New drug hope for babies with leukaemia

New drug hope for babies with leukaemia Researchers at Telethon Kids Institute have discovered a new drug combination.

Researchers at the Telethon Kids Institute have discovered a new drug combination that could help improve survival rates for babies with leukaemia.

The findings, published today in the leading international journal Leukemia, gives new hope to babies under the age of 12 months who have poorer outcomes compared to older children.

Head of Leukaemia and Cancer Research at the Telethon Kids Institute, Professor Ursula Kees said while there have been significant improvements in cure rates for children over 12 months of age, cure rates for children under that age have remained stagnant over the past 20 years.

“The chance of survival for a child over the age of one diagnosed with the most common form of leukaemia has steadily increased to over 90 per cent,” Professor Kees said.

“Unfortunately though, the cure rate for an aggressive type of leukaemia in children under 12 months is still only 40 per cent.”

But the study’s lead author, Dr Mark Cruickshank from the Telethon Kids Institute, said this new research, supported by the Children’s Leukaemia and Cancer Research Foundation and The Kids Cancer Project, could change that.

“Using leukaemia cancer cells from babies, our team has developed a collection of sophisticated laboratory models,” Dr Cruickshank said.
Read more: In Focus: Telethon Kids Institute.

“For the first time, we have directly compared cells grown in test tubes with patient samples, allowing us to measure the reproducibility with unprecedented resolution.”

“We found that these models accurately mimic the diverse and aggressive features of cancer from very young patients.”

Professor Kees said the team used these models to test a range of drugs, which are already used in some adult cancer treatments but aren’t used to treat childhood cancer.

“What we found was one drug in particular which enhanced the effect of an important drug that is commonly used to treat childhood leukaemia,” said Professor Kees. 

Telethon Kids Institute researcher and Oncologist, Dr Rishi Kotecha said not only did this new drug make the existing drug more effective when used in combination, it also appeared less toxic than currently used drugs.

“The reason we have such difficulty treating babies with leukaemia is because the treatment we use, chemotherapy, is very toxic to their small bodies,” said Dr Kotecha. “It is very effective in destroying the cancer cells but unfortunately it destroys healthy cells as well.”

“Our findings could see changes to the current dosage schedule, so babies are exposed to less chemotherapy when used in combination with the new drug," he said.

Researchers now plan to refine the timing and dosage of the drug combination with the aim for the combination to be integrated into future clinical trials.
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