Bone marrow transplant drug trial closer

Posted: June 6, 2012 at 9:15 am

A drug that stops the body from rejecting bone marrow transplants in cancer patients could be ready for human trials in three years time.

The latest development comes after more than a decade of research unlocking the function of a protein called perforin, which kills rogue cells in the body.

Australian researchers involved in unravelling perforin's molecular structure, a discovery published in the journal Nature in 2010, are now working towards developing a safe drug to block the protein.

Perforin plays a key role in the body's immune response by punching holes in, and killing, cells which have been hijacked by viruses or cancer to rid the body of disease.

However, the protein is problematic for bone marrow transplant patients because it can cause the body to reject the treatment.

For this reason, a project led by the Peter MacCallum Cancer Centre in Melbourne is developing a drug to inhibit the protein in bone marrow stem cell transplant patients to help their recovery.

The drug works in mouse models, but a $6.8 million grant from the UK's Wellcome Trust will allow the drug to be fine-tuned for human trials.

'In the mouse models we use, we know the inhibitors are effective,' project leader Professor Joe Trapani, executive director of cancer research at Peter Mac, told AAP.

'They actually help stem cells survive when they would otherwise be rejected.'

The Peter Mac team is working with New Zealand chemist Prof Bill Denny to refine the drug, along with Monash University and Queensland Institute of Medical Research scientists.

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Bone marrow transplant drug trial closer

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