Australian scientists say they are hoping to soon begin human trials of a new class of experimental drug, which would act as a "molecular assassin" of diseases causing genes.
Researchers at Sydney's University of New South Wales said the smart drug had the potential to treat a range of illnesses including cancer, heart disease and inflammatory illnesses such as arthritis and eye disease.
Professor Levon Khachigian said the drug, Dz13, was revolutionary because it was made of genetic DNA or RNA material and was designed to target and destroy disease-causing genes known as mass-regulators. RNA is an intermediary stage between DNA and protein.
"Our assassin is a potential new class of drug," Khachigian said. "This is made of DNA or RNA and serves as a magic bullet which seeks out and destroys bad genes.
"This is kind of like a bull's eye technology. The desired effect is for it to hit the core of the mechanism of disease which is to go after the godfather (mass regulator)."
Khachigian said one of the benefits of the drug is that it could be used very early in the disease process, and that it could be used to treat more than one type of illness. "Because we are focusing on the godfather genes we should be able to use this technology in more than one disease type," he said.
"It's not just eye disease, it's the narrowing of the arteries, it's the growth of solid tumours, it's arthritis," he said.
Khachigian said researchers hoped to start trials on humans early next year with skin cancers likely to be the first disease targeted.
"It hasn't been yet tested on humans and that's what we have to be very careful about," he said.
Khachigian said there were still many unknowns about the effectiveness of the drug, despite some pleasing results from animal trials.
"Whether or not our drug is best used alone or whether it is best used in combination with other drugs remains to be seen," he said.
"But we can add this to the tool box of the therapeutic alternatives." The research is published in the July issue of US journal Nature Biotechnology.
Sapa - AFP
Researchers at Sydney's University of New South Wales said the smart drug had the potential to treat a range of illnesses including cancer, heart disease and inflammatory illnesses such as arthritis and eye disease.
Professor Levon Khachigian said the drug, Dz13, was revolutionary because it was made of genetic DNA or RNA material and was designed to target and destroy disease-causing genes known as mass-regulators. RNA is an intermediary stage between DNA and protein.
"Our assassin is a potential new class of drug," Khachigian said. "This is made of DNA or RNA and serves as a magic bullet which seeks out and destroys bad genes.
"This is kind of like a bull's eye technology. The desired effect is for it to hit the core of the mechanism of disease which is to go after the godfather (mass regulator)."
Khachigian said one of the benefits of the drug is that it could be used very early in the disease process, and that it could be used to treat more than one type of illness. "Because we are focusing on the godfather genes we should be able to use this technology in more than one disease type," he said.
"It's not just eye disease, it's the narrowing of the arteries, it's the growth of solid tumours, it's arthritis," he said.
Khachigian said researchers hoped to start trials on humans early next year with skin cancers likely to be the first disease targeted.
"It hasn't been yet tested on humans and that's what we have to be very careful about," he said.
Khachigian said there were still many unknowns about the effectiveness of the drug, despite some pleasing results from animal trials.
"Whether or not our drug is best used alone or whether it is best used in combination with other drugs remains to be seen," he said.
"But we can add this to the tool box of the therapeutic alternatives." The research is published in the July issue of US journal Nature Biotechnology.
Sapa - AFP
