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Expert Opin Emerg Drugs. 2014 Sep;19(3):353-65. doi: 10.1517/14728214.2014.914171. Epub 2014 Apr 29.

Emerging drugs and combination strategies for basal cell carcinoma.

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University Hospital Zurich, Department of Dermatology , Gloriastrasse 31, CH-8091 Zurich , Switzerland.



Basal cell carcinoma (BCC) is a malignancy that is driven by an activated Hedgehog (Hh) pathway. Smoothened inhibitors are a new promising treatment option for patients with locally advanced or metastatic BCC or basal cell nevus syndrome. But long-term data are still limited, the optimal treatment duration is not yet defined and there are already documented cases with acquired resistance.


Treatment modalities with Hh inhibitors, side effects and potential pharmacological combination options are discussed. The current literature, including PubMed, Cochrane database and registered trials on, was searched.


BCCs typically regress during therapy with Hh inhibitors. Muscle toxicity, dysgeusia and hair loss can be considered as on target adverse reactions. Muscle toxicity is the dose-limiting toxicity of sonidegib. It was not seen with vismodegib because of its high binding to plasma protein α-1-acid glycoprotein. Sonidegib is different and shows a clear dose-toxicity relationship, which allows to address the question of whether there is a dose dependency of regression rate, cure rate and progression-free survival. In addition, basic research has offered strategies to enhance efficacy by the combination with other molecules, such as EGFR inhibitors, MEK inhibitors or immunotherapy.


Hedgehog inhibitors; adverse events; basal cell carcinoma; combination therapy; resistance; side effects; smoothened inhibitors; sonidegib; toxic myopathy

[Indexed for MEDLINE]

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