HOLY GRAIL CANCER DRUG

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 proved that a ferocious form of the blood cancer, which kills half the infants who contract it, became undetectable in mice that were treated with chemotherapy and the new drug CBL0137.

But Children’s Cancer Institute researcher Dr Klaartje Somers said the treatment had even greater promise because it could be used in all types of cancer, including solid tumours such as breast and bowel cancer.

Trials are underway in adults in the US 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.

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 Cancer, Dr Somers reported her study where mouse avatars who were given cancer cells from sick Australian patients were successfully treated with CBL0137 and 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.