New hope for bone marrow transplant patients

Clinical trials led by QIMR Berghofer Medical Research Institute and the Royal Brisbane and Women’s Hospital are set to change international practice for the treatment of leukaemia patients undergoing bone marrow transplants.

QIMR Berghofer Senior Scientist, Professor Geoff Hill, said the Phase I/II trials resulted in a significant drop in cases of acute graft versus host disease (GVHD), a potentially fatal complication from stem cell transplants.

“The incidence of acute GVHD was reduced from the usual 50%, to 12% of transplant patients in the trial,” Professor Hill said.

“Severe cases – which often result in death – were reduced from 21% to 4%.”

The trials were conducted at the RBWH Transplant Unit.

Professor Hill said irradiation followed by bone marrow transplant was now the standard treatment for blood cancers such as leukaemia, and was generally successful.

“The treatment does result in a ramped up response in the patient’s immune system, and unfortunately in acute GVHD this is directed towards normal tissues in the skin, gut and liver,” Professor Hill said.

“To try to prevent this from happening, we administered the drug Tocilizumab (TCZ) to inhibit the immune system’s production of the protein IL-6, which stimulates the body’s response to trauma.

“We found the addition of TCZ to the usual regime of post-transplant medication resulted in a large drop in the incidence of acute GVHD for those participating in the study.”

RBWH Bone Marrow Transplant Unit Acting Director, Associate Professor Glen Kennedy, said the results of the Phase I/II trial represented a significant advance in bone marrow transplant treatments.

“The new therapy has the potential to make transplant safer, and applicable to a larger group of patients.”

A Phase III study now underway at RBWH will be the final test before the addition of TCZ to the GVHD prevention regime is registered and adopted in clinical practice.

TCZ is currently approved for use treating rheumatoid arthritis.

Queensland Health Minister Lawrence Springborg congratulated the team.

“This is a brilliant example of the high quality of translational research underway here in Queensland,” Mr Springborg said.

“We all stand to gain from advances in medical research, and yet again QIMR Berghofer is producing results that add to the world’s knowledge and change lives.”

The results of the Phase I/II clinical trials have been published in the prestigious medical journal, the Lancet Oncology: http://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(14)71017-4/abstract

Professor Hill is a Queensland Health Senior Clinical Research Fellow and a National Health and Medical Research Council (NHMRC) Australia Fellow.