Kiwi scientists developing 'groundbreaking' drug

By Abby Gillies

Photo / Thinkstock
Photo / Thinkstock

Kiwi scientists are part of a team developing a groundbreaking drug to stop the body from rejecting bone marrow transplants in cancer patients.

Auckland University's Professor Bill Denny and Dr Julie Spicer are part of a group working on the research led by the Peter MacCallum Cancer Centre in Melbourne.

After more than a decade of research, the team has discovered the function of a protein called perforin, a toxin discharged by cells.

The protein works to rid the body of cancerous cells, but can have a negative effect for bone marrow transplant patients by causing the body to reject the treatment.

'If we don't make enough perforin, the body can't effectively fight off many viruses. But perforin can also mark the wrong cells for elimination, such as when the immune system attempts to rid the body of donated organ tissue or bone marrow following a transplant," said Peter McCallum executive director, and project leader Professor Joe Trapani.

Professor Denny and Dr Spicer have identified a number of synthetic 'drug-like' compounds that block perforin's function in the test tube - taking the team a step closer to prolonging the survival of bone marrow stem cells.

The drug has been successfully trialled on mice.

If it works in humans it could have a significant effect on the recovery rate of transplant patients.

"Essentially this will mean that patients with blood cancers will be able to recover their infection-fighting white blood cells and platelets, critical for normal blood clotting, more rapidly after transplantation," said Queensland Institute of Medical Research Professor Geoff Hill.

The research team are now working on developing a safe drug for humans to protect transplanted bone marrow stem cells from the protein.

'If we can translate these results into humans, a greater number of cancer patients will be able to undergo stem cell transplantation, as the perforin inhibitor will enable them to be matched with more varied donors," said Professor Hill.

Human trials, which should start in 2016, have been boosted by a $6.8 million grant from the UK's Wellcome Trust.


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