Australian researchers and oncologists have been awarded $2.4 million to investigate the causes and treatments of neuroblastoma, the deadliest and most common solid tumor in children under the age of five.
Associate Professor Yeesim Khew-Goodall and Associate Professor Quenten Schwarz of the University of South Australia and the SA Pathology’s Center for Cancer Biology will lead two separate projects to identify the molecular drivers of neuroblastoma and find more effective drugs to fight it, using of patient data in the first instance, and genetically engineered stem cells in the second.
The projects, involving Women’s and Children’s Hospital and the Royal Adelaide Hospital, are two of 106 groundbreaking medical research projects announced last week by the federal government under the Medical Research Future Fund, including $5.7 million. for UniSA.
Neuroblastoma is a devastating disease that accounts for 15 percent of all childhood cancer deaths, and less than 50 percent of high-risk patients live five years after diagnosis.
“It usually affects very young children, usually under the age of five, with an average age of about one to two years,” said Assoc Prof Yeesim Khew-Goodall, a world expert in cancer and microRNA biology.
“For high-risk neuroblastoma, relapse is not uncommon, and these children often have to undergo multiple rounds of therapy. Due to the young age of the children and the high toxicity of current treatments, including chemotherapy and radiation therapy, those who survive may end up with debilitating side effects that stay with them for a lifetime.”
Tumors form (usually in the abdominal area) when immature nerve cells called neuroblasts continue to divide and grow, developing into cancer cells instead of becoming functioning, mature nerve cells. Faulty genes are thought to be partly responsible, but scientists have yet to find the definitive causes.
“Despite the highly toxic nature of current therapies, they are only effective in some children, so our priority will be to predict which patients will or will not respond to current treatments. Currently, there is a lack of reliable diagnostic criteria to predict disease course or treatment outcomes, and our goal is to fill that void.”
The $1.4 million project by Assoc Prof. Khew-Goodall aims to improve risk classification using clinical information linked to molecular profiles of patient samples, and to identify therapeutic drugs that can be personalized for each child.
“We have found the first evidence that important microRNAs (molecules that regulate gene expression) are deleted in one type of neuroblastoma, for example. Increasing the expression of these microRNAs could be significant in halting cancer progression.”
“Right now, we have a sledgehammer approach to treating neuroblastoma that can lead to developmental effects, including deafness, and problems with speech, mobility and cognition.”
Assoc Prof Schwarz, a world expert in neuron development, will use genetically engineered stem cells to model the fetal origin of the disease and screen FDA-approved drugs.
“Stem cell modeling will help us mimic the disease process so that we can understand how different genetic changes cause different forms of this cancer. We hope that this new information will allow us to identify the best therapies for each tumor type, as well as more accurately identify the predict patient outcomes,” says Prof. Schwarz of Assoc.
“A major shortcoming of current treatment strategies is that they fail to treat the underlying cause of tumor growth. By modeling the disease, we will have better resources to identify new drugs for this condition that are already approved for clinical use in other disease situations.” he says.
The researchers will work with the families of current patients over the next three years, combining laboratory studies with patient profiling.
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