A team of Perth scientists are using a relatively new cellular therapy to treat a life-threatening complication that occurs in 50 per cent of bone marrow transplant patients, Graft-Versus-Host Disease (GVHD).
The cellular therapy uses mesenchymal stromal cells (MSC), which are obtained from donor bone marrow and then grown and expanded in the laboratory over a period of several weeks.
GVHD occurs as a result of the complex interaction between donor cells and recipient tissues after a bone marrow transplant is used to treat disorders like leukaemia.
The standard treatment for GVHD is corticosteroid therapy. This steroid-based chemical therapy acts to suppress the immune system, but more and more patients find that it is unable to stop the complications, which can range from a skin rash, abdominal pain and diarrhoea to liver dysfunction.
Once second-line treatments are employed, patients who do not respond have a poor survival rate with only 20 per cent of affected patients surviving beyond 200 days post treatment.
The Perth researchers are hoping that infusing the MSC into patients will help to increase the survival rate after bone marrow transplants.
“One of the great advantages of this cell therapy is that it is not necessary to match donor cells with the recipient so that anybody is a potential donor for any recipient,” said Prof Richard Herrmann, Director Cell and Tissue Therapy W.A., clinical manufacturing facility at Royal Perth Hospital.
“When the cells are infused into the patient,” according to Prof Herrmann, “the signs and symptoms of GVHD are reduced. These MSC are able to tune down the recipient’s immune response. The other remarkable property is they have the ability to differentiate or change into multiple tissue types. That is, they can convert to cells of cartilage, bone, muscle, heart muscle and neural origin and this may assist with healing,” he explained.
Prof Herrmann said that the team at Royal Perth’s CTTWA has been conducting a Phase I trial to evaluate the safety of these cells in GVHD patients and have also monitored the patients’ response to this therapy. He said it is still ‘early days’ but indicated that of the group of nine patients trialled, “all nine have responded to MSC infusion.”
He added that an international Phase III study conducted by Osiris Ltd that compares active cell treatment with placebo treatment has just recently been completed, but the results have not yet been released.
“MSCs are an exciting and emerging cellular therapy with potential for diverse clinical applications”, according to Dr Stewart Hay, CEO of Research Infrastructure Support Services (RISS) Ltd, the Melbourne-based, not-for-profit funding agency that supports Australian cell therapy researchers including Cell Tissue Therapy of WA (CTTWA).
“Prof Herrmann and his team have made significant in-roads in this area, however a major challenge is that MSC research costs approximately $10,000 per patient. The financial support delivered through the Commonwealth Government’s National Collaborative Research Infrastructure Strategy (NCRIS), has significantly reduced these costs and in doing so has made a valuable contribution in fast-tracking CTTWAs’ research program”, Dr Hay added.
According to Dr Marian Sturm, Principal Scientist and Production Manager of CTTWA, the team is currently in collaboration with other groups in WA and interstate looking at using these cells in other conditions involving immune responses and tissue repair. The group is particularly interested in incorporating these cells into different types of scaffolds as a means of delivering the cells to where they are needed for tissue repair.
Dr Sturm has no doubt that MSC therapy could prove beneficial in any number of treatment areas, but prefers to remain less forthcoming until further studies can be completed.
“This treatment has improved the clinical outcome and quality of life for those patients who have responded,” Dr Sturm began. “The benefit to the community lies in the reduced dependence on medical services and in the ability to return to a normal life.”
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