Repositioning canakinumab for non-small cell lung cancer-important lessons for drug repurposing in oncology
Related Experiment Video
Updated: Sep 6, 2025

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Mark P Lythgoe1, Vinay Prasad2
1Department of Surgery and Cancer, Imperial College London, Hammersmith Hospital, Du Cane Road, London, W12 0HS, UK. M.Lythgoe@imperial.ac.uk.
Abstract:
Canakinumab is an anti-interleukin-1β monoclonal antibody approved for use in a range of immune-related disorders. During the clinical investigation (CANTOS trial) for prevention of cardiovascular complications, therapy was linked to a reduction in both the occurrence and mortality of lung cancer. This unexpected observation fuelled the rapid initiation of four large clinical trials to evaluate potential anticancer efficacy (in combination with chemotherapy and/or immunotherapy), before fully validating these observations in a dedicated study. The first two trials (CANOPY-1 and 2) have now been reported and have both have failed to meet their primary efficacy endpoints. In this article, we explore the scientific and clinical rationale behind the development of canakinumab in oncology, the repurposing approach utilised and implications this may have for the wider drug repurposing field in the development of new cancer medicines.
Insights
Canakinumab, an anti-interleukin-1β therapy, showed promise in reducing lung cancer in a cardiovascular trial. However, subsequent oncology trials (CANOPY-1 and 2) failed to meet primary efficacy endpoints, impacting cancer drug repurposing strategies.
Area of Science:
- Oncology
- Immunology
- Drug Repurposing
Background:
- Canakinumab (anti-interleukin-1β) is approved for immune-related disorders.
- The CANTOS trial observed reduced lung cancer incidence and mortality with canakinumab.
- This led to trials investigating canakinumab for cancer treatment.
Purpose of the Study:
- To explore the rationale for developing canakinumab in oncology.
- To analyze the drug repurposing approach for canakinumab in cancer therapy.
- To discuss the implications for future cancer drug development.
Main Methods:
- Review of scientific and clinical rationale for canakinumab in oncology.
- Analysis of the drug repurposing strategy.
- Evaluation of reported clinical trial outcomes (CANOPY-1 and 2).
Main Results:
- Canakinumab demonstrated an unexpected reduction in lung cancer in the CANTOS trial.
- The CANOPY-1 and CANOPY-2 trials did not meet their primary efficacy endpoints.
- The initial promising signal for canakinumab in lung cancer was not validated.
Conclusions:
- The development of canakinumab in oncology, based on initial observations, faced setbacks.
- The failure of CANOPY-1 and 2 highlights challenges in drug repurposing for cancer.
- This case study offers insights into the complexities of developing new cancer medicines through repurposed drugs.
Related Concept Videos
Targeted Cancer Therapies
There are several types of targeted therapies against...
Treatment Resistant Cancers
Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
Tumor Immunotherapy
Cancer Therapies
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...

