# WINTHER

Source: https://onco.cc/trials/winther/  
OnCo record `winther` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

WINTHER was the first trial to pick cancer drugs using RNA, comparing what genes a tumour switched on against a biopsy of the patient's normal tissue, for patients whose DNA showed no obvious target. It missed its formal goal, but showed the approach was workable and that better-matched treatment went with better outcomes.

## Summary

WINTHER was run by the WIN Consortium, an international network founded around Gustave Roussy, and enrolled 303 patients between 2013 and 2015 at Gustave Roussy, Vall d'Hebron, Sheba Medical Center, McGill and UC San Diego. Each patient had a tumour biopsy and a matched normal tissue biopsy. Arm A used DNA sequencing (Foundation Medicine's panel) to match a targeted drug; patients with no DNA match moved to arm B, where a transcriptomic algorithm ranked drugs by the difference in gene expression between the tumour and the normal tissue. In all, 107 patients were treated, 69 in the DNA arm and 38 in the RNA arm.

The primary endpoint, comparing each patient's progression-free survival on study therapy with their progression-free survival on their previous line of treatment, was not met in either arm, so the trial is formally negative. The Nature Medicine report in 2019 nonetheless showed that RNA-based matching was feasible at scale across countries, that treatment recommendations could be produced within a clinically useful time, and that a higher matching score and less prior therapy were associated with longer progression-free survival, echoing I-PREDICT.

WINTHER's lasting effects are methodological: it normalised matched-normal biopsies, showed transcriptomics can widen the pool of patients with a rational option, and informed the design of later WIN Consortium trials that combine DNA, RNA and immune profiling. It also illustrated how hard it is to prove benefit with a within-patient comparison rather than a randomised control.

## Fields

- Kind: Trial
- Status: negative
- Last checked: 2026-09-17
- Also known as: WINTHER trial; WIN Consortium WINTHER
- Registry id: NCT01856296
- Phase: 2
- Setting: Advanced solid tumours after standard therapy across five centres in France, Spain, Israel, Canada and the United States: DNA sequencing, and where it finds nothing actionable, RNA expression of tumour against matched normal tissue, to choose therapy
- Sponsor: Gustave Roussy for the WIN Consortium
- Enrolled: 303
- Result: Primary endpoint (progression-free survival ratio against the prior line) not met in either arm; transcriptomic matching was feasible and higher matching scores tracked with longer progression-free survival.

## Sources

- ClinicalTrials.gov NCT01856296: https://clinicaltrials.gov/study/NCT01856296
- Rodon et al., Nature Medicine 2019: genomic and transcriptomic profiling expands precision cancer medicine, the WINTHER trial: https://doi.org/10.1038/s41591-019-0424-4

## Connected records

- terms: [Basket, umbrella, and platform trials](https://onco.cc/terms/basket-umbrella-platform/), [Master protocol (platform, basket and umbrella trials)](https://onco.cc/terms/master-protocol/), [Rare cancers](https://onco.cc/terms/rare-cancers/)
- technologies: [Cancer variant knowledgebases and molecular tumour boards](https://onco.cc/technologies/variant-knowledgebases/), [Comprehensive genomic profiling](https://onco.cc/technologies/cgp/), [RNA sequencing & expression profiling](https://onco.cc/technologies/rna-seq/)
- companies: [Foundation Medicine (Roche)](https://onco.cc/companies/foundation-medicine/)
- institutions: [Gustave Roussy](https://onco.cc/institutions/gustave-roussy/), [Rosalind and Morris Goodman Cancer Institute, McGill University](https://onco.cc/institutions/mcgill-goodman/), [Sheba Medical Center](https://onco.cc/institutions/sheba/), [UC San Diego Moores Cancer Center](https://onco.cc/institutions/ucsd-moores/), [Vall d'Hebron University Hospital / VHIO](https://onco.cc/institutions/vall-dhebron/)
- people: [Razelle Kurzrock](https://onco.cc/people/razelle-kurzrock/)

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