A world-first study is underway into a potentially lifesaving therapy for children having stem cell transplants.
A world-first study is underway into a potentially lifesaving therapy for children having stem cell transplants.

Trial therapy could ‘turbocharge’ sick kids’ immune cells

Queensland researchers have begun a world-first trial of a new treatment aimed at preventing life-threatening infections in children having stem cell transplants.

The two-year study of up to 20 children as young as three months old will test whether giving them immunotherapy after their transplant reduces the risk of dangerous viral infections.

Two patients at the Queensland Children's Hospital have already received the treatment manufactured at the QIMR Berghofer Medical Research Institute.

Scientist Rajiv Khanna, who developed the immunotherapy, said the research targeted children with blood cancer or genetic immune deficiencies who had received stem cells from a family member.

QIMR Berghofer Medical Research Institute’s Professor Rajiv Khanna. Photo: Adam Head.
QIMR Berghofer Medical Research Institute’s Professor Rajiv Khanna. Photo: Adam Head.

Professor Khanna said the immunotherapy was made by taking blood from the donors before the stem cell transplants, then collecting and "turbocharging" their immune cells in the laboratory, priming them to identify and attack four "very, very nasty" viruses - cytomegalovirus, Epstein-Barr virus, adenovirus and BK virus.

He said the immunotherapy would be given to the children as an infusion at least 28 days after their stem cell transplant and then at fortnightly intervals for about two months.

Their aim is to reduce the children's risk of developing a potentially lethal complication of stem cell transplants - severe infection.

Professor Khanna said up to 60 per cent of stem cell transplant patients developed complications as a result of viral infections.

Before the children's transplants, key immune cells are removed from the donor stem cells to prevent the risk of graft-versus-host disease, which can also be fatal. This leaves the child patients highly susceptible to infection.

Scientist Dr Rajiv Khanna, from the QIMR Berghofer Medical Research Institute. AAP image, John Gass.
Scientist Dr Rajiv Khanna, from the QIMR Berghofer Medical Research Institute. AAP image, John Gass.

"By taking white blood cells from the same donor and training them in the laboratory to recognise and destroy cells infected with the four most common viruses that affect these patients, we hope to effectively prevent complications arising," Professor Khanna said.

Cancer specialist and director of the Queensland Children's Hospital Blood and Marrow Transplant Program, Chris Fraser, said the aim of the immunotherapy was to protect the children from viral complications while their stem cell transplants worked to kill off their blood cancer or correct their immune deficiency.

"Stem cell transplants for blood cancers or inherited immune deficiencies are high-risk procedures that are required to cure otherwise life-threatening diseases," Dr Fraser said.

"Our aim is to see if this new therapy can be safely used in these vulnerable children and in the future we hope that … immunotherapy may be used to reduce the risk of potentially fatal viral infections after transplantation."

The Children's Hospital at Westmead, Sydney Children's Hospital and the Royal Children's Hospital in Melbourne are expected to join the trial in the next few months.

QIMR Berghofer and the Children's Hospital Foundation are jointly funding the study.

To donate to research: childrens.org.au

Originally published as Trial therapy could 'turbocharge' sick kids' immune cells



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