Corporate Banner
Satellite Banner
Immunology
Scientific Community
 
Become a Member | Sign in
Home>News>This Article
  News
Return

New Muscular Dystrophy Treatment Shows Promise in Early Study

Published: Monday, September 09, 2013
Last Updated: Monday, September 09, 2013
Bookmark and Share
A preclinical study has found that a new oral drug shows early promise for the treatment of muscular dystrophy.

The results, which are published today in EMBO Molecular Medicine, show that VBP15 decreases inflammation in mice with symptoms similar to those found in patients with Duchenne muscular dystrophy. The authors found that the drug protects and strengthens muscle without the harsh side effects linked to current treatments with glucocorticoids such as prednisone.

Duchenne muscular dystrophy results in severe muscle degeneration and affects approximately 180,000 patients worldwide, mostly children. Treatment with the current standard therapy, glucocorticoids, can only be used for a short time due to serious side effects leading to fragile bones and suppression of both the immune system and growth hormone production.

The researchers also observed that VBP15 inhibits the transcription factor NF-κB, a key cell-signaling molecule found in most animal cell types that plays a role in inflammation and tissue damage.

The study authors previously found out that NF-κB is active in dystrophin-deficient muscle years before the onset of symptoms, suggesting that very early treatment of Duchenne Muscular Dystrophy patients with VBP15 may prevent or delay the onset of some clinical symptoms.

“It is becoming increasingly clear that membrane integrity and repair are crucial factors in muscle, cardiovascular, neurodegenerative and airway disorders. The chemical properties of VBP15 also suggest potential for the treatment of other diseases.” remarked Kanneboyina Nagaraju, DVM, PhD, the lead author of the study and a principal investigator in the Center for Genetic Medicine Research, Children’s National Medical Center in Washington, DC.

The authors conclude that VBP15 merits further investigation for efficacy in clinical trials.

The study was funded in part by the National Institutes of Health, the US Department of Defense, Muscular Dystrophy Association, Foundation to Eradicate Duchenne, and CureDuchenne Foundation.


Further Information
Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 2,400+ scientific posters on ePosters
  • More than 3,700+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

Endocrine Disruptors Impair Human Sperm Function
Endocrine disruptors may contribute to widespread fertility problems.
Tuesday, May 13, 2014
EMBO Announces 52 New Members for 2013
EMBO announced today that 52 outstanding researchers in the life sciences were newly elected to its membership.
Tuesday, May 21, 2013
EMBO, EMBC and the National Science Council of Taiwan Sign Cooperation Agreement
New ways of global scientific interaction have been created following a cooperation agreement between EMBO, the European Molecular Biology Conference (EMBC), and the National Science Council of Taiwan (NSC).
Thursday, November 29, 2012
Scientific News
Sorting Through Cellular Statistics
Aaron Dinner, professor in chemistry, and his graduate student Herman Gudjonson are trying to read the manual of life, DNA, as part of the Dinner group’s research into bioinformatics—the application of statistics to biological research.
Women’s Immune System Genes Operate Differently from Men’s
A new technology reveals that immune system genes switch on and off differently in women and men, and the source of that variation is not primarily in the DNA.
Experimental MERS Vaccine Shows Promise in Animal Studies
A two-step regimen of experimental vaccines against Middle East respiratory syndrome (MERS) prompted immune responses in mice and rhesus macaques, report National Institutes of Health scientists who designed the vaccines.
HIV Susceptibility Linked to Little-Understood Immune Cell Class
High levels of diversity among immune cells called natural killer cells may strongly predispose people to infection by HIV, and may be driven by prior viral exposures, according to a new study.
New Weapon in the Fight Against Blood Cancer
This strategy, which uses patients’ own immune cells, genetically engineered to target tumors, has shown significant success against multiple myeloma, a cancer of the plasma cells that is largely incurable.
Scientists Create CRISPR/Cas9 Knock-In Mutations in Human T Cells
In a project spearheaded by investigators at UC San Francisco, scientists have devised a new strategy to precisely modify human T cells using the genome-editing system known as CRISPR/Cas9.
Researchers Develop Vaccine that Protects Primates Against Ebola
A collaborative team from The University of Texas Medical Branch at Galveston and the National Institutes of Health have developed an inhalable vaccine that protects primates against Ebola.
Universal Flu Vaccine in the Works
A new study has demonstrated a potential strategy for developing a flu vaccine with potent, broad protection.
Immunotherapy Shows Promise for Myeloma
A strategy, which uses patients’ own immune cells, genetically engineered to target tumors, has shown significant success against multiple myeloma, a cancer of the plasma cells that is largely incurable.
Immune System 'On Switch' Breakthrough Could Lead to Targeted Drugs
A crucial 'on switch' that boosts the body's defenses against infections has been successfully identified in new scientific research.
SELECTBIO

Skyscraper Banner
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
2,400+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
3,700+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FREE!