Walter and Eliza Hall Institute scientists ‘cure’ childhood leukaemia in animal tests

MELBOURNE researchers have found a way to “cure” in animals the most common form of childhood leukaemia.

Scientist Ian Majewski in the Hilton lab at the Walter and Eliza Hall Institute.

MELBOURNE researchers have found a way to “cure” in animals the most common form of childhood leukaemia.

Walter and Eliza Hall Institute scientists are the first to switch the Pax5 gene off and on in animal test subjects, clearing the leukaemia.

PhD student Grace Liu said the finding could lead to the development of faster-acting drugs with fewer side effects.

Pax5 is mutated in about a third of children with B-progenitor acute lymphoblastic leukaemia. Essential for white blood cell development, the gene causes cells to become cancerous when mutated.

It had been shown that switching it off could cause leukaemia, but this was the first time researchers had been able to toggle it on and off in an animal, “effectively curing” it.

“Simply reactivating Pax5 causes tumour cells to resume normal development and lose their cancerous properties,” Ms Liu said.

She used genetic switch technology, designed by supervisor Dr Ross Dickins, to stop and reactive the gene in mice.

“We found that just restoring Pax5 was able to allow the leukaemia cells to become ‘normal’ again,” Ms Liu said.

Ms Liu said it was an exciting first step towards developing a drug with the same effect.

The research, published in Genes & Development, was backed by the National Health and Medical Research Council, the Leukaemia Foundation, the Sylvia and Charles Viertel Charitable Foundation, the Victorian Endowment for Science, Knowledge and Innovation, and the State of Victoria.

And NSW researchers have made progress in finding ways to treat cancer that could spare patients debilitating chemotherapy, and help children with a rare ageing syndrome.

A new centre focuses on telomeres, which protect chromosome ends from cell division damage, and the replenishing enzyme telomerase.