The majority of adults and 20% of children with Acute Lymphoblastic Leukaemia (ALL) relapse and die of their disease. Recent advances in understanding the biology driving ALL have been discovered with the aid of powerful new molecular technologies. Specifically, the use of transcriptome (genetic) analysis has identified genetic changes in a significant number of ALL patients which result in the formation of new proteins. Importantly, drugs which target these proteins are in use in the clinic for other diseases and are safe. We will develop new tests to rapidly identify these patients at diagnosis, and assess the efficacy of these drugs in a clinical trial, identifying treatment regimens which will improve the generally low cure rates in these patients. We anticipate the outcome for these patients will improve significantly using this approach.
What we aim to achieve
These studies will culminate in a clinical trial, as well as correlative studies designed to assess the effectiveness of targeted therapies in this setting. This study is designed to fully assess high risk ALL patients on an individual basis and evaluate the effectiveness of new therapeutic strategies. It is anticipated that this study will result in significantly improved outcomes for these patients and in a paradigm shift in treatment regimens in this fatal disease.
Our next steps and milestones
Our next steps are to establish the Acute Leukaemia Registry, and to identify the frequency of ALL patients who are eligible for these treatments in Australia. We also aim to begin a Phase II clinical trial to assess the effectiveness of targeted therapies in this disease, while working to develop new targeted therapies for this disease.
What motivates me
I am motivated by the challenge of understanding the underlying basis, both genomic and biological, of leukaemia, such that new targeted therapeutic strategies may be implemented. This will lead to reduced toxicity and better survival for our vulnerable young and also adult patients. This is critical research, which along with our other projects motivates me on a daily basis.
My message to supporters
Acute Lymphoblastic Leukaemia (ALL), a malignant disorder of lymphoid progenitor cells, affects both children and adults. ALL remains one of the most challenging adult malignancies, and while it is largely considered to be a curable disease in children, approximately 20% relapse. Significantly, the overall disease free survival rate across all ages remains less than 50%.
Without research funding this project and subsequent linked projects cannot be undertaken. We are significantly indebted to the generous supporters of Cancer Council SA who have made this project possible.