A pilot clinical study, led in Australia by a Telethon Kids Institute and Perth Children’s Hospital researcher, has found an immunotherapy drug can dramatically increase survival rates for babies with a rare form of leukaemia, paving the way for a major international clinical trial.

The phase two study tested the safety and effectiveness of a drug called blinatumomab for the treatment of acute lymphoblastic leukaemia (ALL) in infants diagnosed under 12 months of age.

The results, published in the prestigious New England Medical Journal, showed that it was safe and feasible to administer blinatumomab to babies with ALL and remarkably also identified a strong signal for efficacy, with an almost 30% improvement in disease-free survival at two years from diagnosis – from 49.4% to 81.6%.

Associate Professor Rishi Kotecha, co-head of the Telethon Kids Institute’s Leukaemia Translational Research team and consultant in clinical haematology and oncology at Perth Children’s Hospital, said the small-scale pilot study involving 30 babies from around the world, has significant global implications for the disease.

“Infant ALL constitutes a subgroup of childhood leukaemia which has really poor survival rates, significantly lower than many of the other forms of blood cancers we see in children.”

“The pilot study has shown a massive early improvement to survival, and it really shows that this strategy can have a real impact on the way we treat babies with ALL worldwide,” he said.

Findings from the pilot trial will now be expanded to test the drug in a larger cohort of babies in the upcoming global Interfant-21 trial. Thanks to further funding from the Medical Research Future Fund and The Kids’ Cancer Project, the trial will involve all 10 of the tertiary paediatric cancer centres in Australia and New Zealand, including Perth Children’s Hospital.

Associate Professor Kotecha said there have been very few improvements in survival rates for babies with ALL over the past 20 years, and the new trial was an exciting step forward.

“Blinatumomab is an immunotherapy drug that links the immune system to destroy the leukaemia cells,” Associate Professor Kotecha said.

“What’s exciting is that it doesn’t have the toxic side effects we see from chemotherapy, which is known to kill healthy cells as well as cancer cells. This is what causes the horrendous side effects we associate with chemotherapy, and when we’re treating very young babies it’s particularly distressing.”

“The immunotherapy allows babies the chance to recover in between the chemotherapy cycles, while still attacking the cancer cells,” Associate Professor Kotecha said.

In the pilot trial, babies were still given conventional chemotherapy in addition to blinatumomab, but in the upcoming Interfant-21 trial, one of the chemotherapy treatment blocks will be completely replaced by the immunotherapy drug.

“The most distressing thing is seeing a newborn baby with this disease; for parents it’s the worst scenario you can imagine.”

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With this treatment strategy, in addition to improving survival outcomes we’re hoping that the babies will not only experience less short-term toxicity but also fewer of the long-term side effects as well.

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Recruitment of babies with ALL from Australia and New Zealand into the new clinical trial will begin in the third quarter of 2023.

Associate Professor Kotecha will continue in his role from Perth as the National Principal Investigator for the Interfant-21 trial, with continued support for centralised trial management and administration of funding from the Australian and New Zealand Children’s Haematology/Oncology Group, based at the Hudson Institute of Medical Research, Monash University.