New drug fights leukaemia in pre-clinical trials

Scientists at QIMR Berghofer Medical Research Institute have found a new treatment approach that could offer hope to patients with the aggressive blood cancer acute myeloid leukaemia (AML).

Dr Steven Lane and cancer biologist Claudia Bruedigam, from QIMR Berghofer’s Translational Leukaemia Research Laboratory, said the drug was found to be highly effective against human leukaemia cells in pre-clinical trials.

“We tested the drug imetelstat against human leukaemia models and found that it killed or impaired progression of the disease,” Dr Lane said.

“It does this by inhibiting a protein needed for the formation of the leukaemia stem cells, which otherwise have enormous self-renewal capacity.

“The study found that by turning off a gene called telomerase, the cancer cells become unstable and eventually the cells die.”

The QIMR Berghofer team also found that imetelstat delayed or prevented relapse of AML following chemotherapy.

“Unfortunately, relapse is a common and devastating setback for many AML patients and this treatment could effectively prolong remission,” Dr Lane said.

“It has potential for use in addition to chemotherapy, or as an alternative treatment particularly in elderly patients.

“Early phase clinical trials of imetelstat are already underway for a number of blood cancers, including myelofibrosis so it should be straight forward to start clinical trials in AML,” Dr Lane said.

Dr Lane said he will be talking with the company developing the drug in the United States this week about their plans to start a clinical trial for AML patients here in Brisbane, hopefully within the next 12 to 18 months.

The study has been published in Cell Stem Cell: http://www.cell.com/cell-stem-cell/abstract/S1934-5909(14)00522-0

The project has been funded by the Leukaemia Foundation of Queensland, Cancer Australia and Cure Cancer Australia.

Dr Lane is a recipient of the Rhys Pengelly Fellowship from In Vitro Technologies and a career development fellowship from the NHMRC.