Could Functional Precision Medicine Be the New Precision Oncology?
Research news: In an NCI commentary dated Aug 6, 2026, Anthony Letai, MD, PhD, argues that “functional precision medicine”—testing a patient’s living cancer cells against therapies—could complement genomics-based precision oncology. Letai cites past laboratory sensitivity testing that helped advance venetoclax toward later FDA approval in CLL, while emphasizing that broader clinical utility of functional testing will require rigorous evidence, standardized workflows, and practical implementation pathways.
Could Functional Precision Medicine Be the New Precision Oncology? (Research news)
Date: Aug 6, 2026
In a National Cancer Institute (NCI) commentary, NCI Director Anthony Letai, MD, PhD, described “functional precision medicine” as a potential complement to mutation-guided precision oncology. Rather than selecting therapy based primarily on tumor genomics, functional precision medicine tests a patient’s living cancer cells ex vivo against candidate treatments to observe drug response.
Letai notes that while genomics has enabled major advances (e.g., imatinib in CML), only a small proportion of patients benefit from mutation-targeted therapies, suggesting limitations to genomics alone. He likens functional testing to antibiotic susceptibility testing in microbiology.
As an example of functional testing informing translation, Letai recounts early lab experiments in chronic lymphocytic leukemia (CLL) in which exposure of patient leukemia cells to a novel compound led to cell death; he writes that this “paved the clinical path” for later FDA approval of venetoclax for CLL. (This article does not report a new regulatory action.)
Letai reports that several NCI-Designated Cancer Centers are conducting feasibility studies and clinical trials incorporating functional precision medicine, and that ASCO has emphasized support for its advancement. He also highlights implementation challenges discussed at NCI’s July workshop in Rockville, Maryland, including preserving viable tissue, standardizing sample handling across laboratories, and barriers related to insurance coverage and guidelines for associated testing and off-label drug use.