World first Sydney trial aims to eradicate children's cancer by growing tumours in lab, testing treatments

By Anne Barker - ABC-Australia - Posted Wed at 5:55am - See Original Here

Scientists in Sydney hope a revolutionary new trial will help eradicate cancer as a cause of death for children in Australia.

Every year, 950 infants, children and adolescents in Australia are diagnosed with cancer and nearly three die each week.

Of those diagnosed about 150 have less than a 30 per cent chance of being cured — either because they have a particularly aggressive form of the disease or because they suffer a relapse, which is much harder to treat.

But scientists at Sydney's Children's Cancer Institute say a new approach where every individual tumour is analysed and replicated in the laboratory — then implanted in mice — will help them identify the best possible drugs to treat the disease, and ultimately cure even the most drug-resistant forms of cancer.

They have called the new campaign Zero Childhood Cancer — with the aim of reducing the death rate of children with cancer from the current level of 20 per cent to zero.

Those children most likely to benefit from the program are those suffering from the most aggressive forms of childhood brain tumours, sarcomas, infant leukaemias and neuroblastomas.

Maria Psaradellis, 9, was diagnosed as a toddler with a life-threatening cancer called Primitive Neuro Ectodermal Tumour (PNET). Her case is a prime example of a drug-resistant form of childhood cancer.

The tumour was found in her neck when she was just three months old, leaving her struggling for breath.

"It got to a stage where she'd go blue, get into a grunt, cry, then turn blue, her airway would close up," her mother Cathy Psaradellis said.

"I had to resuscitate her. So did my husband."

Today Maria shows no outward sign of illness. After months of treatment in hospital she went into remission when she was two.

She has no memory of the illness or the pain of treatment, and runs around like any other nine-year-old.

Although she now has an overactive thyroid and has taken several years to catch up on lost schooling.

There is also some ongoing pain.

"Every time I try pressing on my neck here it hurts a bit," she says with a grin on her face.

Seven years in remission means Maria's chance of a relapse are slim, but other kids are not so lucky.

Kayne Cook, 4, was diagnosed with a very rare and very aggressive brain cancer when he was only just three months old.

"He was waking up in the morning vomiting, he was screaming [in] pain at night and lethargic during the day," his mother Dani Cook said.

"And he became unbalanced on his feet. And he lost all his speech."

Ms Cook said the local hospital kept turning her son away until one night she refused to leave until more tests were done.

Finally he was diagnosed with cancer and was rushed to Sydney's Children's Hospital for surgery the next day.

At such a young age, Kayne had a tough time with the treatment. Chemotherapy and radiation gave him very painful ulcers from his mouth to his bowels, leaving him with lasting nerve damage.

Today he still has problems with balance and a slight tremor in his hands. He also faces the risk of needing hormone replacement.

After a year in remission, Kayne still faces a high risk of relapse and if the cancer returns he would have less than a 30 per cent chance of survival.

"When children relapse — or they have one of these very drug-resistant cancers — there is very little doctors can do," Professor Michelle Haber, the executive director of the Children's Cancer Institute said.

"There are so many drugs out there but they have no way knowing which are likely to work on a child's cancer."

One in five Australian children with cancer dies from the disease and there are thousands of drugs available, but because every tumour is different so knowing which to use is often a stab in the dark.

"They basically may try one or two things, but normally it's the end of the line," Professor Haber said.

Trial will allow scientists to test potentially fatal drugs.
But now, in a world-first trial, researchers are planning a new, more targeted approach by taking cancer cells out of the child and growing a smaller, identical form of the tumour in the lab.

High tech robots then screen the cells against potentially hundreds of drugs to see which ones show the most promise.

If the same approach was tried on the children themselves, the drugs would likely kill them.

In the controlled laboratory trial, the results are known within days and a shortlist of promising drugs is drawn up.

The same tumour is implanted in several mice who are each put on a different drug or combination of drugs.

The mouse which responds best — or ideally is cured — tells the scientists which drug is likely to work on the child.

There is no guarantee that what works for the mouse will cure the child, but scientists are optimistic.

"Our hope with this campaign is we take it to a 100 per cent cure rate," Professor Glenn Marshall, director of the Centre for Children's Cancer and Blood Disorders at Sydney Children's Hospital, said.

"Knowing which drugs will not be effective in a patient is as important as knowing which drugs will be effective."

In the first stage of the program, researchers and doctors will open a pilot study to high-risk NSW cancer patients in 2016.

After the successful completion of the pilot study, a national clinical trial involving 120 children will begin in 2017.

Once it is fully implemented the program will be offered to children throughout Australia who are at the highest risk of relapse or treatment failure.

"This is a very exciting initiative that will revolutionise the way in which treatment decisions about childhood cancer are made," Professor Haber said.

Families dealing with childhood cancer are also optimistic.

"It will give a lot of hope to families with a high risk of cancer recurring and to kids who relapse," Kayne's mother Ms Cook said.

"If he were to relapse there'd be no chemotherapy option but this gives great hope if he relapses that there'll be something there waiting for him.

"I never want to be in that situation, but it's always good to know that there's something there."