Navigation Links
Muscular Dystrophy Treatment Shows Promise

In early research, scientists were able to rid mice of muscle stiffness, weakness

THURSDAY, Nov. 15 (HealthDay News) -- When injected into mice, a novel compound eliminated the severe muscle stiffness and weakness that is a disabling symptom of one form of muscular dystrophy.

But the research, though promising, is still in its infancy, the investigators and other experts stressed.

"It's an important step forward that should be encouraging for people with the condition and their families and it shows that doctors and scientists are really now beginning to fix their attention on fixing this disease, rather than trying to understand what goes wrong," said study senior author Dr. Charles Thornton, a professor of neurology at the University of Rochester School of Medicine and Dentistry.

Thornton cautioned that he would not use the term "breakthrough" to describe the results, however.

"It's an important first step and these results are encouraging that effective therapies for myotonic [muscle-stiffening] dystrophy can be developed that can actually ameliorate some of the clinical symptoms," added Dr. Valerie Cwik, medical director and vice president of research at the Muscular Dystrophy Association (MDA). "But this is a study in mice, and we know from lots of other disorders and studies in mice that what works in mice doesn't always translate into effective therapy in humans."

According to the MDA, myotonic dystrophy is one of nine forms of muscular dystrophy, a group of genetic, degenerative and potentially disabling diseases which primarily affect voluntary muscles.

Myotonic dystrophy type 1 is the most common form of muscular dystrophy affecting adults. It is caused when a section of DNA is repeated on either chromosome 19 or chromosome 3.

"Myotonic dystrophy affects multiple systems in the body, so it causes central nervous system problems with altered cognition in some people, sleep disturbances, cataracts, droopy eyelids, not only myotonia," Cwik explained. Myotonia involves a "difficulty relaxing muscles but also muscle weakness, which is actually more disabling for many people," she said. It also involves "intestinal problems and, importantly, it can affect the heart, with potentially life-threatening arrhythmias," Cwik explained. "It's a multi-system disorder."

"Presently there is no cure. There are some treatments that help manage symptoms. They're not highly effective," Thornton added. "The disease has been known for 100 years but, until about six years ago, hardly anything was understood about what causes muscle problems. Now there are detailed theories about what's going wrong. This was an attempt to test one of those to see if symptoms could be fixed."

Five years ago, Thornton's team showed that a genetic flaw leads to the overproduction of a certain type of messenger RNA (mRNA) in cells, which affects muscle control and other cellular functions.

The current study, published online Nov. 15 in the Journal of Clinical Investigation, focuses on myotonia.

The researchers postulated that myotonia was caused by a problem with the pathway that electrical charges use to move in and out of muscle cells.

When researchers injected a synthetic molecule directly into the muscles of mice with myotonic dystrophy and then corrected the electrical control problem, the symptoms were eliminated.

"When a particular chemical imbalance in the muscle tissue was restored, it eliminated stiffness," Thornton said. "This would be a partial solution to the person's symptoms with myotonic dystrophy, but it's also proof of principle that once we identify the cause that it can be corrected."

Considerably more work is needed, however.

For one thing, scientists need to find a way to inject the compound into a person's bloodstream so a systemic effect would take place. Injecting into muscles spurs only a local effect. "That's one obstacle that must be overcome in getting wide distribution," Thornton said.

Safety issues also have to be considered before human trials begin, Cwik added.

"They are only looking at one symptom of myotonic dystrophy," she noted. "Would this therapy or kind of therapy, which can reverse or eliminate myotonia, have any effect on some of the other potentially more disabling symptoms? While it's encouraging, there are a lot of cautions and caveats."

More information

There's more on myotonic dystrophy at the Muscular Dystrophy Association.

SOURCES: Charles A. Thornton, M.D., professor, neurology, University of Rochester School of Medicine and Dentistry; Valerie Cwik, M.D., medical director and vice president, research, Muscular Dystrophy Association; Nov. 15, 2007, Journal of Clinical Investigation

Copyright©2007 ScoutNews,LLC.
All rights reserved  

Related medicine news :

1. AGA Medical Corporation Receives Approval for the AMPLATZER Muscular Ventricular Septal Defect Occluder
2. Summit on Spinal Muscular Atrophy
3. Jerry Lewis Urges Congress to Protect Medicare Benefits of People With Neuromuscular Diseases
4. New center to lead international muscular dystrophy research effort
5. New treatment effective in counteracting cocaine-induced symptoms
6. New treatment boosts muscle function in myasthenia gravis
7. Broad-based group of physicians calls for improvement in stroke treatment
8. Cranberry Could Juice Up Ovarian Cancer Treatment
9. Non-medicinal treatment touted for pre-schoolers with ADHD
10. Nonmedicinal treatment touted for preschoolers with ADHD
11. U of M study: Early treatment can reverse heart damage
Post Your Comments:
Related Image:
Muscular Dystrophy Treatment Shows Promise
(Date:11/27/2015)... ... November 27, 2015 , ... An ... way to dispense prescription medications at home, so he invented the patent-pending ELECTRONIC ... and dispense prescription medications. In doing so, it could help to prevent potential ...
(Date:11/27/2015)... ... November 27, 2015 , ... MPWH, the No.1 Herpes-only dating community in the world, revealed ... 1-1 ). More than 3.7 billion people under the age of 50 – or ... to WHO's first global estimates of HSV-1 infection . , "The data shocks us ...
(Date:11/27/2015)... ... November 27, 2015 , ... ... innovative online platform for mental health and wellness consultation, has collaborated with a ... will bridge the knowledge gap experienced by parents and bring advice from parenting ...
(Date:11/27/2015)... ... November 27, 2015 , ... Indosoft Inc., developer ... incorporation of Asterisk 11 LTS (Long Term Support) into its Q-Suite 5.10 product ... Q-Suite 5.10 up-to-date with a version of Asterisk that will receive not only ...
(Date:11/26/2015)... ... November 26, 2015 , ... The ... bioavailability and the need to integrate dose form selection in early phase drug ... the membership organization supporting and bringing together the UK’s emerging life sciences companies, ...
Breaking Medicine News(10 mins):
(Date:11/26/2015)... , November 26, 2015 ... "Self Administration of High Viscosity Drugs" ... ) has announced the addition of the ... to their offering. --> Research and ... of the "Self Administration of High Viscosity ...
(Date:11/26/2015)... DUBLIN , November 26, 2015 /PRNewswire/ ... the addition of the "Radioimmunoassay Market ... User (Hospital, Pharmaceutical Industry, Academics, Clinical Diagnostic ... - Global Forecast to 2020" report ... ) has announced the addition of the ...
(Date:11/25/2015)... , Nov. 25, 2015 ... the addition of the "Global Brain ... their offering. --> ) ... "Global Brain Monitoring Devices Market 2015-2019" ... Research and Markets ( ) has ...
Breaking Medicine Technology: