Target intelligence / Profile preview

Basal cell carcinoma (BCC)

Target
BCC
Molecular classification
Hedgehog signaling pathway components, Cancer cell type
01

Overview

Basal cell carcinoma (BCC) is a common, typically slow-growing malignancy of the skin that arises from the basal layer of the epidermis [1, 14]. It is the most frequent cancer globally and is strongly associated with cumulative ultraviolet (UV) radiation exposure [13]. The defining molecular characteristic of BCC is the dysregulation of the Hedgehog (Hh) signaling pathway, which is essential for normal embryonic development but becomes inappropriately reactivated in these cells, usually through loss-of-function mutations in the Patched-1 (PTCH1) receptor or gain-of-function mutations in the Smoothened (SMO) protein [4, 8]. Pharmacological treatments for advanced BCC focus on inhibiting this pathway using SMO antagonists such as vismodegib and sonidegib, or utilizing immunotherapy like cemiplimab to enhance the anti-tumor response [2, 5]. While rarely metastatic, BCC can cause significant local tissue destruction if left untreated, making early detection and targeted intervention critical [7, 11].

Other names
Basal cell epitheliomaRodent ulcerUlcus rodensBasalzellenkrebsNon-melanoma skin cancer
02

Mechanism of action

Inhibition of the Hedgehog signaling pathway through Smoothened (SMO) protein antagonism, blockade of the PD-1 immune checkpoint to enhance T-cell response, and activation of local immune responses via TLR7 agonism [2, 4, 7].

03

Biological functions

Cell proliferationSignal transductionCell survivalStem cell differentiation
04

Disease associations

CancerSkin cancer
05

Safety considerations

TeratogenicityMuscle spasmsDysgeusia (taste disturbance)AlopeciaFatigueImmune-related adverse events (irAEs)
06

Interacting drugs

Vismodegib

6 more in the full profile.

07

Biomarkers

Patched-1 (PTCH1) mutationSmoothened (SMO) mutationHedgehog pathway activationProgrammed death-ligand 1 (PD-L1) expressionp53 (TP53) mutationGLI1 expression level

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