Improving effectiveness of chemotherapy for treating leukaemia

This research aims to overcome two major problems associated with chemotherapy for leukaemia.  Stem cells within the leukaemia often survive chemotherapy and seed relapses of the disease; and chemotherapy kills many normal stem cells resulting in potentially life-threatening loss of production of new blood cells. The work of Associate Professor Ingrid Winkler and Professor Jean-Pierre Levesque shows that by inhibiting E-selectin, the leukaemic stem cells can be sensitised to chemotherapy while the normal stem cells are protected.

Head Researcher A/Prof Ingrid Winkler and Prof Jean-Pierre Levesque
Team Members Stem Cells and Cancer Group, Mater Research Stem Cell Biology Group, Mater Research
Body Part Blood, Bone
Process Used E-selectin, the leukaemic stem cells
Research Areas Cancer, Stem Cell Biology
Disease List any diseases this research considers
Commercial Partnerships GlycoMimetics Inc., US biotechnology company
Institutions Mater Medical Research Institute – The University of Queensland (MMRI-UQ); Princess Alexandra Hospital

About the Project

Every year more than 2,800 Australians are diagnosed with leukaemia and over 1,300 people die from this type of cancer.  Each year over 100,000 Australians undergo therapy for cancer and over one third suffer potentially life-threatening cancer therapy-related complications due to collateral damage to healthy stem cells.

This research, highlighted by a 2013 Nature Medicine article, explores the function of the stem cells in the bone marrow that renew blood cells over the life of an individual. It identified a molecule, E-selectin, found on blood vessels that regulates stem cell function. 

A/Professor Ingrid Winkler’s and Professor Jean-Pierre Levesque’s work shows that by inhibiting E-selectin, the leukaemic stem cells can be sensitised to chemotherapy while the normal stem cells are protected. 

This new treatment offers the potential to not only alleviate the side effects of chemotherapy in patients with a difficult to treat leukaemia, but to greatly improve the effectiveness of chemotherapy, potentially saving many lives.

There are opportunities to use the E-selectin inhibitors to make chemotherapy safer in a broad range of cancers treated by chemotherapy. This offers the prospect of alleviating the side effects of chemotherapy and/or allowing oncologists to use more effective doses of these drugs, potentially saving lives, reducing the morbidity of cancer therapy and substantially reducing health care costs.

The intellectual property generated by these researchers has been licensed by US biotechnology company, GlycoMimetics Inc., which is an active research partner.

Translational Research - Milestone T2

First patents were filed in 2008.  Between 2009 and 2016, collaborative and contract research was undertaken with the industry partner, GlycoMimetics Inc. 

In March 2016, GlycoMimetics announced an overall response rate of 62% in its clinical trial of the E-selectin inhibitor drug, GMI-1271, in combination with chemotherapy in patients with acute myeloid leukaemia (AML). This response rate is well above what would be expected for chemotherapy alone. 

The trial is now proceeding, including at the Princess Alexandra Hospital in Brisbane, with patients being monitored by A/Prof Winkler’s team within the Translational Research Institute. 

> For inquiries about research, potential research collaborations or postgraduate student opportunities, contact A/Prof Ingrid Winkler email: [email protected]  or Prof Jean-Pierre Levesque email: [email protected]

To inquire about how you could help support and advance this research, contact Mater Foundation at