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

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).

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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