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King's College London - Health: More than a medical matter

Gene therapy hope for muscle disorder

30 March 2006

By BioNews

Appeared in BioNews 352

US researchers have begun a clinical trial to test a new gene therapy treatment for Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disorder. The team, based at the Columbus Children's Hospital in Ohio, will test the safety and effectiveness of gene injections in six affected boys aged 8-12 years. Scientists at the Universities of North Carolina and Pittsburgh have spent 20 years developing the new treatment, which involves the use of a 'miniaturised' version of a gene that makes a crucial muscle protein.    

DMD is a serious genetic disorder that mainly affects boys. It is caused by mutations in the dystrophin gene, which makes one of a cluster of proteins that hold muscle cells together. Although the main symptom of the condition is progressive wasting of the muscles used for movement, the heart is also often affected. Efforts to develop a gene therapy treatment for DMD have been hampered by the unusually large size of the dystrophin gene, which is too big to deliver using standard methods. The new trial will use a trimmed-down version of the gene, called 'minidystrophin', which will be delivered using a modified adeno-associated virus (AAV).

The six boys will receive injections of the replacement gene into the bicep of one arm, and injections of a placebo into the other. After six weeks, the researchers will analyse samples of muscle and test the strength of both arm muscles, to see if the treatment - dubbed Biostrophin - makes a difference. The scientists, who formed the company Asklepio BioPharmacuetical (AskBio) to make and test the treatment, obtained approval to proceed with the trial from the US Food and Drug Administration (FDA) on 3 March. 'After years of encouraging pre-clinical results, I'm excited that AskBio will help bring this promising new therapy into the clinic', said team leader Jude Samulski.

The first boy to receive the treatment was eight-year-old Andrew Kilbarger, who was given three injections in each arm on 29 March. His parents hope that by taking part in the research, funded by the US Muscular Dystrophy Association, they might one day improve the outlook for other boys affected by the disease. 'We don't hit the moon with our first shot', said Andrew's mother, Julie Kilbarger, adding 'somebody has to step up. Somebody has to be the first one'. The scientists hope that eventually, it will be possible to inject the gene treatment at higher doses, and into the patients' legs. 'Then, we really could improve the quality of life', said neurologist Jerry Mendell, who is running the trial.


Boy, 8, is pioneer for gene therapy
The Columbus Dispatch | 29 March 2006
EurekAalert | 29 March 2006


25 October 2009 - by Marianne Neary 
A potential new gene therapy for Duchenne Muscular Dystrophy (DMD) is now set to enter clinical trials after encouraging results in mice. The multinational team of scientists, publishing their findings in the journal Molecular Therapy, demonstrated that the therapy dramatically prevented severe muscle deterioration and extended the lifespan of mice with symptoms of DMD.... [Read More]
27 January 2008 - by Dr Rebecca Robey 
Researchers at the University of Texas Southwestern Medical Centre have developed a new technique to treat the symptoms of muscular dystrophy using embryonic stem (ES) cells. The group, reporting in the February 2008 issue of Nature Medicine, successfully manipulated mouse ES cells to transform them into muscle-forming... [Read More]
24 September 2007 - by Ailsa Stevens 
The first trial of a new treatment for Duchenne muscular dystrophy (DMD) will start later this year in the UK. The treatment, which has been developed by using human cells and mice experiments, hopes to overcome the effects of the genetic defect that causes the muscle wasting... [Read More]
03 April 2006 - by BioNews 
A team of scientists based in Frankfurt, Germany, has become the world's first to treat a rare and 'incurable' hereditary immune disease in adults, using gene therapy. Gene therapy has been used successfully in the past, but this has generally been in the treatment of children. Over the past two... [Read More]

03 April 2006 - by BioNews 
A team of scientists based in Frankfurt, Germany, has become the world's first to treat a rare and 'incurable' hereditary immune disease in adults, using gene therapy. Gene therapy has been used successfully in the past, but this has generally been in the treatment of children. Over the past two... [Read More]
26 July 2004 - by BioNews 
Researchers have overcome one of the major hurdles in using gene therapy to treat muscle diseases: delivery of the gene to muscles throughout the body. The study, to be published in the journal Nature Medicine next month, is further progress towards treating diseases such as Duchenne muscular dystrophy (DMD), which... [Read More]
29 March 2004 - by BioNews 
Gene therapy trials for haemophilia, muscular dystrophy and childhood blindness are to receive £3 million funding, the UK Department of Health has announced. A further £1 million has been set aside for research into the long-term safety of some gene therapy techniques. The funding is part of a £50 million... [Read More]
18 February 2004 - by BioNews 
Rats injected with a gene that promotes tissue growth develop stronger muscles after regular exercise, according to research presented at the annual meeting of the American Association for the Advancement of Science (AAAS) in Seattle. Scientists based at the University of Pennsylvania in Philadelphia, US, are hoping to develop new... [Read More]
14 July 2003 - by BioNews 
Two new studies promise new treatments for the muscular dystrophies, a group of genetic conditions that cause progressive muscle weakness. Italian researchers have combined stem cell and gene therapy techniques to successfully treat mice with symptoms of limb-girdle muscular dystrophy, while a UK group has used a novel form of... [Read More]

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