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Stem cell breakthrough gives hope to those affected by muscle wasting diseases

9 March 2009
Appeared in BioNews 498

It was reported this week in the journal Stem Cell that a group of researchers from the University of New South Wales, Australia, have made a major breakthrough in the success of regrowing damaged muscle tissue using adult stem cells.

Previous research carried out at the University of Texas Southwestern Medical Centre, reported by BioNews in January 2008, outlined the technique of adapting embryonic stem cells (ES cells) from mice to treat the same genetic disorder that causes Duchenne muscular dystrophy (DMD) - a form of muscular dystrophy in humans.

Then, the lead author of the paper, Professor Peter Gunning, reported that the previous problem facing this type of muscle regeneration was that 'when you introduce stem cells they tend to get out-competed by the locals and they disappear quite quickly'. The success of the UNSW research is that the Australian team have found a method by which the inserted donor stem cells are not killed off by the immune system or the damaged host tissue cells.

The method used to achieve these results is that adult stem cells are given a gene which makes them immune to chemotherapy treatment. Once the stem cells are implanted, chemotherapy is then used to destroy the host cells and allow the donor cells to propagate and create new healthy muscle tissue. Professor Gunning explained: 'What this will do is provide a provide a way in which you can give an advantage to the incoming stem cells so that they can stick around and do what they need to do, which is actually repair the muscle and turn it into healthy muscle'.

The direct focus and hope for such a breakthrough is for muscle wasting diseases such as muscular dystrophy - the term used to describe a variety of hereditary muscle wasting diseases. It has also been suggested that this success could eventually go towards helping people who undergo chemotherapy to treat cancer. 'Chemotherapy can have quite adverse effects on muscles and you can reach a point called cachxia where you've got extraordinary amounts of muscle wasting,' Professor Gunning said, adding: 'To help regrow the muscle you can imagine using this kind of approach'.

The trials using this procedure are still at the pre-clinical stage and the success has all been found in mice. However, researchers are hoping to begin human trials on specific forms of muscular dystrophy within three to five years.

Aust scientists develop stem cell survival technique
ABC News |  5 March 2009
Muscles find their stem cell niche
Life Scientist |  6 March 2009
Muscular Dystrophy: Stem Cell Breakthrough Gives New Hope To Sufferers Of Muscle-wasting Diseases
ScienceDaily |  6 March 2009
Stem cell breakthrough by Sydney scientists
Sydney Morning Herald |  5 March 2009
13 June 2011 - by Sarah Pritchard 
The NHS must be prepared to screen every newborn baby for Duchenne muscular dystrophy (DMD) – the most severe form of muscular dystrophy – in three years time, says a leading UK geneticist who explains that promising treatments are close to becoming a reality...
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...
7 January 2008 - by Ailsa Stevens 
An experimental treatment for boys with the inherited muscle wasting disease Duchene Muscular Dystrophy (DMD) has showed promise in human safety trials, according to a study published in the New England Journal of Medicine. In the first ever trial on humans, the new drug was shown to...
17 December 2007 - by Ailsa Stevens 
Adult stem cells harvested from human patients with the muscle wasting disease Duchene Muscular Dystrophy (DMD) can be genetically corrected and used to improve muscle strength in mice with DMD, according to a study published in the journal Cell Stem Cell last week. The researchers, based at...
19 November 2006 - by Heidi Nicholl 
A team of scientists based in France and Italy have published work showing a huge improvement in the condition of dogs suffering from a canine version of muscular dystrophy when treated with adult stem cells. Duchenne muscular dystrophy is a serious disease that affects 1 in every...
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