MRI-UQ research leads to promising clinical trial for leukaemia

1 Dec 2016

A novel leukaemia drug is showing encouraging results in a new clinical trial based on discoveries by Mater Research Institute – University of Queensland (MRI-UQ) Associate Professor Ingrid Winkler.

Under a research collaboration with GlycoMimetics Inc., a US-based biotech company, Associate Professor Winkler demonstrated that by inhibiting E-selectin not only are leukaemic stem cells sensitised to chemotherapy, but normal stem cells are also protected.

“To date, most things that have sensitised the cancer cells to chemotherapy have also sensitised normal stem cells, which is why there are so many side effects to chemotherapy,” said Associate Professor Winkler.

As bone marrow suppression is a severe side effect of high-dose chemotherapy, transient blockade of E-selectin could result in less infection and less of a requirement for blood product and hospital support. The full details of the “protective effects” of E-selectin blockade on immune cells were published in Nature Medicine in 2012.

Based in part on these findings GlycoMimetics have initiated a Phase I/II clinical trial in patients with acute myeloid leukaemia (AML) using their selective antagonist of E-selectin, GMI-1271.

The first results from this clinical trial in the US with GMI-1271 were released in June at the European Haematology Association 21st Congress in Copenhagen, Denmark, demonstrating an overall response rate (combined complete remission (CR) and remission with incomplete recovery (CRi)) of 47 per cent.

Associate Professor Winkler said she is delighted by the success of initial results.

“Nine of the nineteen patients showed a better than expected response, with the leukaemia cells disappearing from their blood. Eight of the nine had a complete response, meaning the leukaemia cannot be seen at all at this stage.”

The trial will now extend to Australia with eligibility expanded to include patients with newly diagnosed, previously untreated AML as well as those with relapsed/refractory disease.

Associate Professor Winkler said she is pleased that Australian patients will be among the first to benefit from this new treatment through the trial.

“The fact it’s come to a clinical trial and is getting good results is a fantastic feeling. I previously worked in an oncology unit overseas where I was treating these patients. I decided when I came back to Australia I would go into research and really help these patients.”

“Over 900 Australians were diagnosed with AML last year and 813 passed away. The median survival time is only 1.3 years from diagnosis. Our findings can go straight into helping patients… and these are the patients that really need help.”

Contact: Bernadette O’Connor,, +61 7 3365 5118, 0431 533 209.