Trial for children with severe immune deficiency disorder

Murdoch Children’s Research Institute is leading a gene therapy trial for children with severe immune deficiency disorder that makes them extremely vulnerable to infections.

The trial for children diagnosed with RAG-1 deficient Severe Combined Immunodeficiency (RAG-1 SCID) who are born without infection-fighting immune cells is offered to patients in Europe through the Leiden University Medical Center (LUMC) in the Netherlands.

The trial is led by Professor Frank Staal, principal investigator at the Novo Nordisk Foundation Center for Stem Cell Medicine, and Professor Arjan Lankester from LUMC’s Willem-Alexander Children’s Hospital.

Staal has recently partnered with Associate Professor Rachel Conyers, a paediatric oncologist and co-lead of the Bone Marrow Transplantation Group from Murdoch Children’s and The Royal Children’s Hospital, to broaden a clinical trial site to Australia. Conyers is also an Associate Investigator with the Novo Nordisk Foundation Center for Stem Cell Medicine.

The Murdoch Children’s trial will see stem cells taken from the child’s own bone marrow and provided with a healthy copy of the RAG-1 gene in a specialised laboratory. The genetically modified stem cells are then injected into the child’s bloodstream where they become healthy white blood cells that will build a new, functional immune system.

Under the trial, the stem cells will be transferred to LUMC and treated in a specialised laboratory before being infused back into patients in Australia.

“Given the patient is their own donor, we avoid the major problems that can come with stem cell transplants,” Conyers said.

“With the addition of the Melbourne site, we will reach two important milestones. We make this life-saving therapy available for the first time to Australian babies, but we will also tick over the 10 patient mark of those who have completed our trial to show its efficacy,” Staal said.

The Australian arm of the clinical trial will be led by Murdoch Children’s and run at The Royal Children’s Hospital, supported by Novo Nordisk Foundation Center for Stem Cell Medicine. It will run in partnership with the Melbourne Children’s Trials Centre (MCTC) and is aligned with the Advanced Therapies Initiative.

The trial has been made possible through an investment of DKK6.2 million from the Novo Nordisk Foundation Center for Stem Cell Medicine and in partnership with the team at LUMC.

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