New directions in leukaemia research

Rasko will address the New Directions in Leukaemia Research Conference, being held on the Sunshine Coast from March 30 to April 2, on another area of leukaemia research that his team is actively pursuing - the role of two zinc finger transcription factors called BORIS and CTCF and their implied role in cancer.

"Our studies have taken us a long way towards seeing how the pathway of these two important genes relates to cell development and control of cell proliferation and differentiation. We have a small group of half a dozen who are exploring the interactome of these zinc finger transcription factors to see what these molecular machines are actually composed of so we can basically understand their mechanism of action."

BORIS and CTCF are part of the basic research aspect of Rasko's work. "I embrace the idea that in order to achieve successful gene therapy you have to identify the genes involved in a given disease, and genes of course can't work outside the context of cells. So in order to understand how to improve gene therapy, you have to not only understand the root cause of any given disease but you also have to understand the target cells and how to alter them genetically in order to achieve that therapy."

A lot of the team's work is focused on improving gene therapy and gene transfer into haemopoietic cells, specifically using adenovirus, adeno-associated virus, retrovirus and lentivirus vectors.

"We've also collaborated in delivering oncogenes and the 'Yamanaka genes' into stem cell types," he says. "Our focus and our role in that has been in improving the gene delivery. Refining the viral vector technology to improve the efficiency of gene delivery rather than exploring those particular genes in of themselves.

"We have collaborations where we are helping modify embryonic stem cells to achieve the kind of results that Yamanaka has achieved and also to deliver the genes involved in CML so we can explore the biology of these diseases."

Embracing the future

So, basic, translational and clinical research are all feathers in John Rasko's cap. He's a working haematologist, however, so is obviously interested in clinical applications and how we can improve treatments for patients. He's particularly concerned at how difficult it is to run clinical trials in Australia, particularly long-term ones, and would like to urge the new Minister for Innovation, Industry, Science and Research to pay some attention to this field.

"I'd like to congratulate the minister on continuing on with the important NCRIS initiative," he says. "That is extraordinarily important for promoting cell therapeutics in Australia. I want to encourage him to continue on that theme, to specifically invest in both the clinical as well as the basic aspects of cell therapeutics. In other words, divert specific funds to those initiatives.

"One of the difficulties in some of these areas is that because they are exclusively clinical in their applicability they oftentimes don't get the immediate attention of the standard grant review process. Having sat on such committees for many years, the basic research tends to succeed and get the grants, whereas the clinical has much more difficulty. The translational is very much third place in line, but if you are trying to establish a rationale and preliminary data to underpin a clinical trial, it is almost impossible to get funds to do that in Australia.

"For example, we started our haemophilia gene therapy trial in 2001. It was put on hold for various reasons by the regulatory authorities, but the current trial that we have now requires us to monitor our subjects for 15 years. There is no means by which that can be funded in Australia. There is no funding body that is prepared to commit to funding to follow a recipient of gene therapy for that long, but that's exactly the kind of time-frame we need. These kinds of things are crucial, the kind of Clever Country innovations, that the minister might be looking towards.

"We have an opportunity in Australia, we have such respect internationally, but we have lost some of our key stem cell researchers, for example, and I won't hesitate to mention who they are - Paul Simmons, Martin Pera and Alan Trounson - and that haemorrhage will continue whenever we hesitate to embrace the future. We've had our Senate meetings, we've gone past the time when these things are the subject of serious controversy. Every opportunity is there for governments to really make a mark in the f