Thomas had a rare brain cancer and no good options. Then he joined a clinical trial

The Sydney Morning Herald

By Rachel Clun

October 6 2020

A life-saving program uses genetic testing to find treatments that shrink or stabilise aggressive tumours in children.

Research on the ZERO Childhood Cancer program, published in Nature Medicine on Tuesday, found the testing of the genetic makeup of the tumours led to suggested alternative treatments for the majority of children in the study.

Researcher and co-chair of ZERO’s national clinical trial Associate Professor David Zeigler said the program's success rates was higher than the team had ever dreamt it would be.

Thomas Edney, pictured with his sister Lucy and parents Abigail and John, was part of the ZERO trial.CREDIT:PENNY STEPHENS

Thomas Edney, pictured with his sister Lucy and parents Abigail and John, was part of the ZERO trial.

"We were hoping when we started this, we would at least see tumours slow down in their growth, or stabilised for a period of time," he said.

 That’s because these high-risk cancers form "very aggressive tumours", and the children with these cancers have failed most standard treatments, Associate Professor Zeigler said.

"But in 30 per cent of cases we've actually seen the tumours shrink, and in some cases completely disappear; in another 40 per cent of cases, we've seen the tumours stabilise and stop growing for a prolonged period of time.

"That's quite an incredible result to see."

About 1000 children and young adults are diagnosed with cancer each year.

ZERO Childhood Cancer researcher Associate Professor Paul Ekert said the majority of those children will be cured of their cancer, but others - including those with neuroblastoma - have few good treatment options.

"This is a really important way forward to improve outcomes of particularly those children with the most difficult to treat cancers," he said.

Of the first 250 children enrolled in the program, the testing helped identify at least one new treatment that could help in 67 per cent (134) of those cases. In the children where new treatments were identified, 43 have received that therapy.

Rather than treating the cancer based on where it was in the body, genetic testing has allowed them to "lift the lid" on the cancer cells and find out not only what is driving the cancer, but also what might treat it best, he said.

"Cancer is a disease of genes. They're the normal genes in a cell that have gone wrong; they've been mutated, or there's too many of them, or there's not enough of them," Associate Professor Ekert said.

"Sometimes those molecular changes that are present in a cancer cell are really strongly indicative of a particular type of treatment that might work."

 
Thomas' father John Ebney said the trial has saved his son's life.

Thomas' father John Ebney said the trial has saved his son's life.CREDIT:PENNY STEPHENS

Hope and options

Thomas Edney was one of those children who had no standard therapy options left. First diagnosed with a rare brain cancer as a two-year-old, his parents John and Abigail discovered, after years of treatment, that the cancer had come back.

Doctors could offer little treatment for their then-six-year-old son other than surgery to remove as much of the tumour as possible, Mr Edney said.

"We were really, at that point, at a bit of a loss as to what to do," Mr Edney said. "The thing you look for in those circumstances is hope, and options."

 
 
 

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