Research co-led by a Newcastle professor is already changing how children with a type of leukaemia are treated, potentially sparing some from the full effects of chemotherapy. The study, published in the medical journal Blood Advances, is the first of its kind and focuses on the immunotherapy drug blinatumomab.
Breakthrough in childhood leukaemia treatment
Professor Anthony Moorman, from Newcastle, worked with Dr Sujith Samarasinghe, a consultant at Great Ormond Street Hospital in London, to show that some children can be treated with blinatumomab and a reduced course of chemotherapy. This matters because chemotherapy can be dangerous, and around a third of young patients experience side effects from their treatment.
Prof Moorman told ChronicleLive: "The basic background is that this is about childhood leukaemia. We have treated patients with chemotherapy for decades and decades. Through iterative improvements, we have successfully improved outcomes, and in the last few years there has been a new drug in town - blinatumomab. This is a different kind of drug: It's an immunotherapy drug and is not chemotherapy."
He added: "We have known for a little while that it's a great drug for treatment, but there have been unanswered questions - what's the best dose and scheduling for it? Which patients will benefit most? And what this study shows very clearly is that there is a group of patients who can be treated with blinatumomab and a reduced course of chemotherapy."
Predicting relapse and personalising care
The research also found that how a child's body responds to blinatumomab can predict how likely they are to relapse after initial cancer treatment. Prof Moorman explained that this enables a more targeted, "personalised" treatment approach.
He said: "Blinatumomab is transforming the treatment of childhood ALL, but until now we did not know whether the risk factors used for decades in chemotherapy-based treatment still applied. Our findings show that many of them do not. Instead, a child's early response to blinatumomab, together with specific genetic features of their leukaemia, provides a much more accurate picture of relapse risk. This research will help clinicians identify which patients are likely to do exceptionally well with reduced chemotherapy and which will require a different treatment approach."
The study involved more than 200 young patients with B-cell acute lymphoblastic leukaemia treated across the UK and Ireland. Their responses were analysed, including through genetic testing, and two "genetic subtypes" of the illness were identified for which patients respond more poorly to treatment.
Already changing UK treatment guidelines
Prof Moorman said the findings have already led to changes in treatment guidelines for children with ALL under the age of 18. He noted: "This has been a national study involving children across the UK. And now on the basis of these results we have already been able to alter the treatment guidelines for children with ALL under the age of 18. For children being diagnosed now, some of them may well be treated differently because of this work."
Dr Samarasinghe added: "Being able to accurately predict how this kinder, more effective medicine will help our patients means less chemotherapy, more days at school, more time at home and ultimately more time spent being children. We are already seeing the real-world impact of this work, with the findings now helping guide how children across the UK are treated."
The research was supported by Horizon Europe, Marie Slodowska-Curie Action and Cancer Research UK. Further work will now consider why some kinds of leukaemia are resistant to blinatumomab.
