Abstract
Rhabdomyosarcoma (RMS) is the most frequent pediatric soft-tissue tumor accounting for about 7% of childhood malignancies. Multimodal therapy is the standard treatment for individuals with RMS but generally fails to cure high-risk group patients and can result in long-term side effects. Therefore, understanding the mechanisms driving RMS might help to find new candidate targets for more specific and effective therapeutic modalities. One of the molecular machineries which is often deregulated in cancer and specifically involved in tumorigenesis of RMS, is Hedgehog (Hh) signaling. There is increasing evidence that targeting this developmental pathway may hold promise in future treatment strategies for RMS. In this review, we discuss the contribution of the Hh pathway in RMS, the challenges of inhibiting this embryonic signaling in children with an update on recent preclinical data and ongoing clinical trials.
Keywords: Pediatric cancers, rhabdomyosarcoma, developmental pathways, hedgehog signaling, mouse models, targeted therapy.
Current Drug Targets
Title:Interfering with Hedgehog Pathway: New Avenues for Targeted Therapy in Rhabdomyosarcoma
Volume: 17 Issue: 11
Author(s): Gabriele Manzella and Beat W. Schäfer
Affiliation:
Keywords: Pediatric cancers, rhabdomyosarcoma, developmental pathways, hedgehog signaling, mouse models, targeted therapy.
Abstract: Rhabdomyosarcoma (RMS) is the most frequent pediatric soft-tissue tumor accounting for about 7% of childhood malignancies. Multimodal therapy is the standard treatment for individuals with RMS but generally fails to cure high-risk group patients and can result in long-term side effects. Therefore, understanding the mechanisms driving RMS might help to find new candidate targets for more specific and effective therapeutic modalities. One of the molecular machineries which is often deregulated in cancer and specifically involved in tumorigenesis of RMS, is Hedgehog (Hh) signaling. There is increasing evidence that targeting this developmental pathway may hold promise in future treatment strategies for RMS. In this review, we discuss the contribution of the Hh pathway in RMS, the challenges of inhibiting this embryonic signaling in children with an update on recent preclinical data and ongoing clinical trials.
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Cite this article as:
Manzella Gabriele and Schäfer W. Beat, Interfering with Hedgehog Pathway: New Avenues for Targeted Therapy in Rhabdomyosarcoma, Current Drug Targets 2016; 17 (11) . https://dx.doi.org/10.2174/1389450116666150505122604
DOI https://dx.doi.org/10.2174/1389450116666150505122604 |
Print ISSN 1389-4501 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-5592 |
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