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ASCO 2025 | Precision medicine in pediatric cancer treatment

Francois Doz, MD, Institut Curie, Paris, France, emphasizes the potential of precision medicine in pediatric cancers, particularly in cases like the the Phase I/II iMATRIX Alectinib trial (NCT04774718) of alectinib in children and adolescents with ALK fusion-positive solid or CNS tumors. However, he notes that the main barrier to its adoption is the need for comprehensive molecular documentation at diagnosis, including sequencing and genome sequencing, to enable rapid treatment adjustments. This is further complicated by the need for timely access to treatment results through clinical trials to ensure efficacy and safety. This interview took place during the 2025 American Society of Clinical Oncology (ASCO) Meeting in Chicago, IL.

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Transcript

What is precision medicine? Precision medicine is, of course, a big step, but is probably not concerning all pediatric cancers. But we should not miss the opportunity to use it when it’s so active and so specific than, for instance, this Alectinib study. And the main barrier is the right and full documentation, molecular documentation of all pediatric cancers at diagnosis with sequencing, RNA sequencing, genome sequencing, and to have the data quickly available to make rapid changes in the treatment...

What is precision medicine? Precision medicine is, of course, a big step, but is probably not concerning all pediatric cancers. But we should not miss the opportunity to use it when it’s so active and so specific than, for instance, this Alectinib study. And the main barrier is the right and full documentation, molecular documentation of all pediatric cancers at diagnosis with sequencing, RNA sequencing, genome sequencing, and to have the data quickly available to make rapid changes in the treatment. So that is probably the main barrier and to have the results soon enough to be able to use these treatments, preferably in the context of clinical trials, except that we have already targeted treatments which are now standard treatment even in first line in some indications. But for those that are developed, we still need to do clinical trials. And this is a second barrier to have access to this drug through clinical trials in order to be able to be sure that they are about the efficacy and toxicity.

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