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Clinical trials to test new way to prevent transplant complications

Scientists at QIMR Berghofer Medical Research Institute have identified a way to prevent bone marrow transplant patients from suffering serious complications.

In world-first research, the team, led by QIMR Berghofer’s Dr Kelli MacDonald, has worked out the cellular process which causes chronic Graft-Versus-Host Disease (GVHD), a life-threatening condition which can affect up to 80% of all bone marrow transplant patients.

The team is now working towards starting clinical trials next year, to test an antibody which could block this process.

“This is an important and exciting step forward in developing new treatments for transplant patients,” Dr MacDonald said.

“Bone marrow – or stem cell – transplants are a standard and successful treatment for about 1000 Australian leukaemia patients every year.

“But while the transplant can provide a cure for leukaemia, the transplant-related complication of chronic GVHD is devastating and often life threatening,” Dr MacDonald said.

Chronic GVHD occurs when the donor graft recognises the patient’s tissue as foreign, and starts to reject it. Over time the tissue becomes severely damaged and the scarring, or fibrosis, can affect a person’s skin, lungs, and mouth.

Chronic GVHD is fatal in 20% of patients and there are currently very limited treatment options.

“Now that we’ve identified the white blood cells, or macrophages, that are driving the process, we’re keen to test an antibody we think will block the process,” Dr MacDonald said.

“This is a big step towards improving the health outcomes for hundreds of cancer patients in Australia alone, each year,” Dr MacDonald said.

The findings were published online today in the prestigious The Journal of Clinical Investigation and can be viewed at

This research was funded by the Rio Tinto Ride to Conquer Cancer, the NHMRC and Cancer Council Queensland.