How robots and ‘smart-bomb’ drugs saved dying baby Ellie
LITTLE Ellie Caterjian was not expected to see her first birthday. But the plucky youngster is alive today thanks to new ‘smart bomb’ cancer drugs which will revolutionise treatment for other gravely-ill children.
NSW
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JUST a year ago, Ellie Caterjian could barely breathe because the 11-month-old had a tumour the size of a football inside her chest.
Terrified parents Mina and Rob were given the devastating news that the rare cancer was resistant to chemotherapy.
“We were told we might lose her. Here is our perfect child, our only child and we just could not take it all in,” Mrs Caterjian told The Sunday Telegraph.
“She did one round of chemo and it did nothing and it was too big to operate on. She was so sick we didn’t even know if she would reach her first birthday.”
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But Ellie, with her rosy cheeks and glossy dark curls, enjoyed her second birthday yesterday.
Her miraculous recovery is thanks to the Zero Childhood Cancer personalised medicine program.
Some cancers, like Ellie’s, are unique and require medicines tailored to attack the specific tumour.
The Zero Childhood Cancer program, led by the Children’s Cancer Institute and the Kids Cancer Centre at Sydney Children’s Hospital, is pioneering this approach to personalised medicine.
Ellie’s tumour was analysed for its genetic makeup and it was identified as having a rare genetic rearrangement that could be targeted with a new drug called larotrectinib — only available in the USA.
Loxo Oncology, the makers of the drug, provided compassionate access to Ellie.
The drug was like a smart bomb, which went straight for the tumour.
The turnaround was remarkable, with the cancer shrinking from the size of a football to the size of a coin.
Barely nine weeks after diagnosis, Ellie came off life support, had her tracheal tube removed and said her first words.
“I heard her talk for the first time — she said ‘Mumma’ and it just broke me,” Mrs Caterjian said.
Today, on Father’s Day, Mr Caterjian knows his daughter’s second birthday is a gift from the Zero Childhood Cancer trial.
“It’s the only present I wanted, I wanted a healthy, happy girl, that’s it,” he said.
Executive Director of the Children’s Cancer Institute Professor Michelle Haber said Ellie’s case offered hope to others.
“To see a tumour like Ellie’s, that would have killed that child and to find a drug that destroyed it is just fantastic,” Prof Haber said.
And the drug only targeted the cancer cells, not healthy cells, so Ellie was spared the usual side effects of chemotherapy.
Science has made incredible bounds in childhood cancer, converting certain tragedy to a survival rate of up to 80 per cent but at least three children still die every week in Australia from cancers that do not respond to standard treatment.
Around half of all children have a cancer that does not have a detectible genetic marker.
For these children, a robot called Baby uses tiny amounts of biopsied cancer cells to find out which one of the more than 100 anti-cancer drugs available will work best.
“It’s a precision medicine program that provides the best options for children,” Prof Haber said.
“It is an incredibly exciting time to be in cancer research. This is the future of medicine.”
A clinical trial of larotrectinib, the drug used to treat Ellie, is now open in Australia for all children whose cancer is identified by the Zero Childhood Cancer program as having the same genetic marker.
The Sunday Telegraph is a proud partner of the Children’s Cancer Institute, which is wholly dedicated to curing childhood cancer. September is Childhood cancer awareness month. To support the institute’s world-leading research, visit www.ccia.org.au