Mater research shows positive signs for acute myeloid leukaemia patients

Tuesday 28 April 2020

A drug currently in clinical trials has the potential to improve the survival rate of Australians diagnosed with acute myeloid leukaemia, preclinical research published by Mater Research’s Associate Professor Ingrid Winkler suggests. 

The findings, published today in the highly respected research journal Nature Communications, shows that blocking the interaction between leukaemia cells and their environment in the bone marrow (niche), at the same time as administering chemotherapy, enables the chemotherapy to work much more effectively. 

A/Prof Winkler said her preclinical (laboratory) research showed that the bone marrow protected the malignant cell.

“Once we discovered the molecule involved, we blocked it using a drug called Uproselesan (GMI-1271), and indeed the chemotherapy became much more effective at destroying the leukaemia.

“When Uproleselan was administered together with chemotherapy, it removed the protective environment around the malignant cells so they again become sensitive to the chemotherapy. 

“In our laboratory studies, adding this drug to standard chemotherapy greatly improved the survival rate and also reduced the chemotherapy side-effects,” she said. 

Uproleselan is made by US drug company GlycoMimetics which is currently running clinical trials on this drug to treat acute myeloid leukaemia. 

Mater Research Executive Director and Haematologist Professor Maher Gandhi said that for the 900 patients in Australia diagnosed with acute myeloid leukaemia each year, prognosis was poor, as chemotherapy wasn’t typically effective in killing all the malignant cells. 

“At the moment the five-year survival for older acute myeloid leukaemia patients on standard chemotherapy is only about 25 per cent. Ingrid and her team have been working hard to improve that,” Professor Gandhi said. 

These positive results support clinical trials that are now being carried out on adult patients in the US and Australia, including a trial site at Princess Alexandra Hospital for adults who have relapsed from the disease. 

“I am very proud that Australian patients with this particularly aggressive leukaemia may be among the first to benefit from this research,” A/Prof Winkler said. 

For more information on the clinical trial visit or email Jason Kelly at the Princess Alexandra Hospital at