Funding secured for stem cell research to accelerate new treatments

Four Murdoch Children's Research Institute (MCRI) researchers have received funding for stem cell research that will accelerate new treatments for a range of serious childhood conditions.

Professor Melissa Little, Associate Professor Enzo Porrello,  Dr Nicole Van Bergen and Associate Professor David Elliott have secured grants to advance their research into kidney disease, heart failure and a debilitating seizure disorder. Federal Health Minister Greg Hunt, announced the funding at MCRI as part of the 2021 MRFF Stem Cell Therapy Mission round.

Professor Little's project will harness human stem cells to create mini kidneys that will help find treatments for genetic kidney disease, a life-threatening condition that is largely untreatable.

"While a genomic diagnosis can now be made in almost 50 per cent of patients with genetic kidney disease, this has not led to any specific treatment options," she said. "Our study will focus on congenital nephrotic syndrome, a type of potentially fatal inherited kidney disease."

Professor Little said her team would use their well-established patient stem cell-derived "mini kidneys" to model human kidney disease in a dish, accelerating the development pathway to clinical trials and eventually personalised therapies.

As pioneers who developed bioengineered heart tissue from pluripotent stem cells, Associate Professor Porrello and his team will now test whether this technology can treat heart disease, the leading cause of Australian infant deaths.

"This project aims to evaluate the safety and effectiveness of bioengineered heart tissue in a pre-clinical model of paediatric heart failure," he said. "If successful, this approach could radically transform patient outcomes and improve the quality of life of children affected by heart disease."

Associate Professor Porrello said that these critical proof-of-concept studies would determine if bioengineered heart tissue could be used for congenital heart repair and pave the way for further commercial development of this technology and future clinical trials.

Dr Van Bergen's project will use stem-cell-derived human brain cells grown in a 3D environment to find treatments for a neurological disorder, which causes severe seizures and developmental delays in young children.

"CDKL5 Deficiency Disorder (CDD) is a debilitating disorder with, sadly, no effective treatment options," she said. "To address this, we will use bioengineered brain cells grown in a 3D-matrix to closely mimic the brain's cellular structure in order to study molecular pathways involved in CDD, coupled with a high-throughput drug screening program to identify potential targeted therapies for children with CDD."

Dr Elliott's project aims to prevent heart failure that can occur in childhood cancer survivors as a result of chemotherapy, by using miniature human hearts to identify heart-protecting drugs.

"Using heart muscle cells produced in the lab, we are looking for drugs that stop the damage that often occurs during chemotherapy and persists into adulthood," he said. "Our aim is to significantly reduce the damaging side effects of anti-cancer drugs on the hearts of children."

Associate Professor Elliott, in collaboration with Professor Jonathan Baell from the Monash Institute of Pharmaceutical Sciences, is using approved drugs, which will enable his team to quickly move toward clinical trials.