Victoria backing Australia's first stem cell trial for rare genetic disorder

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The Murdoch Children’s Research Institute (MCRI) is rolling out an Australian-first stem cell therapy trial for an extremely rare genetic condition.

Victoria's minister for medical research, Ben Carroll, visited the MCRI at the Royal Children’s Hospital (RCH) in Melbourne to announce the start of the clinical trial for young Australians with RAG-1 deficient Severe Combined Immunodeficiency (RAG-1 SCID).

It is an expansion of a trial underway at Leiden University Medical Centre in the Netherlands. It will be supported by the Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW consortium) in partnership with Melbourne Children’s Trials Centre.

The Victorian government is providing MCRI with a $1 million investment to explore the expansion of its stem cell research and regenerative medicine capabilities.

RAG-1 SCID leaves affected children extremely vulnerable to common illnesses. Most children born with the rare genetic condition will die from infection within their first years of life.

In the trial, 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.

While rare, several babies are diagnosed with RAG 1 SCID in Australia each year.

“This groundbreaking trial will give young Aussies born with RAG-1 SCID the chance to live a happy, healthy and long life," said Minister Carroll. “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.”