|New wonder drug for genetic conditions could be on the market by 2010|
|16 September 2008|
RESEARCHERS in the States hope a new drug which would revolutionise the treatment of genetic disorders such as muscular dystrophy and haemophilia will be available within two years.
Early trials into PTC Therapeutics’ PTC124 formula show the simple packet of powder can make cells less sensitive to the genetic mutations associated with around 2,400 health problems including cystic fibrosis (CF). Given early enough, there is even evidence the drug can halt the progress of some genetic illnesses, many of which are incurable.
Researcher Dr Stuart Peltz explained: “Many inherited diseases are caused by mutations in genes, which stop cells from making vital proteins and, at present, there are many serious illnesses of this type for which there is currently no effective treatment. We hope to be able to bring this new drug to market, not only quickly for cystic fibrosis, but over time to many different patients with many different diseases for which there is currently no treatment.”
In cystic fibrosis a mutation stops the body’s production of a protein called CTFR. Its absence causes the body’s fluids to become abnormally thick and sticky, causing breathing and digestive problems.
In tests on CF sufferers, PCT124 boosted production of the protein and improved breathing. It is thought the drug could help 10 per cent of the 8,000 UK CF sufferers.
Tests on youngsters with Duchenne muscular dystrophy have also proved promising.
Dr Peltz said: “The difference between this and other drugs is it doesn’t just treat the symptoms, it treats the underlying cause. The drug allows the body to make the lost protein.”
It is hoped PCT124, which would have to be taken daily for life, could be on the market as early as 2010 as widespread trials continue.