An ambitious Australian programme to use personalised medicine to reduce the number of children who die of cancer to zero has already kept alive more than 150 children with aggressive cancers who would have otherwise died. The success of the scheme 鈥� the Zero Childhood Cancer Program, or Zero 鈥� means it will be made available to all Australian children with cancer from 2023.
One of Zero鈥檚 participants is Jack Burai in Sydney, who was diagnosed with a brain tumour in 2017, when he was 9 years old. His cancer was surgically removed, but came back aggressively a year later and spread through his brain and spine, leaving him unable to walk, eat or see out of his right eye. 鈥淚t was an end-of-life situation,鈥� recalls his father Alex. 鈥淲hen his mum was out of the hospital room, he would turn to me and say, 鈥楧ad, am I going to die?鈥�.鈥�
Now, Jack is a healthy, seemingly cancer-free 14-year-old who runs marathons and rides his BMX bike daily thanks to the personalised care he received through Zero. The idea of the programme is to move away from giving standardised treatments and to view every child鈥檚 cancer as unique, says at the Children鈥檚 Cancer Institute in Sydney, where Zero is based alongside the Sydney Children鈥檚 Hospital.
The first step is to try to work out more about each child鈥檚 cancer. To do this, Mayoh and her colleagues genetically sequence a child鈥檚 tumours and run other tests. Once the possible drivers are identified, the team searches medical literature and talks to colleagues to determine what sort of treatment might work best.
The team also tries a scattergun approach by testing more than 100 different cancer drugs on tumour cells taken from the child and grown in Petri dishes. Drugs that seem most active against the cells are then tested in mice that have been injected with the child鈥檚 tumour cells to check their safety and efficacy before they are given to the child.
In Jack鈥檚 case, genetic sequencing showed that a mutation called V600E in a gene called BRAF was driving his cancer. The Zero team knew that cancers with this mutation often respond to drugs called dabrafenib and trametinib and recommended them to his oncologists.
Within a week of taking these drugs, Jack was walking again, and after four weeks, he was playing tennis. Scans showed the tumours in his brain and spine rapidly shrinking, before disappearing altogether. 鈥淚t was truly miraculous because he had been slipping away in front of our eyes, and then he was suddenly back to his normal self,鈥� says his mother Viv.
Zero is the brainchild of , the executive director of the Children鈥檚 Cancer Institute. In 2013, she convened several cancer experts and explained her idea for the personalised medicine scheme. 鈥淭hey told me I was nuts, but they agreed to give it a go,鈥� she says.
After securing A$20 million (US$14 million) of funding from the Australian government, the team started by enrolling children with cancers that give people a less than a 30 per cent chance of survival, like Jack鈥檚. 鈥淭hese are the children whose parents are told, 鈥楪o spend some quality time together鈥�,鈥� says Mayoh.
Since 2015, 700 children have joined Zero from children鈥檚 cancer hospitals in Australia. Individual cancer drivers have been identified for 94 per cent of them and targeted treatments have been recommended for 72 per cent.
Of the children who have had targeted treatments, 31 per cent have responded, meaning their cancers have partially or completely disappeared or stabilised, says Mayoh, who will present the results at the next month.
With standard care, these children would have died, says Mayoh. 鈥淲e always said that helping just one patient would be amazing, so we think it鈥檚 been a huge success.鈥� Because the treatments in Zero are targeted, they also tend to have fewer side effects than conventional therapies, says Mayoh.
Zero hasn鈥檛 been able to save every child, often because they succumb to their disease before a personalised treatment can be found. The team has managed to cut the time it takes from sequencing a child鈥檚 tumour to giving them a personalised treatment from 12 to six weeks on average, and hopes to get it down to four.
The programme currently costs A$10,000 per child for the tumour sequencing and analysis, plus extra for the studies in cells and mice and the drugs that end up being prescribed.
Other countries are experimenting with personalised medicine programmes for children with cancer, but Zero is the most comprehensive to date, says Mayoh. 鈥淚 think it鈥檚 only a matter of time before other countries follow.鈥�
Zero was recently named by the US National Advisory Council for Human Genome Research as being one of the .
The scheme has also now secured another A$55 million from the Australian government to make it available to every Australian child with cancer from next year, not just those with the worst cancers.
Haber is confident we will get to a point where zero children die of cancer, especially as new immunotherapies and other treatments become available. 鈥淚n the 1950s, cancer was a death sentence for children, and now the survival rate is over 80 per cent,鈥� she says. 鈥淚 do think that personalising treatments 鈥� making sure you get the right drug for the right child at the right time 鈥� will get us towards our goal of one day curing all children of cancer.鈥�
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