- Published:
- Sunday 15 October 2023
Victoria’s reputation as a global leader in medical research continues, with the Murdoch Children’s Research Institute (MCRI) rolling out an Australian-first stem cell therapy trial for an extremely rare genetic condition.
Minister for Medical Research Ben Carroll today visited the MCRI at the Royal Children’s Hospital (RCH) in Parkville to announce the start of a groundbreaking clinical trial for young Australians with RAG-1 deficient Severe Combined Immunodeficiency (RAG-1 SCID).
Only the second worldwide, it is an expansion of a trial underway at Leiden University Medical Centre in the Netherlands and will be supported by Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW consortium), in partnership with Melbourne Children’s Trials Centre.
The Allan Labor Government is backing MCRI with a $1 million investment to explore the expansion of its stem cell research and regenerative medicine capabilities – supporting its key role in the new reNEW consortium.
Born without infection-fighting immune cells caused by a genetic defect, RAG-1 SCID leaves affected children extremely vulnerable to common illnesses – most children born with the rare genetic condition will tragically die from infection within their first years of life.
The trial will see participants have stem cells taken from their own bone marrow to be genetically modified with a healthy copy of the RAG-1 gene, before being injected into the child’s bloodstream where it will become healthy white blood cells that will build a functional immune system.
While rare, every year several babies are diagnosed with RAG 1 SCID in Australia and usually fatal during their first two years of life. The trial could enable these children to live a long, happy and healthy life.
The Labor Government is also proud to support cutting-edge medical research and in paediatric care, including the Victorian Newborn Bloodspot Screening program to screen for 27 conditions in newborn babies, while a pilot for SCID and Spinal Muscular Atrophy is also underway.
The Victorian Budget 2023/24 included $9 million towards genetic testing which will help deliver children targeted treatment options, increase treatment access, and improve the outcomes of patients with rare diseases and cancer who are receiving highly specialised therapies.
MCRI is one Victoria’s 12 independent medical research institutes, the largest child health research institute in Australia and is ranked the third highest in the world.
Quotes attributable to Minister for Medical Research Ben Carroll
“This groundbreaking trial will give young Aussies born with RAG-1 SCID the chance to live a happy, healthy and long life.”
“Victoria is renowned as a global centre for medical research and the Murdoch Children’s Research Institute is just one example of the incredible work being done that is having a positive impact on people’s lives.”