New treatment for all cancers could save babies with leukaemia

A new drug that improves the performance of chemotherapy is providing hope for all cancer patients, but is particularly promising for babies who can die from their treatment.

Exclusive: A breakthrough holy grail drug is providing hope to tiny babies at risk of dying from an aggressive form of Acute Lymphoblastic Leukaemia — and ultimately could help all cancer patients.

A team from the Children’s Cancer Institute in Sydney has proven a ferocious form of the blood cancer that kills half the infants who contract it, became undetectable in mice treated with chemotherapy and the new drug CBL0137.

But Children’s Cancer Institute researcher Dr Klaartje Somers says it has even greater promise because it can be used in all types of cancer including solid tumours like breast and bowel cancer.

Trials are underway in adults in America and it is hoped children in Australia whose cancer has returned will get the chance to take part in the first trial of the new drug in minors at the Sydney Children’s Hospital next year.

New treatment provides great hope for childhood cancers. Picture: Getty Images

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The drug works by reactivating the body’s P53 pathway, so it kills off damaged cells.

This pathway is commonly mutated in cancer patients and cancer cells suppress it so they can stay alive and keep spreading.

In the International Journal of Cance r, Dr Somers reported her study where mouse avatars who were given cancer cells from sick Australian patients were successfully treated with CBL017 combined with chemotherapy.

“When we used the molecule or the chemotherapy by themselves there was a small response but when combined the treatments were highly effective and deleted the cancer cells in the mouse,” Dr Somers said.

Dr Somers’ work, under the lead of Dr Michelle Henderson and Professor Michelle Haber, focuses on a deadly blood cancer that devastates tiny babies.

Half the babies with a mutation in their MLL gene will die from the aggressive chemotherapy required to beat it.

Those who survive are at risk of heart problems, mental retardation, growth problems and thyroid and breast cancer, when they become adults.

Dr Somers hopes by combining the new treatment with chemotherapy doctors will be able to dial down the amount of chemotherapy required and reduce these side effects.

Ava Barker is now thriving after a bone-marrow transplant. Picture: Justin Lloyd

As a toddler Ava Barker was made so ill by chemotherapy she couldn’t speak, walk or smile and in her darkest moments her mother Kirsty O’Brien asked herself if she was doing the right thing.

“The hardest thing as a parent was to watch your child be so unwell from the treatment and you questioned whether you were making the right decision,” she said.

Ava developed leukaemia as an 18-month-old and endured 19 months of chemotherapy but the cancer returned when she was four and she had to have even more aggressive chemotherapy followed by a bone-marrow transplant.

The preschooler had to be kept in an isolation room at the hospital for 100 days and when she came home had to live in a bubble for months to prevent infection that could put her life at risk.

Now a thriving nine-year-old she plays soccer, Austag, hockey, T-ball, loves dancing and is taking part in her first Eisteddfod.

Ms O’Brien said she was excited by the promise of the new cancer treatment which could help other families facing the devastating treatments required for childhood cancer.

“If you can make the treatment less invasive on their little bodies, as a parent, that’s what you want,” she said.