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Updated: Apr 24, 2026

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Translational hematology
15th Department of Internal Medicine-Oncology/Hematology, Vienna and Ludwig Boltzmann Institute for Clinical Oncology, Krankenhaus Hietzing, Wolkersbergenstraße 1, 1130, Vienna, Austria, klaus.geissler@wienkav.at.
Abstract:
Translational research is scientific research that helps to make findings from basic science useful for practical applications in the clinic. The successful use of a drug that interferes with the specific molecular pathophysiology of cancer remains the ultimate vision in cancer medicine. Translational research is a multistep process including the discovery of a cytogenetic/molecular aberration as well as the demonstration of its pathophysiological relevance and its druggability by in vitro experiments and in vivo animal models. Information obtained from preclinical research paves the way for clinical trials in which a drug of interest is developed until its clinical application. Modern pathophysiology-oriented anticancer drugs that have been developed by translational research are available for clinical applications since the beginning of this millennium. By using these drugs higher efficacy and lower toxicity could be achieved as compared with previous treatments. In this article, we will present some of the most prominent examples of this translational approach.
Insights
Translational research bridges basic science and clinical cancer medicine by developing targeted drugs. This approach yields more effective cancer treatments with fewer side effects.
Area of Science:
- Oncology
- Translational Medicine
- Drug Development
Background:
- Translational research is key to translating basic science discoveries into clinical cancer treatments.
- The ultimate goal is developing drugs targeting cancer's specific molecular pathophysiology.
- This research involves identifying molecular aberrations, confirming their relevance, and testing drug efficacy.
Purpose of the Study:
- To highlight the importance of translational research in modern cancer medicine.
- To showcase prominent examples of successful translational research in oncology.
- To demonstrate the benefits of pathophysiology-oriented anticancer drugs.
Main Methods:
- Discovery of cytogenetic/molecular aberrations.
- Demonstration of pathophysiological relevance through in vitro experiments.
- Preclinical testing in animal models to inform clinical trials.
- Development of drugs until clinical application.
Main Results:
- Modern pathophysiology-oriented anticancer drugs are now available.
- These drugs offer higher efficacy compared to traditional treatments.
- Associated toxicity is significantly lower with these novel therapies.
Conclusions:
- Translational research has successfully delivered advanced cancer therapies.
- Targeting specific molecular pathways improves treatment outcomes.
- Continued investment in translational research is crucial for future cancer medicine advancements.
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