Immunotherapy trial to protect children

It’s a devastating scenario that no parent should go through. Your beloved child, full of life and the apple of your eye, is suddenly struck down by life-threatening blood cancer or diagnosed with an inherited immune deficiency and requires a life-saving stem cell transplant.

Tragically, this is the reality for hundreds of parents around Australia each year. Children who receive life-saving bone marrow transplants can often fall victim to secondary infectious complications as their bodies try to fight off their blood cancer or disorder and build a new immune system with donor stem cells.

Professor Rajiv Khanna AO and his team at the Centre for Immunotherapy and Vaccine Development at QIMR Berghofer are determined to develop new and better treatment options for children who develop these life-threatening viral infections after a bone marrow transplant.

His research team is running a clinical trial, jointly sponsored by QIMR Berghofer and the Children’s Hospital Foundation, to test a new cellular immunotherapy treatment aimed at preventing dangerous side effects for sick children who have to undergo stem cell transplants.

The trial will involve up to 20 children aged between 3 months and 18 years, who need stem cell transplants from a family member whose bone marrow is not an exact match, to treat blood cancers or genetic immune deficiencies.

The immunotherapies are being manufactured at QIMR Berghofer in Brisbane and will be administered at participating clinical trial sites at the Queensland Children’s Hospital, Sydney Children’s Hospital and the Royal Children’s Hospital in Melbourne.

The cellular immunotherapy is made by turbo-charging immune cells taken from the same person who donates their stem cells for the child’s transplant.

‘Before patients receive a stem cell transplant, certain immune cells – like killer T cells and B cells – are removed from the donor’s cells. This lowers the risk of potentially fatal complications like graft-versus-host disease, where the donor’s immune cells attack the recipient’s tissues,’ Professor Khanna explained.

‘But this necessary step of removing immune cells from the stem cells leaves transplant patients at high risk of developing viral infections.

‘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.’

In the first stage of the clinical trial, the donor’s blood is collected ahead of the stem-cell transplant and turbo-charged at QIMR Berghofer’s cell therapy manufacturing facility Q-Gen Cell Therapeutics.

Within a few weeks, the child receives their stem cell transplant as part of their standard treatment. Once the transplant has engrafted, the patient will start receiving fortnightly T cell immunotherapy infusions aimed at boosting their immune system before they show any signs of viral complications.

Paediatric Oncologist and Director of the Blood and Marrow Transplant Program at Queensland Children’s Hospital, Dr Chris Fraser, said the timing of the immunotherapy was important because viral complications could occur as early as a month post-transplant.

‘The immunotherapy will continue for approximately 2 months, after which we will follow these patients for eight months or more to confirm the therapy is safe,’ Dr Fraser said.

‘Stem cell transplants for blood cancers or inherited immune deficiencies are high-risk procedures that are required to cure otherwise life-threatening diseases. Our aim is to see if this new therapy can be safely used in these vulnerable children, and in the future we hope that T cell immunotherapy may be used to reduce the risk of potentially fatal viral infections after transplantation.’

Children’s Hospital Foundation Chief Executive Officer Rosie Simpson said the organisation was proud to help fund the clinical trial as the Foundation increases its support of innovative research into areas such as immunotherapy to ensure sick Queensland children receive the very best possible care and treatment.

‘The Children’s Hospital Foundation is focused on helping enable world-class research and clinical trials that advance treatment options for sick kids and improve their chances of survival,’ Ms Simpson said.

‘This trial provides important hope of better outcomes for the families going through the stem cell transplant process, and also adds to the growing wealth of knowledge on how best to treat viral complications.’

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