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The following is a summary of “Phase 2 Trial of Topical Application of the Hedgehog Inhibitor Patidegib in Patients With Gorlin Syndrome,” published in the November 2024 issue of Dermatology by Lear et al. 

Patients with Gorlin syndrome (GS) experience various phenotypic abnormalities due to overactive hedgehog (HH) signaling, often caused by a PTCH1 gene mutation. A topical gel containing a cyclopamine-derived molecule was developed to target basal cell carcinomas (BCCs) locally, aiming to reduce the risk of systemic side effects seen with oral HH inhibitors (HHi).  

Researchers conducted a retrospective study to evaluate Patidegib Topical Gel (2% or 4%) could achieve sufficient local anti-BCC efficacy without reaching systemic concentrations that would cause adverse effects typical of oral HHi treatment.  

They assessed the clinical and molecular efficacy and adverse effects of 6 months of twice-daily application of Patidegib Topical Gel application to the face and treatment-targeted surgically eligible BCCs at 2 UK sites.   

The results showed Patidegib Topical Gel reduced the development of new surgically eligible BCCs and lowered HH signaling levels. This effect was observed with minimal adverse effects.  

Investigators concluded the Patidegib Topical Gel demonstrated potential for reducing  BCCs and warranted further clinical development. 

Source: academic.oup.com/bjd/advance-article-abstract/doi/10.1093/bjd/ljae444/7900902