04 April 2019

A new breakthrough treatment combination could offer improved treatment for patients with acute myeloid leukaemia (AML) after a successful phase Ib/II trial in Australia.

The study, led by Associate Professor Andrew Wei, from the Alfred Hospital and Monash University Clinical School, found that combining venetoclax with low-dose cytarabine (LDAC) resulted in the bone marrow clearing in almost 60% of AML patients.

Early results from this and another similar trial were so impressive that that the US Food and Drug Administration approved the use of the combination for the treatment of AML last November.

The study, which is published in the Journal of Clinical Oncology, used the combination of venetoclax and LDAC to treat older adults with AML. Taken separately, these drugs are not successful, with only a 19% response rate to venetoclax, the researchers say.

However, combining the two led to a 54% response rate, with a median overall survival of 10.1 months.

Seventy-four-year-old Kaye Oliver, who was the first patient in the world to enrol on the trial, had a poor prognosis and was told she would only have months to live. She took part in the trial and has survived for more than four years and there is no longer any evidence of cancer.

The trial tested 82 patients with a median age of 74 years and in Australia, Europe and the USA. Venetoclax switches off the BCL-2 protein and activates a self-destruct program in the cell.

Professor Wei said the findings are important not just because of the success of the treatment in a disease that was fatal - but because AML is likely to become more prevalent in the future because of an ageing population.

The current research is supported by another trial in older AML patients, which combined venetoclax with azacytidine and led to a 71% remission rate, with an average life expectancy of almost 17 months.




Wei, A.H., Strickland, S.A. Jr., Hou, J.Z., Fiedler, W., Lin, T.L., Walter, R.B., Enjeti, A., Tiong, I.S., Savona, M., Lee, S., Chyla, B., Popovic, R., Salem, A.H., Agarwal, S., Xu, T., Fakouhi, K.M., Humerickhouse, R., Hong, W.J., Hayslip, J., Roboz, G.J. (2019) “Venetoclax Combined With Low-Dose Cytarabine for Previously Untreated Patients With Acute Myeloid Leukemia: Results From a Phase Ib/II Study”, Journal of Clinical Oncology, available at doi: 10.1200/JCO.18.01600