Navigation Links
Human protein improves muscle function of muscular dystrophy mice
Date:12/27/2010

PROVIDENCE, R.I. [Brown University] A novel potential therapy based on a natural human protein significantly slows muscle damage and improves function in mice who have the same genetic mutation as boys with the most common form of muscular dystrophy, according to a paper published online Dec. 27 in the Proceedings of the National Academy of Sciences.

Duchenne Muscular Dystrophy is a fatal genetic mutation in about one of every 3,500 boys. They are unable to produce a protein called dystrophin that keeps muscles strong. By eight years of age, the boys begin to have trouble walking. By their teens they are often in wheelchairs, and by their 20s muscle function is so degraded that they die.

"This is all aimed at getting a therapy that will meaningfully improve the condition of patients," said Justin Fallon, professor of neuroscience at Brown University and the senior author of the paper. "This is an important step along that path."

This fall, the startup company Tivorsan Pharmaceuticals licensed rights from Brown to the key protein, biglycan, hoping to bring the potential therapy through clinical trials.

Biglycan restores the muscle-strengthening presence of a protein called utrophin, which is normally prevalent only in very young children. Utrophin still exists in adults, but in fewer places and not where it can help muscular dystrophy sufferers who cannot produce dystrophin, which keeps adult muscles strong.

Encouraging experiments

In experiments described in the paper, Fallon's team showed that biglycan delivered to the bloodstream draws utrophin to the cellular membranes of muscle cells. Much as utrophin does when it is present in fetuses, infants and toddlers, the protein works to help the cells build and retain their strength.

In one experiment, Fallon's team found a 50-percent reduction in "centrally nucleated" fibers in the muscle tissue of biglycan treated mice compared to untreated mice. Biologists recognize the fibers as indicators of recent tissue damage and repair, so a reduction in them suggests that the muscle tissue is suffering less damage.

The team also subjected mouse muscles to a standardized stress test where they are simultaneously stretched and caused to contract. The test ultimately weakens even healthy muscle, but in the tests conducted by researchers and co-authors from the University of Pennsylvania, the muscles of muscular dystrophy mice treated with biglycan lost their strength 30 percent more slowly than similar mice who were untreated.

In more recent tests using an improved formulation of biglycan, Fallon said, the team has seen that figure rise to 50 percent in some muscles, meaning that mice treated with biglycan are holding on to more of their function for a longer time.

Fallon said the effects of treatment with biglycan lasted through months of testing. Several basic tests for side effects during that time frame, such as kidney and liver function, did not indicate any harm from the therapy.

With the efficacy of biglycan apparent in the mouse model of Duchenne Muscular Dystrophy, Fallon is eager to see if it can improve the lives of thousands of children.

"The next big step is testing in humans," Fallon said.


'/>"/>

Contact: David Orenstein
david_orenstein@brown.edu
401-863-1862
Brown University
Source:Eurekalert

Related medicine news :

1. Actor and Humanitarian Harrison Ford Assists Operation Smiles Relief Efforts in Haiti
2. Human Dental Technology Leads to Breakthroughs for Pet Dental Health at Sergeants Pet Care Products, Inc.
3. Scripps Health Begins Pioneering Study of Human Tumor Sequencing in Cancer Patients
4. New drug for kidney transplant recipients effective in humans
5. Agility Consulting Partners with pan (Performance Assessment Network) to Bring Custom Private Label Assessment Capability to Human Resource Professionals and Consultants
6. Dolphins health shed light on human and ocean health
7. Dolphins could be ideal model to study human cervical cancer, UF veterinarians say
8. Dolphins May Warn of Health Risks in Humans
9. Inadequate access to opioid-based pain relief is a human rights issue for cancer patients
10. Sociologist expands US conversation on human rights
11. State of Oregon Department of Human Services Selects Netsmart Technologies for Comprehensive Behavioral Healthcare Integration Project
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/22/2017)... ... 22, 2017 , ... An inventor from Columbia, S.C., wants to offer people ... was damaged in a firework accident, so she couldn’t grip a pen properly," he ... He then designed and created a prototype for the HELPEN HAND, a special pen ...
(Date:5/22/2017)... ... , ... Galena, Alaska, has a population of less than 500 people. But, ... targeted to build a nuclear plant by the TOSHIBA Corporation. , In “The Galena ... describes the journey to get power to his small town, off the electrical grid. ...
(Date:5/22/2017)... , ... May 22, 2017 , ... ... a program on Wellocity to empower WeightZone Factor members to track their exercise ... get an excellent health education, all on their mobile phones. It also provides ...
(Date:5/21/2017)... (PRWEB) , ... May 19, 2017 , ... Chris Cornell, 52, was found dead on ... why one of the most respected lead singers in the rock industry would take his ... of mental illness. FindaTopDoc investigates how mental illness played a role in the death of ...
(Date:5/21/2017)... ... May 20, 2017 , ... GetLinked® software ... and financial systems. , Dozens of clubs using ABC Financial Services are ... through GetLinked into their club’s accounting system , The latest implementation is ...
Breaking Medicine News(10 mins):
(Date:5/10/2017)... 10, 2017 Radiology has become the number ... have also spiraled to the number one ranking as ... than ever before as the most complete and reliable ... with lower back pain an MRI may confirm a ... pain, resulting in entirely different treatment protocols.  In these ...
(Date:5/9/2017)...  Oramed Pharmaceuticals Inc. (NASDAQ: ORMP ... focused on the development of oral drug delivery ... Office has granted Oramed a patent titled, "Methods ... patent covers Oramed,s invention of an oral glucagon-like ... incretin hormone that stimulates the secretion of insulin ...
(Date:5/6/2017)... Texas , May 5, 2017   Provista , ... with more than 200,000 customers, today announced Jim Cunniff ... a wealth of executive and business experience to Provista, including ... compounding pharmacy in California . He assumed ... "Jim is a great fit for Provista," says ...
Breaking Medicine Technology: