Klin Padiatr 2013; 225(03): 115-119
DOI: 10.1055/s-0032-1331762
Review
© Georg Thieme Verlag KG Stuttgart · New York

Molecular Targeted Therapies for Rhabdomyosarcoma: Focus on Hedgehog and Apoptosis Signaling

Molekular zielgerichtete Therapieansätze beim Rhabdomyosarkom: Fokus auf dem Hedgehog- und Apoptose-Signalweg
S. Fulda
1   Institute for Experimental Cancer Research in Pediatrics, Goethe-University, Frankfurt a. Main, Germany
› Author Affiliations
Further Information

Publication History

Publication Date:
26 April 2013 (online)

Abstract

Dysfunction of cell death and proliferation pathways can contribute to rhabdomyosarcomagenesis, tumor progression and treatment resistance. Therefore, the identification of key signaling hubs and molecules that govern the decision between life and death of a cancer cell is expected to open new perspectives for drug discovery. For example, programmed cell death pathways can be engaged in rhabdomyosarcoma (RMS) cells by recombinant soluble proteins, monoclonal antibodies or small-molecule inhibitors. In addition, the hedgehog (Hh) cascade is often aberrantly activated in RMS and represents a promising target for therapeutic intervention. The development of molecular targeted cancer therapeutics will likely lead to more effective treatment options for patients with RMS.

Zusammenfassung

Die gestörte Funktion von Zelltod- und Wachs­tumssignalwegen kann zur Entstehung, Progression und Therapieresistenz des Rhabdomyosarkoms (RMS) beitragen. Daher wird erwartet, dass die Identifizierung von Schlüsselpunkten und -molekülen, welche die Ent­scheidung zwischen Überleben und Absterben einer Krebszelle bestimmen, neuartige Perspektiven für die Medikamentenentwicklung eröffnet. Beispielsweise können Zelltodsignalwege durch die Verwendung von rekombinanten Liganden, monoklonalen Antikörpern oder niedermolekularen Inhibitoren aktiviert werden. Darüber hinaus gilt der Hedgehog-Signalweg, der häufig beim RMS aberrant aktiviert ist, als eine vielversprechende Zielstruktur für therapeutische Interventionen. Die Entwicklung molekular zielgerichteter Therapieansätze wird voraussichtlich zu besser wirksamen Therapieoptionen für Patienten mit RMS führen.

 
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