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Updated: Aug 17, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
[Molecular target structures in oncology]
1Klinische Kooperationsgruppe Leukämie, Medizinische Klinik III, Klinikum Grosshadern der Ludwig-Maximilians-Universität München und GSF-Hämatologikum, Marchioninistrasse 15, 81377, München, Germany. karsten.spiekermann@med.uni-muenchen.de
Abstract:
Substantial progress has been made in recent years in understanding the molecular pathogenesis of malignant disorders, especially in identification of molecular targets for therapeutic interventions ("targeted therapies"). An important group of therapeutical targets are signaling cascades, e.g. protein tyrosine kinases (PTK) that are activated by mutations, translocations or overexpression. Small molecule inhibitors that compete with ATP and inhibit kinase activity have produced clinical impressive responses in chronic myeloid leukemia, gastrointestinal stroma tumors and non-small cell lung cancer. Another group of cellular targets is represented by tumor-selective cell surface proteins that can serve as target structures for antibodies. Therapeutical concepts using monoclonal antibodies have substantially improved response rates in patients with malignant lymphomas and are currently evaluated in other types of cancer. The definition of molecular target structures critical for the malignant phenotype is driving a new era of integrated diagnostics and therapeutics in the field of oncology.
Insights
Recent advances in oncology focus on molecular targets for "targeted therapies." Inhibitors of protein tyrosine kinases (PTKs) and monoclonal antibodies show promise in treating various cancers.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Context:
- Understanding the molecular basis of cancer has led to the development of targeted therapies.
- Signaling cascades, such as protein tyrosine kinases (PTKs), are frequently dysregulated in malignant disorders.
- Tumor-specific cell surface proteins offer targets for antibody-based therapies.
Purpose:
- To review the progress in identifying and targeting molecular pathways in cancer.
- To highlight the therapeutic potential of small molecule inhibitors and monoclonal antibodies.
- To emphasize the integration of diagnostics and therapeutics in oncology.
Summary:
- Targeted therapies exploit molecular alterations driving cancer, including activated protein tyrosine kinases (PTKs) and tumor-specific cell surface proteins.
- Small molecule inhibitors targeting PTKs have shown significant clinical responses in cancers like chronic myeloid leukemia and non-small cell lung cancer.
- Monoclonal antibodies targeting cell surface proteins have improved outcomes in lymphomas and are being explored for other malignancies.
Impact:
- Molecularly targeted therapies represent a paradigm shift in cancer treatment, moving towards personalized medicine.
- The identification of critical molecular targets is crucial for developing effective integrated diagnostic and therapeutic strategies.
- This approach holds the potential to significantly improve patient response rates and outcomes across various cancer types.
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