A/Prof Louise Purton, Dr Meaghan Wall and A/Prof Carl Walkley
St Vincent's Institute of Medical Research
Myelodysplastic syndromes (MDS) are a group of malignant blood cell diseases that arise from blood-forming stem cells. Although we don't yet know why, MDS cells can develop into leukaemia.
We have identified a new protein that causes blood-forming stem cells to develop into MDS blood cell disease and become cancerous.
We now want to understand why this protein causes the MDS cells to develop from normal blood-forming cells, and how the MDS cells develop into leukaemia.
We are also performing a very large drug screen on the MDS cells to identify better treatments for MDS.
What is the need?
Apart from a blood-forming stem cell transplant (for which most patients are too old), there are no cures for the MDS blood cell diseases. Current treatments (such as blood transfusions) help to reduce symptoms, but are not a cure. There is therefore a significant need for better treatments for these blood cell diseases.
We also need to understand how MDS blood cell diseases develop from normal blood-forming stem cells, and how the MDS cells then becomes cancerous. This will potentially allow us to find ways of preventing MDS blood cell diseases and leukaemia from occuring in the future. Our work will improve the chance of a cure for the 2,300 Australians diagnosed with MDS blood cell diseases each year.
What impact will this research have?
Patients with MDS have few treatment options to help control their symptoms and there are no treatments that prevent them from developing leukaemia. A large number of MDS patients do not respond to the treatment options available and require multiple blood transfusions to stay alive. Our research aims to find better treatments that will hopefully lead to a cure.
We will also understand more about how cancer develops. Moreover, as the protein we have identified in MDS blood cell diseases is also present in other cancers (e.g. breast, prostate, lung and bowel), we may understand more about how these cancers develop and can be treated as well.
Analysis of patient samples to obtain more information about MDS.
Perform the large drug screen to identify better treatments for MDS. Start to validate the potential new therapies identified in this drug screen, and prioritise 70 therapies to trial further.
| Complete studies validating the potential new therapies identified in the drug screen.
"Our research will improve the chance of a cure for the 2,300 Australians diagnosed with MDS blood cell diseases each year."