Blood cancer find raises new treatment hopes for MDS and AML patients

Pharma News: New QIMR Berghofer research has identified how an early genetic change in blood and bone marrow cells paves the way for the development of some blood cancers.

The discovery provides a new target for treatment of the blood cancers myelodysplastic syndrome (MDS) and acute myeloid leukaemia (AML). MDS is often a precursor cancer to AML, a highly aggressive form of leukaemia.

Lead researcher and the head of QIMR Berghofer Medical Research Institute’s Cancer Program, Associate Professor Steven Lane, said “We found that Cdx2 hijacks and corrupts how other genes behave in blood and bone marrow cells. It sows the seeds of vulnerability which then allows the development of other genetic mutations that lead to cancer.

“It’s a step-wise process that closely reflects the progression of human MDS to AML, where genetic mutations occur in blood and bone marrow stem cells and these immature cells become a reservoir for leukaemia.

“It’s possible that targeting the early processes leading to MDS in patients might prevent the acquisition of other mutations that lead to acute leukaemia.”

As part of the study the researchers also tested how Celgene’s existing anti-leukaemia drug azacitidine affected the Cdx2 gene.

Associate Professor Lane, who is also a clinical haematologist at the Royal Brisbane and Women’s Hospital, said Celgene’s azacitidine is currently funded under the Pharmaceutical Benefits Scheme (PBS) for the treatment of MDS.

“We found that reducing the dosage of the medication and giving it over a longer period of time was more effective in killing off Cdx2 cells and leukaemia cells than the standard treatment regimen that provides high dosages of azacitidine at intermittent intervals,” he said.

“There is a new, oral form of azacitidine that might be more suited to the lower dose, extended treatment regimen, but further tests will be necessary to determine its efficacy in patients.”

According to the Leukaemia Foundation, MDS is a common blood cancer in patients over the age of 60. It can affect children but is less common in younger age groups. It can turn into AML, which is a rare type of leukaemia, accounting for 0.8 per cent of diagnosed cancers, and is hard to treat.