Gene therapy success stories? The debate in Australia -
Perinatal Society of Australia and New Zealand, 5th Annual Congress, Canberra
The first 'medical miracles' from gene therapy are occurring around us - using DNA as an alternative to conventional drugs - according to genetics specialist, Professor Ron Trent, from the University of Sydney.
Professor Trent is presenting latest developments into the future of gene therapy at the Perinatal Society of Australia and New Zealand (PSANZ) 5th Annual Congress in Canberra this week.
"While gene therapy (using DNA as a form of therapy) has been around for 10 years, the first true success story only took place a few months ago.
"In France in December last year, three children with the rare SCID (Severe Immuno Deficiency Disorder) disease - in which cells of the immune system don't function - were treated with gene therapy.
"SCID leaves infants fragile and infection-prone and in some cases means death within a year because there is no medical therapy available," Professor Trent said.
"These three children, all boys, now have normal immune cell function after receiving injections of normal genes into their immune cells."
He said the first clinical trials of gene therapy (on melanoma) in Australia were approved in 1995. "Most gene therapy studies underway in Australia are related to cancer."
Professor Trent said while Australia still needed to further expand its technology to allow DNA delivery to be more efficient and more long term, Australian scientists had an outstanding reputation in basic medical research and clinical trials.
However, Dr Panos Ioannou, from The Murdoch Children's Research Institute in Melbourne (also presenting a paper at the PSANZ Congress), has called for restraint in certain experiments into gene therapy.
"Certain gene therapy experiments are very risky in terms of creating new viruses. We should be concentrating on restoring the health of people, not playing around with injecting DNA before we can control the process very precisely," he said.
"Using the results of the recently completed Human Genome Project we should instead be learning how to turn on and turn off the function of genes within our bodies using new drugs. If we perfect this, we can turn on a certain gene to complement a defect within the body, not to necessarily correct that defect."
Professor Trent and Dr Ioannou will be presenting research papers and joining a debate on Genome Advances at the PSANZ Congress this Thursday 15 March 2001.