).

Researchers from the Institute of Genetic Medicine in the United Kingdom, the University of Florida's Powell Gene Therapy Center, Cedars-Sinai's Department of Neurology, the University of California at Los Angeles Department of Biomathematics, and UCLA's School of Public Health Department of Biostatistics contributed to the article.


Story Source:

The above story is based on materials provided by Cedars-Sinai Medical Center. Note: Materials may be edited for content and length.


Journal Reference:

  1. E. A. Martin, R. Barresi, B. J. Byrne, E. I. Tsimerinov, B. L. Scott, A. E. Walker, S. V. Gurudevan, F. Anene, R. M. Elashoff, G. D. Thomas, R. G. Victor. Tadalafil Alleviates Muscle Ischemia in Patients with Becker Muscular Dystrophy. Science Translational Medicine, 2012; 4 (162): 162ra155 DOI: 10.1126/scitranslmed.3004327

Cite This Page:

Cedars-Sinai Medical Center. "Common drug reverses common effect of becker muscular dystrophy." ScienceDaily. ScienceDaily, 28 November 2012. <www.sciencedaily.com/releases/2012/11/121128142653.htm>.
Cedars-Sinai Medical Center. (2012, November 28). Common drug reverses common effect of becker muscular dystrophy. ScienceDaily. Retrieved October 25, 2014 from www.sciencedaily.com/releases/2012/11/121128142653.htm
Cedars-Sinai Medical Center. "Common drug reverses common effect of becker muscular dystrophy." ScienceDaily. www.sciencedaily.com/releases/2012/11/121128142653.htm (accessed October 25, 2014).

Share This



More Health & Medicine News

Saturday, October 25, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

IKEA Desk Converts From Standing to Sitting With One Button

IKEA Desk Converts From Standing to Sitting With One Button

Buzz60 (Oct. 24, 2014) IKEA is out with a new convertible desk that can convert from a sitting desk to a standing one with just the push of a button. Jen Markham explains. Video provided by Buzz60
Powered by NewsLook.com
Ebola Protective Suits Being Made in China

Ebola Protective Suits Being Made in China

AFP (Oct. 24, 2014) A factory in China is busy making Ebola protective suits for healthcare workers and others fighting the spread of the virus. Duration: 00:38 Video provided by AFP
Powered by NewsLook.com
WHO: Millions of Ebola Vaccine Doses by 2015

WHO: Millions of Ebola Vaccine Doses by 2015

AP (Oct. 24, 2014) The World Health Organization said on Friday that millions of doses of two experimental Ebola vaccines could be ready for use in 2015 and five more experimental vaccines would start being tested in March. (Oct. 24) Video provided by AP
Powered by NewsLook.com
Doctor in NYC Quarantined With Ebola

Doctor in NYC Quarantined With Ebola

AP (Oct. 24, 2014) An emergency room doctor who recently returned to the city after treating Ebola patients in West Africa has tested positive for the virus. He's quarantined in a hospital. (Oct. 24) Video provided by AP
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins