Yana Pikman, MD
An advanced understanding of the biology of hematologic malignancies has significantly improved survival outcomes. However, in pediatric acute leukemias, said Yana Pikman, MD, the integration of real-time genomic sequencing data into clinical practice has fallen behind.
, Pikman further discussed the clinical implications of using this approach to match targeted therapy to pediatric patients with leukemia.
OncLive: Please provide some background to this study.
: Pediatric acute leukemia is the number 1 cause of pediatric cancer. In 2018, it still accounts for a significant number of cancer-related deaths; in fact, it is the number 2 cause of pediatric cancer deaths overall. We need to find a way to provide better treatment for these patients.
The study we are conducting is evaluating the feasibility of identifying targetable mutations in pediatric patients with relapsed or high-risk leukemia. [Our strategy is to] match these targets with available therapies and create a precision medicine approach in this space.
What is the design of the trial?
At the time of enrollment, patients have a sample of their leukemia sequenced; those data are then reviewed by a panel comprised of pediatric oncologists, experts in therapeutics, and pathologists. This [process] is to determine if we are able to identify any targetable mutations. If a targetable recommendation is made, we pass that information along. [It is important to remember that] this is not a therapeutic study; it is a feasibility study. Therefore, we are not treating any patient, but we hope this research paves the way for a therapeutic study.
We are discovering several novel genomic alterations that can be of interest and we are now evaluating the significance of these [alterations]. Ongoing work is happening to see if we can tailor our approach to these [alterations].
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