Breakthrough in quest to cure the incurable cancer that strikes kids
“Sometimes we have no answers and there is nothing.”
That is what Danielle and Darryl Squires were told just moments after their three-year-old daughter Monique was diagnosed with incurable brain cancer.
“No, we can’t operate. There is no chemo for this,” doctors said.
“Your child is going to die within six to 12 months.”
DIPG, or diffuse intrinsic pontine glioma, has been described as the worst of all child cancers.
Sneaky and cruel, it typically hits those aged around five to seven. It is aggressive, doesn’t respond to chemotherapy and, in an inoperable part of the brain stem, it is deemed terminal on diagnosis. It affects about 10 to 15 Australian children each year.
But now, more than a year after Monique Squires died, Australian researchers have made a significant breakthrough they hope could one day result in children surviving the disease.
Following more than 250 unsuccessful drug trials around the world, a team from the Children’s Cancer Institute in Sydney has been using a new approach.
Using donated tumours from children who have died, they have been able to regrow DIPG cancer cells in the lab, and they have subjected them to thousands of different drugs using a robot.
One combination is looking so promising it is likely adult drug trials could result within a couple of years.
It is a ray of hope that Danielle Squires did not have when Monique was diagnosed with DIPG in 2015.
The symptoms were mild at first. Monique complained about dizziness and would sometimes vomit in the morning. Danielle later noticed that she was squinting at the television and one of her eyes had turned in towards her noise.
“We thought maybe she needed glasses,” she said.
Monique had always been the biggest personality in the family. A twin, she was the larger one, born first, and loved being the centre of attention. So when she became unusually sleepy one day she was taken to the emergency department in Echuca, near her home on the border of Victoria and NSW.
Hours later, doctors at the Royal Children’s Hospital in Melbourne were telling the family that Monique had a terminal condition and that all they could offer them was radiotherapy, to perhaps buy her a little bit more time.
For a while, it did. Monique started kinder with her twin Zoe, but then the little warning signs returned, the droopy mouth, the limp. She was still keen to cut and draw with her classmates, but it had to be with her right side.
Monique’s parents were determined to find a miracle. They tried surgery to remove some of the tumour and Monique underwent 2½ bouts of radiotherapy before it became clear to them that their little girl was not going to recover any further, and they took her home.
She was no longer able to talk or walk. She died just after her firth birthday.
“It’s wrong,” said Danielle. “It shouldn’t be like this.”
The drug breakthrough from the Children’s Cancer Institute involves a combination of two drugs – one an untested medication, and another that has been used without success to treat other cancers.
Leading the research, Associate Professor David Ziegler, said the drugs by themselves had a mild effect on the cancer cells, but combined appeared to have profound results.
He said it targeted the basic building blocks the cells used to reproduce, at multiple points.
“Suddenly we are seeing unprecedented results where it completely stops the tumour growth, and prevents the tumour from causing any damage to the brain.”
While the drug combination is still a number of years from adult trials, and would need to go through safety testing and regulatory approval, there is hope that the cancer could now be treated in the future.
“For children with leukaemia, 50 years ago there was no treatment,” said Professor Ziegler.
“Over 50 years we have gone from an incurable disease, to an over 90 per cent survival rate.
"That’s what we are hoping to do with DIPG.”