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Anti-cancer hope for 'assassin' drug
Pia Akerman and Adam Cresswell
04jul06

THE first of a new class of smart drugs that could one day lead to 
treatments for heart disease, skin cancer and blindness is under 
development by Australian scientists.

The drug, Dz13, has shown encouraging results in animal studies and 
could lead to better treatments that avoid the unpleasant side 
effects of existing therapies such as chemotherapy. 
Announcing the research in Sydney yesterday, lead scientist Levon 
Khachigian said the "molecular assassin" drug targeted a specific 
gene called c-Jun,which controls disease-causing proteins. 

The gene is essential for human growth, but has been linked to 
serious health problems when activated during adulthood. 

Professor Khachigian said that targeting this "godfather" gene could 
potentially aid in the treatment of solid tumours and certain skin 
cancers, specifically basal cell carcinoma, one of the most 
frequently occurring in Australia. 

Laboratory trials on animals showed the drug to be highly effective 
in restricting the growth of skin cancers and tumours by choking off 
the blood supply to the cancerous cells. 

Hindering blood supply also helps prevent age-related macular 
degeneration, the leading cause of blindness and severe vision 
impairment in Australia. 

No major side effects were recorded. 

Bernard Stewart, head of the cancer control program of theSouth 
Eastern Sydney and Illawarra Health Service, said thedrug "represents 
the new generation of specific gene-targeting therapy in cancer and 
other diseases". 

"The biological models that Professor Khachigian has described are in 
fact the key stage that indicate that we have gone from something on 
paper that might work to something in biology that does work," 
Professor Stewart said. 

The University of NSW study, published in the latest edition of the 
journal Nature Biotechnology, has shown that use of gene-targeting 
therapies could also have significant benefits for cardiac patients. 

When targeting a different gene, the new drug has been shown to 
reduce the severity of a heart attack by up to 50 per cent by 
limiting the damage caused to the heart muscle. 

Cardiologist Ravinay Bhindi said the findings were "exciting and are 
being explored in further studies, and they also highlight another 
potential area that these smart drugs can be used in the future". 

Professor Khachigian said it was too early to tell whether the new 
gene-targeting drugs would be used alone or with other drugs. He 
estimated they would not be available to the general public for 
several years. 

A human trial is scheduled for early next year. 

People suffering certain types of skin cancers and blindness due to 
age-related macular degeneration and diabetic retinopathy are likely 
to be among the first users of the drug. 

Professor Stewart said the drug could help the hundreds of thousands 
of Australians affected by non-melanoma skin cancers who usually have 
to resort to surgery




 
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