How effective is the personalized off-label use of targeted cancer treatment?

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15 April 2026 How effective is the personalized off-label use of targeted cancer treatment? If general cancer treatment fails, a tumour-type-specific therapy might be tried for other cancers with genetic changes in the targeted pathway. How well does this work? By Funda Meric-Bernstam 0 Funda Meric-Bernstam Funda Meric-Bernstam is in the Department of Investigational Cancer Therapeutics, University of Texas MD Anderson Cancer Center, Houston, Texas 77030, USA. View author publications PubMed Google Scholar

Given that there are many emerging targets in the efforts to treat cancer, precision medicine is in the spotlight. Many DNA changes — such as mutations, changes in the number of copies of a gene or fusions of genes — can drive cancer-cell growth and proliferation, and can affect sensitivity or resistance to selected therapies. It is important to know whether a change can be successfully targeted — in other words, whether it is actionable — and whether a therapy that works by targeting a genetic alteration in one type of cancer will be equally effective in other types. Writing in Nature, Verkerk et al.1 report the results from a stage of a clinical trial from the Netherlands called the Drug Rediscovery Protocol (DRUP; see go.nature.com/3ocpqtu).

doi: https://doi.org/10.1038/d41586-026-00911-3

References Verkerk, K. et al. Nature https://doi.org/10.1038/s41586-026-10405-x (2026). O’Dwyer, P. J. et al. Nature Med. 29, 1349–1357 (2023). Le Tourneau, C. et al. Lancet Oncol. 16, 1324–1334 (2015). Download references

Competing Interests The author declares competing interests as follows. Consulting: AstraZeneca Pharmaceuticals; Becton Dickinson; Biocartis NV; Boehringer Ingelheim International GmbH; Calibr (a division of Scripps Research); Crossbridge Bio; Daiichi Sankyo; Dava Oncology; Debiopharm International; DEM BioPharma Inc; EcoR1 Capital; eFFECTOR Therapeutics; Elevation Oncology; Exelixis; GT Aperion; Incyte Corporation; Jazz Pharmaceuticals; LigaChem Biosciences (formally LegoChem); Menarini Group; ModeX Therapeutics, Inc.; Molecular Templates; Precede Biosciences Inc; Protai Bio; Ribometrix; SystImmune; Tacalyx GmbH; TEMPUS; TORL BioTherapeutics; Vir Biotechnology; Zymeworks. Advisory committee: Cybrexa Therapeutics; DEM BioPharma Inc.; go Therapeutics; Guardant Health; Harbinger Health; Illumen Therapeutics; Kivu Biosciences; LOXO-Oncology; OnCusp Therapeutics; Seagen (formerly Seattle Genetics); Sutro Biopharma Inc; Theratechnologies Inc; Zentalis Pharmaceuticals. Sponsored research (to the institution): AstraZeneca; Daiichi Sankyo Co. Ltd.; Debiopharm International; eFFECTOR Therapeutics; Guardant Health Inc; Jazz Pharmaceuticals; Zymeworks.

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