24 February 2005

The Muscular Dystrophy Campaign is encouraged by the announcement that a clinical trial using MYO-029 will be conducted in the United States by the pharmaceutical company Wyeth.

It is anticipated that centres in the United Kingdom will become involved once regulatory and ethical approval has been obtained. MYO-029 is an inhibitor of myostatin, a naturally occurring protein that regulates the growth of muscle. There is currently no cure or treatment to halt the breakdown of muscle cells associated with muscular dystrophy and trials such as these raise the hope that effective treatments for muscle wasting conditions may be found.

This initial safety trial will focus on adults with Becker-, facioscapulohumeral- and limb-girdle muscular dystrophy.

When myostatin is produced naturally in the body it acts to reduce the amount of muscle formed. Research has shown that blocking the action of myostatin can promote extra muscle growth. In 2004 there was a report of a child who had a mutation (error) in the myostatin gene and, although very young, he is considerably larger and appears stronger than his counterparts. This increases the hope that by inhibiting the formation of myostatin it might be possible to make more muscle in those with muscular dystrophy.

Jenny Versnel, Head of Research at the Muscular Dystrophy Campaign says, "Ultimately, in order for the treatment to be truly beneficial it is important that muscle strength is also increased. This and future trials will be key in determining whether this is the case. The underlying genetic cause of muscular dystrophy would remain, but the hope is that a treatment such as this could slow down the progression of muscular dystrophy".

Diane Yeo, acting Chief Executive of the Muscular Dystrophy Campaign said, "These trials will be crucial in determining whether blocking myostatin is useful for the approximately 14,000 people in the UK with muscular dystrophy. We desperately hope it will be. Research into muscular dystrophy is under funded, even though there are a number of research routes that might be promising and need investment. The move to clinical trials by Wyeth is therefore, particularly welcome."

Details of the trial and the participating centres can be found at www.clinicaltrials.gov