Researchers from the Novo Nordisk Foundation Center for Stem Cell Medicine, reNEW expand their on-going gene therapy trial on children suffering from RAG-1 deficient Severe Combined Immunodeficiency (RAG-1 SCID) from the Netherlands to Australia. The therapy will provide these children with a working copy of the RAG-1 gene, meaning they will likely be able to build a healthy immune system and live a normal life.” The trial is possible thanks to funding of DKK 6.2 million from reNEW.
Children with RAG-1 SCID are extremely vulnerable to infections as they are born without infection-fighting immune cells. Without treatment, most children with this immune disorder, caused by genetic defects, die from infection during their first or second year.
A stem cell transplant from a healthy donor can cure RAG-1 SCID, but most patients lack a suitable donor. Gene therapy is now revolutionising treatment by offering a life-saving alternative while avoiding and reducing lifelong complications that can arise from stem cell transplants.
In the up-coming trial at reNEW’s Australian research partner – the Murdoch Children’s Research Institute based in Melbourne, stem cells will be taken from the child’s bone marrow and provided with a healthy copy of their RAG-1 gene in a specialized laboratory in LUMC.
These modified stem cells will then be injected into the child’s bloodstream where they will become healthy white blood cells that will build a new, functional immune system. This new therapy will transform the lives of children with RAG-1 SCID enabling them to fight off common infections and live a normal life. .
Professor Frank Staal, principal investigator at reNEW’s Dutch research partner the Leiden University Medical Center (LUMC) and Professor Arjan Lankester from LUMC’s Willem-Alexander Children’s Hospital are behind this trial. They already successfully treated the first child with this new gene therapy. Staal recently partnered with Associate Professor Rachel Conyers, who is a pediatric oncologist and co-lead of the Bone Marrow Transplantation Group at Murdoch Children’s, as well as an associate investigator at reNEW, to expand the trial to Melbourne.
“With the addition of the Melbourne site, we reach two important milestones: we make this life saving therapy available for the first time to Australian babies, but we also help reach the number of included patients to complete our trial to show its efficacy,” said Staal.
We are excited to bring the RAG-1 SCID trial to the Murdoch Children’s,” the CEO of reNEW, Melissa H. Little added. “It demonstrates the strength of the Novo Nordisk Foundation Center for Stem Cell Medicine (reNEW) international collaboration and provides an ongoing pathway for clinical translation in Australia of gene and cellular therapies.”