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Updated: Aug 31, 2026

A Method for Screening and Validation of Resistant Mutations Against Kinase Inhibitors
Published on: December 7, 2014
Rational therapeutic intervention in cancer: kinases as drug targets
111-934 Factor Building, University of California, Los Angeles Division of Hematology and Oncology, 10833 Le Conte Avenue, Los Angeles, California 90095-1678, USA. csawyers@mednet.ucla.edu
Abstract:
Landmark clinical studies of new drugs developed to target specific forms of cancer were reported in 2001. Herceptin, a monoclonal antibody against the Her2/neu receptor tyrosine kinase, prolonged the survival of women with Her-2/neu positive metastatic breast cancer, when combined with chemotherapy. STI-571, a small molecule inhibitor of the Bcr-Abl, c-kit and platelet derived growth factor receptor tyrosine kinases, produced dramatic clinical responses in patients with Bcr-Abl positive chronic myeloid leukemia and c-kit positive gastrointestinal stromal tumors. These examples have galvanized the cancer research community to extend kinase-inhibitor therapy to other cancers.
Insights
New cancer drugs targeting specific genetic mutations show promise. Herceptin improved survival for HER2-positive breast cancer, and STI-571 benefited leukemia and GIST patients, spurring further kinase-inhibitor research.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Cancer treatment historically focused on broad chemotherapy.
- Targeted therapies represent a paradigm shift in cancer treatment.
- Specific molecular targets are crucial for effective drug development.
Purpose of the Study:
- To report on landmark clinical studies of novel cancer drugs in 2001.
- To highlight the efficacy of targeted therapies against specific cancer types.
- To assess the impact of these findings on future cancer research.
Main Methods:
- Clinical trials evaluating Herceptin, a monoclonal antibody targeting HER2/neu.
- Clinical trials assessing STI-571, a small molecule kinase inhibitor.
- Analysis of patient survival and clinical response rates in targeted cancer groups.
Main Results:
- Herceptin demonstrated prolonged survival in women with HER2-positive metastatic breast cancer when combined with chemotherapy.
- STI-571 produced significant clinical responses in patients with Bcr-Abl positive chronic myeloid leukemia.
- STI-571 also showed efficacy in patients with c-kit positive gastrointestinal stromal tumors.
Conclusions:
- Targeted therapies like Herceptin and STI-571 offer effective treatment options for specific cancers.
- These successes validate the kinase-inhibitor approach for cancer therapy.
- The findings have stimulated broader research into kinase-inhibitor applications for various cancers.
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