A pilot clinical study 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 – led by The Kids Research Institute Australia and Perth Children’s Hospital’s Associate Professor Rishi Kotecha - 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 it was safe and feasible to administer blinatumomab to babies with ALL and, remarkably, also identified a strong signal for efficacy, with over 30 per cent improvement in disease-free survival at two years from diagnosis – from 49.4 per cent to 81.6 per cent.
Associate Professor Kotecha, co-head of The Kids Leukaemia Translational Research team and consultant in clinical haematology and oncology at PCH, said the small-scale pilot study involving 30 babies from around the world had 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,” he said.
“The most distressing thing is seeing a newborn baby with this disease; for parents it’s the worst scenario you can imagine.
“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.”
Associate Professor Kotecha said there had been very few improvements in survival rates for babies with ALL over the past 20 years, so new trial was an exciting step forward.
“Blinatumomab is an immunotherapy drug that links the immune system to destroy the leukaemia cells.
“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 chemotherapy cycles, while still attacking the cancer cells.
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.
Next steps
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, involving all 10 of the tertiary paediatric cancer centres in Australia and New Zealand, including Perth Children’s Hospital