Precision medicine in pediatric oncology: Lessons learned and next steps

Abstract

The maturation of genomic technologies has enabled new discoveries in disease pathogenesis as well as new approaches to patient care. In pediatric oncology, patients may now receive individualized genomic analysis to identify molecular aberrations of relevance for diagnosis and/or treatment. In this context, several recent clinical studies have begun to explore the feasibility and utility of genomics-driven precision medicine. Here, we review the major developments in this field, discuss current limitations, and explore aspects of the clinical implementation of precision medicine, which lack consensus. Lastly, we discuss ongoing scientific efforts in this arena, which may yield future clinical applications.

“Balancing Expectations with Actual Realities”: Conversations with Clinicians and Scientists in the First Year of a High-Risk Childhood Cancer Precision Medicine Trial

“…Precision medicine, with its focus on matching recommended treatments to specific pathogenic molecular aberrations, is a promising and paradigm-shifting strategy in childhood cancer treatment [1,2]. Access to precision medicine trials may be particularly impactful for paediatric cancer patients with poor-prognosis malignancies [3,4].…”

Approaching the third decade of paediatric palliative oncology investigation: historical progress and future directions

“…108 Furthermore, partly because of their novelty, late effects and toxicities from chimeric antigen receptor T-cell therapy and other targeted therapeutics are poorly understood. 109 Future investigations should explore the barriers to and optimal integration of PPC for these patients and families.…”

Molecular profiling in the clinic: Moving from feasibility assessment to evaluating clinical impact

“…This constitutes an important contribution to the already published prospective clinical sequencing studies (CSSs) in pediatric oncology. With six published CSSs in pediatric oncology, this manuscript should be considered in light of the general themes emerging in the nascent field of pediatric precision cancer medicine.…”

“…A recent review nicely summarizes potentially actionable variants most commonly identified by CSSs conducted in pediatric oncology to date. Consistent with some prior studies, the Pincez et al.…”

“…study identified copy number alterations in cell cycle genes as a common class of potentially actionable variants in their patient population. Also reviewed in Mody et al., there is a relationship between the sequencing platform utilized and the potentially actionable variants identified. The sequencing approaches utilized in the Pincez et al.…”