Scientists Pursuing Elimination of Deadly Childhood Cancer

In a significant development for childhood cancer research, two projects led by Associate Professors Yeesim Khew-Goodall and Quenten Schwarz have been awarded funding by the Federal Government. These projects, part of 106 groundbreaking medical research projects announced, aim to uncover the causes and develop more effective treatments for neuroblastoma, the deadliest and most common solid tumour in children under the age of five.

Neuroblastoma forms due to immature nerve cells, known as neuroblasts, continuing to divide and grow, developing into cancer cells instead of becoming functioning, mature nerve cells. This cancer accounts for 15% of all childhood cancer deaths, and fewer than 50% of high-risk patients live five years after diagnosis. High-risk neuroblastoma patients often require multiple rounds of therapy, which can lead to debilitating side effects that stay with them for life.

Associate Professor Khew-Goodall's project focuses on identifying therapeutic drugs that can be personalised for each child with neuroblastoma. The goal is to improve risk classification using clinical information linked to molecular profiles of patient samples. Key microRNAs are deleted in one type of neuroblastoma, and increasing their expression could potentially stop the progression of the cancer.

On the other hand, Associate Professor Schwarz will use genetically engineered stem cells to model the fetal origins of neuroblastoma. The aim is to understand how different genetic alterations drive different forms of neuroblastoma. Stem cell modeling by Schwarz aims to provide insights into the best therapies for each tumour type and more accurate prediction of patient outcomes.

Both projects will continue to use patient data and genetically engineered stem cells. The researchers will work with the families of current patients over the next three years, combining laboratory studies with patient profiling. This collaboration will allow for the tracking of research results via laboratory studies and patient profiling as part of the project starting April 1, 2025.

Australian researchers and oncologists have been awarded a total of $2.4 million for these projects. UniSA received $5.7 million under the Medical Research Future Fund. The research projects will also aim to find new drugs for neuroblastoma that are already approved for clinical use in other disease settings.

These groundbreaking projects offer hope for families affected by neuroblastoma, a disease that typically affects children under the age of five, with the average age of diagnosis around one to two years. The researchers hope that their work will lead to improved outcomes for these children and a better understanding of this devastating disease.