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National Research Program Grant-in-aid Chief investigator: Dr Mark Guthridge Institute: Australian Centre for Blood Diseases/Monash University Project title: New approaches to targeting the survival of AML cells Disease focus: Acute myeloid leukaemia Funding: $99,736 Funding period: 2011 Project summary Acute myeloid leukaemia (AML) only responds well to treatment in the short term. The majority of patients relapse due to the ability of the progenitor (or ‘mother’) AML cells to survive both chemotherapy and the newer targeted treatments. Dr Mark Guthridge said his research team had preliminary evidence for an “entirely new mechanism by which AML progenitor cells may evade conventional as well as new generation drugs”. “We’re tackling an important and critical issue that underpins the short-term transient responses of AML to current therapies,” Dr Guthridge said. “In more than 85% of our AML samples we found a specific cell survival pathway had been activated,” he said. “We believe the family of enzymes called Phosphatidylinositol 3-kinase, or PI3K, may play a previously unsuspected role in activating this cell survival pathway in leukaemia cells and increasing the likelihood of relapse. “If we can identify the specific enzyme responsible, it may provide new avenues for selectively targeting AML progenitor cells and allow new opportunities for blocking survival programs in leukaemia.” P13K is known to be involved in a range of cancers and the enzyme family is being targeted for drug development by pharmaceutical companies.