Sean Stamp is excited by news of the drug for muscular dystrophy sufferers. Photo / Greg Bowker
New Zealand scientists have developed a breakthrough drug that gives hope to people suffering from muscular dystrophy.
The drug is aimed at Duchenne muscular dystrophy, which affects only males and is most commonly inherited.
Boys with it can often have a normal early childhood, but are forced into
a wheelchair around the age of 10 as their muscles weaken. On average they die by 20, their heart and lungs no longer able to work properly.
But an announcement yesterday by Dunedin biotechnology company Orico has brightened this dark outlook.
The company is developing a drug designed to promote muscle regeneration, halting or delaying the progression of the disease.
It temporarily interrupts the body's normal process of controlling the growth of muscle tissue.
The first drug of its type, it was discovered by scientists at AgResearch, a part-owner of Orico.
"It's really exciting that it's potentially a treatment," said the Muscular Dystrophy Association's liaison officer, Miriam Rodrigues.
She said there were currently no therapies on the market to delay or halt muscular dystrophy, although several were in human trials overseas. Cortico-steroid drugs and breathing ventilation machines were used to treat symptoms.
Sean Stamp, who has Duchenne muscular dystrophy, shares her excitement about the drug.
"It looks pretty promising from what I can see," said the 25-year-old, of Pukekohe, who has been in an electric wheelchair since the age of 13.
He has lost a lot of strength from his arms, but continues to use a computer and plays electric-wheelchair hockey.
Mr Stamp said he did not take cortico-steroids but had benefited from physiotherapy, correct positioning in his wheelchair and surgery to correct the spinal curvature that was common with his condition.
Orico chief executive Rob Bower said if the drug proved safe and effective it could be on sale in six years. The company intended to test it for toxicity in animals next year and in the first of several human clinical trials in 2009.
Dr Bower said: "We found after eight weeks' treatment in some mice we couldn't tell the difference between normal mice and the mice that had [muscular dystrophy].
"We were very largely able to get rid of the symptoms."
