A Gene Therapy Approach to Acute Myeloid Leukaemia: Reversion of the Disease Phenotype [ 2000 - 2004 ]

Research Grant

Researchers: Prof Geoffrey Symonds (Principal investigator)

Brief description Acute myeloid leukaemia is a cancer of white blood cells in which there is uncontrolled cell growth filling up the blood with cells that do not function properly. The cells also invade various tissues causing further damage. The patient becomes increasingly compromised due to the lack of appropriate normal cell types. The present standard therapy involves radiation or chemicals which are toxic to the leukaemic cells. However, these treatments are also toxic to normal cells so very high doses that might be more effective to kill the leukaemic cells can not be given. At the doses which can be administered, the leukaemia often (in greater than 50% of patients) becomes resistant. The present project seeks to use a novel treatment strategy in which genes are used to modify the genetic abnormalities present in these leukaemic cells thereby stopping their growth. This growth suppression will be specific to the leukaemic cells as it targets their abnormal genetics leaving normal cells alone. The gene therapeutics have already been identified in part and the aims of this grant are: 1. To show that the gene therapeutic approach is justified to selectively stop the leukaemic cells from growing. 2. To confirm novel means to identify the inhibitory genes. 3. To determine the best inhibitory gene(s). 4. To show how these inhibitory genes can be delivered to the patients' cells and to model their effect in animal models. This project would represent a paradigm shift in the treatment of leukaemia and also has implications for the treatment of other cancer types.

Funding Amount $AUD 744,911.24

Funding Scheme NHMRC Project Grants

Notes Standard Project with Research Fellowship