Targeting kids’ cancer, gene by gene

January 22, 2015

Cancer researchers in Australia are using a revolutionary ‘personalised medicine’ approach to identify effective individualised treatment options for children.

The personalised medicine platform, which is being developed and applied with the support of the Cancer Therapeutics CRC, will tailor each child’s cancer treatment to the particular genetics of their individual tumour.

Then, using a combination of in vitro cell growth and testing on mice, treatment will be determined by the response in the laboratory of their own cancer cells to drugs.

The project, led by Professor Michelle Haber, Executive Director of Australia’s Children’s Cancer Institute, in collaboration with the National Institutes of Health in the USA, has been kickstarted with approximately $7.5 million in funding from the CRC budget.

CCIA Laboratories, Lowy Institute“Although the survival rate of children’s cancer is now about 80%, this still means that on average about three kids in Australia are dying [from the disease] every week,” said Haber, who won the 2014 NSW Premier’s Award for Outstanding Cancer Research.

She said it was clear that individualised treatment is needed. “Two children can have the same diagnosis, but the standard treatment regimen will work for one child and fail with the other,” she explained.

The first step in the new approach is to take cells from a child’s tumour and run them through a set of molecular profiling tests, which reveal the genetic make-up of the cancer.

Haber’s team will soon settle on a panel of about 80 treatable genetic abnormalities for their targeted molecular profiling tests.
“We’ve trawled through the entire literature, pulling out what is known about genes that may be suitable for molecular targeted drug treatment,” she said. “This hasn’t been done for paediatric cancer before.”

The next step is to grow the child’s tumour cells. This is done either in laboratory flasks or in mice with deficient immune systems, known as ‘avatar mice’.

By rapidly scanning the cells, the researchers can test many drugs, either alone or in combinations, to see whether they knock back the cancer. And they don’t just try cancer drugs. Haber said that drugs as disparate as beta-blockers used in heart disease, as well as malaria drugs, can have anti-cancer effects.

Once a drug is shown to work in vitro, the next step is to use it in the avatar mice.

CCIA Laboratories, Lowy Institute“We have been very excited by the excellent responses of the first patients to have their therapy modified by their treating clinicians, on the basis of information being generated from this new personalised medicine platform,” said Haber.

Clinical trials of the platform, to be spearheaded by Sydney Children’s Hospital, are scheduled for 2017. However, Haber hopes it will be sooner than that.

“The CRC funding is invaluable,” she said. “It is paying for vital staff and their research supplies. Of course, this is just the beginning for the platform and we will only be able to handle a few patients at first.

“Our plan is that, eventually, the treatment platform will be offered to every child in the country who has a high-risk malignancy.”

Clare Pain

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