Matched targeted therapy for paediatric patients with relapsed, refractory or high-risk leukemias

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Published: 6 Dec 2018
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Dr Yana Pikman - Dana Farber Cancer Institute, Boston, USA

Dr Yana Pikman speaks to ecancer at ASH 2018 about the LEAP consortium and targeted therapy for paediatric leukaemia patients.

She explains that there are a number of genomic changes in paediatric leukaemia patients that could be targeted, with 13% of patients showing these changes in the study.

Although we’ve made significant progress in the treatment of acute leukaemia this still is the second leading cause of cancer-related death in children so we really need to do better in terms of understanding the disease, the genomics and how to really improve therapies for our patients. Way too many kids die from acute leukaemia.

So the goal of the study was to do that, was to really understand the genomics and how we can use some of the available targeted therapies to help treat kids with cancer and kids with leukaemia specifically. In order to conduct the study we established the LEAP consortium, it is now fifteen institutions across the United States, and we were extremely lucky to get funding and support from the St. Baldrick’s consortium grant to actually be able to fund this study.

A number of the genomic findings that we thought we would find, it’s unclear how relevant they are to the pathogenesis, so whether they could be targeted using targeted therapies. So there are a number of other research studies that are part of this clinical trial that will help us to understand how to best treat kids with leukaemia.

What have you found so far?

So far we’ve found that there are a number of genomic changes, DNA changes, in the leukaemia of paediatric patients with relapsed or refractory disease that actually could be targeted. It’s 13% of our patients that had targetable mutations and if we look at all types of targetable mutations, so those that have both clinical trial evidence to support the therapy as well as pre-clinical trial evidence, it’s actually as high as 70% of our patients that have had targeted therapy recommendations. We’ve also seen patients where the targeted therapy recommendations was used by the treating oncologist so there are certainly a number of patients on our study where the genomic characterisation of the leukaemia that happened as part of the study has really altered the therapy for these patients.

We are also working with Dr Jeff Tyner’s group in Oregon to study the drug sensitivity of primary patient leukaemia cells to a wide range of drugs and we hope that this will inform some of the future studies for targeted therapies in paediatric patients with acute leukaemia.