clinical trial has success for rare tumours

clinical trial has success for rare tumours

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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.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.”

Thomas’ oncologist recommended the ZERO program. It proved to be Thomas’ lifesaver.

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It led to a clinical trial for a drug called Afatinib, a drug which is more commonly used to treat lung cancer. It has shrunk Thomas’ tumour, but Mr Edney said he and his wife noticed changes in Thomas before they could see the effect of treatment on the brain scans.

“Pretty quickly after taking the medication, he no longer had constant headaches, there was no longer vomiting, his energy picked up, he was starting to return to school participating in normal activities,” he said.

Thomas is now eight years old, and the medication is still showing positive results.

“It’s life changing. Without it, he probably wouldn’t be here,” Mr Edney said.

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The future of childhood cancer treatment

Associate Professor Ziegler said the focus of the ZERO program was showing it was possible to do the genetic testing and get results back quickly enough to be able to make a difference for each child.

But they had not been able to see whether the children had been able to access the recommended drugs.

As well as improving access to new cancer treatments, the researchers also want to look at combining treatments to attack the cancer, as well as expand the program to include children with less aggressive tumours.

Dr Anthony Joshua, Associate Professor of Oncology at the Kinghorn Cancer Centre and UNSW, said this “world class” program was at the cutting edge of cancer treatment for children.

“It’s the foundation of what’s going to come in the next 50 years, so this is the way we’re going to advance cancer care both adults and kids,” he said.

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