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Feasibility of functional precision medicine for guiding treatment of relapsed or refractory pediatric cancers.

Arlet M Acanda De La Rocha1, Noah E Berlow2, Maggie Fader3

  • 1Department of Environmental Health Sciences, Robert Stempel College of Public Health & Social Work, Florida International University, Miami, FL, USA.

Nature Medicine
|April 11, 2024
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Functional precision medicine (FPM) offers new hope for children with rare cancers. Combining genomic profiling and drug sensitivity testing (DST), FPM identified effective treatments for most patients, improving outcomes for those receiving FPM-guided therapy.

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Area of Science:

  • Pediatric Oncology
  • Genomics
  • Precision Medicine

Background:

  • Children with rare, relapsed, or refractory cancers have limited treatment options.
  • Few predictive biomarkers exist for personalized treatment in pediatric oncology.

Purpose of the Study:

  • To assess the feasibility of functional precision medicine (FPM) for pediatric patients with relapsed or refractory cancer.
  • To determine if FPM-based treatment recommendations can be delivered in real-time (<4 weeks).
  • To evaluate clinical outcomes associated with FPM-guided treatments.

Main Methods:

  • Prospective observational study of 25 pediatric patients with relapsed/refractory cancers.
  • Genomic profiling and drug sensitivity testing (DST) on patient-derived tumor cells.
  • Real-time FPM tumor board (FPMTB) review for treatment recommendations.

Main Results:

  • FPM recommendations were made for 76% of patients (19/25).
  • Median turnaround times for DST and genomics were 10 and 27 days, respectively.
  • Six patients received FPM-guided therapy, with 83% showing improved progression-free survival.

Conclusions:

  • FPM is a feasible approach for identifying personalized treatments in pediatric relapsed/refractory cancers.
  • FPM-guided therapy demonstrated improved clinical outcomes compared to non-guided treatments.
  • Further validation in larger prospective studies is warranted to confirm FPM's impact.