Related Experiment Video
Updated: Nov 21, 2025

Intramucosal Inoculation of Squamous Cell Carcinoma Cells in Mice for Tumor Immune Profiling and Treatment Response Assessment
Published on: April 22, 2019
Trends in Phase II Trials for Cancer Therapies
Faruque Azam1, Alexei Vazquez1,2
1Wolfson Wohl Cancer Research Centre, Institute of Cancer Sciences, University of Glasgow, Garscube Estate, Switchback Road, Bearsden, Glasgow G61 1QH, UK.
Abstract:
Background: Drug combinations are the standard of care in cancer treatment. Identifying effective cancer drug combinations has become more challenging because of the increasing number of drugs. However, a substantial number of cancer drugs stumble at Phase III clinical trials despite exhibiting favourable efficacy in the earlier Phase. Methods: We analysed recent Phase II cancer trials comprising 2165 response rates to uncover trends in cancer therapies and used a null model of non-interacting agents to infer synergistic and antagonistic drug combinations. We compared our latest efficacy dataset with a previous dataset to assess the progress of cancer therapy. Results: Targeted therapies reach higher response rates when used in combination with cytotoxic drugs. We identify four synergistic and 10 antagonistic combinations based on the observed and expected response rates. We demonstrate that recent targeted agents have not significantly increased the response rates. Conclusions: We conclude that either we are not making progress or response rate measured by tumour shrinkage is not a reliable surrogate endpoint for the targeted agents.
Insights
Identifying effective cancer drug combinations is crucial. Analysis of Phase II trials reveals targeted therapies combined with cytotoxic drugs show higher response rates, but overall progress in cancer therapy remains uncertain.
Area of Science:
- Oncology
- Clinical Trials
- Pharmacology
Background:
- Drug combinations are standard cancer treatment.
- Identifying effective combinations is increasingly complex.
- Many drugs fail Phase III trials despite early promise.
Purpose of the Study:
- Analyze Phase II cancer trials to identify trends.
- Infer synergistic and antagonistic drug combinations.
- Assess progress in cancer therapy efficacy.
Main Methods:
- Analyzed 2165 response rates from recent Phase II trials.
- Employed a null model to distinguish drug interactions.
- Compared current efficacy data with historical data.
Main Results:
- Targeted therapies combined with cytotoxic drugs yield higher response rates.
- Identified 4 synergistic and 10 antagonistic drug combinations.
- Recent targeted agents did not significantly improve response rates.
Conclusions:
- Cancer therapy progress is questionable based on response rates.
- Tumor shrinkage may not be a reliable surrogate endpoint for targeted agents.
Related Concept Videos
Clinical Trials: Overview
Clinical Trials
There are four phases in a clinical trial. A phase one...
Targeted Cancer Therapies
There are several types of targeted therapies against...
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...
Drug Administration and Therapy Phases: Overview
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
Treatment Resistant Cancers

