Muscular dystrophy treatment hope

SCIENTISTS in Ireland and Australia have made a big breakthrough in the development of a gene therapy that reverses the most aggressive form of muscular dystrophy.

There is no cure for the condition but the research offers real hope for a treatment that could counteract muscle wipeout.

Muscular dystrophy is a progressive muscle-wasting disorder in boys and is the most gender-specific inherited disease in Ireland.

Sufferers are usually wheelchair-bound by the age of 12 and the average life expectancy is about 20 years.

The condition affects one in 3,500 Irish boys and there are about 400 current sufferers nationally and millions throughout the world.

Research published yesterday by NUI Maynooth and the University of Western Australia proves that gene therapy can turn around the muscle wasting process.

By ‘exon skipping’ over the defective part of the dystrophic gene, the muscle produces an alternative protein product that re-establishes dystrophin and reverses muscle deterioration.

Prof Kay Ohlendieck, head of the biology department at NUI Maynooth, said the results were very encouraging.

“Our findings set the scene for improved treatment options to counteract the devastating symptoms of inherited muscle wasting disorders,” said Prof Ohlendieck.

The year-long collaboration between the two universities was funded by Muscular Dystrophy Ireland, the Health Research Board and Science Foundation Ireland.

Prof Ohlendieck said the next step in finding a cure for muscular dystrophy was to hold a human clinical trial.

Similar trials had already begun in the United States and Britain and with results expected in 2010.

Chief executive of Muscular Dystrophy Ireland, Joe Mooney, said it was hoped — and it was a big hope — that the therapy by injection would be available in the next two to three years.

“My personal hope is that when we get a treatment for the most aggressive form of muscular dystrophy, it is only a matter of transferring what has been learned to treat different types of the disease,” he said.

But Mr Mooney said the development of a treatment could take longer, pointing out that it was more than 25 years since scientists found the gene that causes muscular dystrophy and it was predicted at the time that a cure would be found within five years.

Muscular Dystrophy Ireland will launch their national awareness campaign on February 14 and is looking for volunteers to help with public collections and chocolate selling that will take place around the same time.

www.mdi.ie.