Navigation Links
MDA supports Duchenne muscular dystrophy research by University of Nevada School of Medicine
Date:8/22/2012

RENO, Nev. A novel treatment in development at the University of Nevada School of Medicine for the most common form of muscular dystrophy is advancing towards human trials with a $308,000 boost from the Muscular Dystrophy Association.

The grant will be used to expand successful research by Associate Professor of Pharmacology Dean Burkin that has shown his laminin-111 protein therapy prevents the onset of the devastating neuromuscular disease in mouse models.

"The early results for laminin-111 as a therapeutic agent for (Duchenne muscular dystrophy) are really exciting," Sanjay Bidichandani, MDA vice president of research, said. "We are eager to see this research go forward."

Duchenne muscular dystrophy is a lethal genetic disease that affects one in 3,500 newborn boys and is caused by mutations in the gene encoding dystrophin. There is no effective treatment or cure.

At the time of diagnosis, DMD patients usually have developed significant muscle disease. The three-year grant will help determine if Burkin's therapy is effective at preventing or reversing disease progression after onset.

"What we aim to discover now is if this therapy is effective at preventing or reversing disease progression after it has already started," he said. "First we will determine if laminin-111 prevents muscle damage after disease onset, preserves muscle function and improves survival of mouse models of DMD. Second, we will determine if laminin-111 prevents cardiomyopathy in mouse models of DMD.

"Finally, in collaboration with researchers at Texas A&M, we will determine if a human version of the protein prevents muscle disease in other animals afflicted with the disease. Results from these studies will pave the way towards developing human recombinant laminin-111 protein as a novel therapeutic for DMD."

Demonstrating that the human version has efficacy in animals with DMD will move this therapy closer to a Food and Drug Administration investigational drug application and potential human clinical trials.

"This MDA grant is a tremendous help in moving this technology forward," Burkin said. "Patients have been waiting a long time for therapies to come about and I think we're at the cusp of major discoveries. Theoretically, laminin-111 protein therapy should be able to treat all patients with Duchenne muscular dystrophy."

Since laminin-111 is a naturally occurring protein already present in our kidneys and other tissues, Burkin said there is a better likelihood that it would not be rejected by the human body.

A postdoctoral researcher in Burkin's lab, Ryan Wuebbles, also received an $180,000 MDA development grant over three years to investigate if laminin fragments can be therapeutic for the treatment of DMD. Wuebbles plans to test different parts of laminin-111 in cells cultured from animals and people with the disease. The most promising of these will be tested and compared to those generated by the full-length protein.

Burkin's work has been funded by the National Institutes of Health and he has a patent license agreement with a biotech company to develop the laminin technology.

His research has been profiled in the internationally acclaimed Better World Report, the American Journal of Pathology and the Proceedings of the National Academy of Sciences. He co-hosted the Myomatrix 2012 conference in April, which brought together 73 international scientists and clinicians to the University of Nevada, Reno campus to explore and share their latest findings and data on treatment breakthroughs related to congenital muscular dystrophy. The results of the Myomatrix Conference were published in the July issue of Neuromuscular Disorders.

In an article published in June in Science Translational Medicine Burkin summarized the impact of a new protein therapeutic, MG53, for the treatment of Duchenne muscular dystrophy.


'/>"/>
Contact: Mike Wolterbeek
mwolterbeek@unr.edu
University of Nevada, Reno
Source:Eurekalert  

Related biology news :

1. Study jointly led by UCSB researcher supports theory of extraterrestrial impact
2. NOAA science supports New Yorks offshore energy planning
3. Leading childhood asthma group supports federal asthma action plan to reduce disparities
4. New compound holds promise for treating Duchenne MD, other inherited diseases
5. New genetically engineered mice aid understanding of incurable neuromuscular disease
6. Real-life spider men using protein found in venom to develop muscular dystrophy treatment
7. Low oxygen levels may decrease life-saving protein in spinal muscular atrophy
8. Study by UC Santa Barbara researchers suggests that bacteria communicate by touch
9. Research reveals first evidence of hunting by prehistoric Ohioans
10. Diabetes Research Institute develops oxygen-generating biomaterial
11. APS issues new policy requiring identification of sex or gender in reporting scientific research
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
MDA supports Duchenne muscular dystrophy research by University of Nevada School of Medicine
(Date:4/5/2017)... , April 4, 2017 KEY FINDINGS ... to expand at a CAGR of 25.76% during the ... is the primary factor for the growth of the ... https://www.reportbuyer.com/product/4807905/ MARKET INSIGHTS The global stem ... technology, application, and geography. The stem cell market of ...
(Date:4/3/2017)... , April 3, 2017  Data captured ... engineering platform, detected a statistically significant association ... prior to treatment and objective response of ... potential to predict whether cancer patients will ... treatment, as well as to improve both pre-infusion ...
(Date:3/30/2017)... -- The research team of The Hong Kong Polytechnic ... by adopting ground breaking 3D fingerprint minutiae recovery and matching technology, ... accuracy for use in identification, crime investigation, immigration control, security of ... ... A research team led by Dr ...
Breaking Biology News(10 mins):
(Date:10/12/2017)... ... October 12, 2017 , ... The Blavatnik ... Winners and six Finalists of the 2017 Blavatnik Regional Awards for Young Scientists. ... Foundation and administered by the New York Academy of Sciences to honor the ...
(Date:10/12/2017)... CA (PRWEB) , ... October ... ... (https://www.onramp.bio/ ) has launched Rosalind™, the first-ever genomics analysis platform specifically designed ... bioinformatics complexity. Named in honor of pioneering researcher Rosalind Franklin, who made ...
(Date:10/11/2017)... ... October 11, 2017 , ... ... today it will be hosting a Webinar titled, “Pathology is going digital. Is ... , on digital pathology adoption best practices and how Proscia improves lab economics ...
(Date:10/11/2017)... ... October 11, 2017 , ... Singh Biotechnology ... drug designation to SBT-100, its novel anti-STAT3 (Signal Transducer and Activator of Transcription ... is able to cross the cell membrane and bind intracellular STAT3 and inhibit ...
Breaking Biology Technology: