Scientists hail new drug's potential

http://www.theage.com.au/national/scientists-hail-new-drugs-potential-20080817-3x2v.html?page=-1

Scientists hail new drug's potential

* Jill Stark
* August 18, 2008

MELBOURNE scientists have developed a drug that may prevent a condition responsible for up to 45% of all deaths in the Western world.

The drug's potential is so great it could mark an end to dialysis for patients with kidney disease and reduce the need for organ transplants.

Scientists from the University of Melbourne, St Vincent's Hospital and Bio21 Institute say the breakthrough could rival Ian Frazer's cervical cancer vaccine in putting Australia on the world map of medical discoveries.

The drug works by preventing the spread of fibrosis - the irreversible scarring of vital organs that underpins many major killers such as kidney disease, heart failure, diabetes and lung and liver problems. As fibrosis advances, it causes such extensive damage that the organ shuts down, eventually resulting in death.

There is no available treatment that prevents or halts the spread of the condition and scientists internationally are racing to be the first to produce an antifibrotic drug.

The Melbourne discovery, named FT-11, has created such international excitement that the US Government has pledged up to $3 million to accelerate the drug to market - the first time such a grant has been awarded in Australia.

The Melbourne project has had a 100% success rate in trials on rats and will be trialled on about 30 Victorian patients within months.

The compound not only produces dramatic improvements but displays no side effects.

Scientists estimate the drug's worldwide market would be worth up to $2 billion in the first year alone. It would also be a financial coup worth hundreds of millions of dollars for Melbourne University because it shares patent rights for the drug through Fibrotech Therapeutics.

Associate Professor Darren Kelly, who led the two-year quest to develop the drug, said: "This is the first drug of its kind in the world and the implications are enormous. If this comes to fruition, and we're confident with the results so far … it would be up there with the cervical cancer vaccine (Gardasil), even bigger. It would be the biggest blockbuster since blood- pressure-lowering drugs."

The drug will initially be developed to treat people with kidney disease caused by diabetes. Fibrosis plays a key part in the progression of the disease, but the indications are that if patients were given the drug in the early stages, their condition would not worsen.

"This would basically mean taking one tablet a day rather than requiring eight hours of dialysis every few days," Professor Kelly said.

Dr Tim Mathew, medical director of Kidney Health Australia, said fibrosis caused the majority of chronic kidney diseases. "There are currently 10,000 Australians on dialysis and life for them is very demanding. Any drug that could stop the onset of scarring to the kidney would be a major advance" Dr Mathew said.

The drug works by inhibiting the production of a protein that causes fibrosis. Importantly, the FT-11 compound has been developed to ensure that it prevents abnormal scar tissue but does not impair healthy scarring such as that formed following surgical procedures.

The drug could also be used to prevent diabetes.

Professor Kelly said that if clinical trials went as planned the drug would be available within eight to 10 years. The drug is being commercialised in collaboration with Melbourne Ventures.