A groundbreaking study from cancer researchers at The Kids Research Institute Australia has identified a promising new therapeutic strategy for children battling the most common childhood cancer – B-cell acute lymphoblastic leukaemia (B-ALL).
While overall survival rates for B-ALL have reached nearly 90% over the past 50 years, certain sub-groups continue to face poor outcomes. The discovery brings a new approach to treating children who face these tougher cases of leukaemia.
B-ALL starts in the bone marrow – the soft tissue inside bones where blood cells are made. In children with B-ALL, the disease often causes bone damage. One reason for this damage is the activity of a bone cell called osteoclasts. Osteoclasts, which normally help in bone remodelling, have now been found to cause the bone loss often observed in children with leukaemia.
The study focused on whether targeting osteoclast cells with the bone-protecting treatment zoledronic acid (ZA) could not only restore bone health in children but also enhance overall treatment efficacy for children with B-ALL.
Co-head of the Institute's Leukaemia Translational Research Team, Associate Professor Laurence Cheung, said that using zoledronic acid to target osteoclasts can really change the way we treat high-risk childhood leukaemia.
"By keeping the bone environment healthier, we see improvements in both bone strength and overall survival when ZA is added to the usual treatments,” Associate Professor Cheung said.
The researchers used special imaging tools to look at how bones responded to the treatment. The images showed that ZA reduced the number of osteoclasts and helped reverse the damage caused by B-ALL, such as bone loss and thinning.
The results were replicated using cells from a Perth Children’s Hospital patient – a 14-year-old boy with relapsed B-ALL. In this model, ZA again improved survival and helped restore healthy bone structure.
The study went further by combining ZA with common chemotherapies used to treat B-ALL. Even in cases where the disease burden was high, this combination treatment led to better survival than chemotherapy alone.
The final part of the study demonstrated safety and feasibility of administering ZA to children receiving chemotherapy for B-ALL.
“In our clinical cases, we also saw that children with severe bone complications benefited from ZA,” Associate Professor Cheung said.
“It was safe to use with other treatments, and it helped reverse bone loss without causing any significant adverse effects.”
The findings show that ZA may offer a two-fold benefit for children with B-ALL: protecting their bones and boosting the effectiveness of standard cancer treatments. The research team now hopes to see clinical trials to further test ZA as a part of everyday treatment for childhood leukaemia.