Zusammenfassung
Die gezielte, auf dem Verständnis der molekularen Grundlagen der Erkrankung beruhende Therapie von Malignomen in der Hämatologie und Onkologie ist beispielhaft für eine wesentliche Weiterentwicklung der modernen Medizin. Durch Grundlagenwissenschaften erzielte Erkenntnisse—wie etwa die Funktion von Fusionsproteinen als Folge von Chromosomentranslokationen, die Identifikation der für das Überleben der Tumorzelle erforderlichen molekularen Strukturen oder tumorspezifischer Oberflächenmoleküle—ermöglichen erstmals eine spezifische Behandlung der Tumorerkrankung, im Gegensatz zur klassischen Chemotherapie, die primär die ubiquitären Stoffwechselwege der Nukleinsäuresynthese und Zellteilung beeinflussen. Neben einer gesteigerten Wirksamkeit ist langfristig bei diesen Ansätzen auch ein günstigeres Nebenwirkungsprofil zu erwarten. Für diese Therapieansätze kommen neben den „kleinen Molekülen“ v. a. auch monoklonale Antikörper zum Einsatz. Aus dem breiten Spektrum an Neuerungen sollen hier einige interessante Therapiestrategien, die mittlerweile Eingang in die Klinik gefunden haben, vorgestellt werden.
Abstract
Targeted approaches to treat malignant diseases in hematology and oncology based on the molecular basis of the disease represent a major breakthrough in modern medicine. Knowledge acquired in basic sciences such as functional understanding of products generated by chromosomal translocations, definition of surface molecules or molecular requirements of tumor-cell survival allow to specifically aim at the cause of or at a requirement for malignancy. This is in sharp contrast to conventional chemotherapy which mainly influences the ubiquitous pathways of nucleic acid metabolism and cell division. In addition to superior efficacy of these approaches one should—on the long run—expect a superior profile of side effects compared to standard regimens. These “designer-approaches” are mainly based on small molecules or monoclonal antibodies. Out of the broad spectrum of current concepts we would like to summarize some of the strategies that have already found their way from bench to bedside.
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Beck, C., Kneba, M. Designermedikamente in der Tumortherapie. Internist 45 (Suppl 1), S38–S47 (2004). https://doi.org/10.1007/s00108-004-1223-6
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DOI: https://doi.org/10.1007/s00108-004-1223-6