Manzella Gabriele, Schäfer Beat W
Department of Oncology, University Children's Hospital, Steinwiesstrasse-75, 8032, Zurich, Switzerland.
Curr Drug Targets. 2016;17(11):1228-34. doi: 10.2174/1389450116666150505122604.
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.
横纹肌肉瘤（RMS）是最常见的小儿软组织肿瘤，约占儿童恶性肿瘤的7%。多模式疗法是RMS患者的标准治疗方法，但通常无法治愈高危组患者，并且可能导致长期副作用。因此，了解驱动RMS的机制可能有助于找到新的候选靶点，以实现更特异且有效的治疗方式。在癌症中常失调且特别参与RMS肿瘤发生的分子机制之一是Hedgehog（Hh）信号通路。越来越多的证据表明，靶向这一发育途径可能在RMS未来的治疗策略中具有前景。在本综述中，我们讨论了Hh通路在RMS中的作用、在儿童中抑制这种胚胎信号通路的挑战，并更新了近期的临床前数据和正在进行的临床试验情况。
