Targeted next-generation sequencing of matched localized and metastatic primary high-risk SCCs identifies driver and co-occurring mutations and novel therapeutic targets

J Dermatol Sci. 2020 Jul;99(1):30-43. doi: 10.1016/j.jdermsci.2020.05.007. Epub 2020 May 29.

Abstract

Background: Cutaneous squamous cell carcinoma (SCC) is the second most common type of skin cancer and is responsible for over one million cases annually. While only 3-5 % of SCCs metastasize, those that do are associated with significant morbidity and mortality. Using gene mutations to help predict metastasis and select therapeutics is still being explored.

Objective: To present novel data from targeted sequencing of 20 case-matched localized and metastatic high-risk SCCs.

Methods: A cancer-associated gene panel of 76 genes was run from formalin-fixed paraffin-embedded samples of 20 case-matched localized (10) and metastatic (10) high-risk SCCs (Vela Diagnostics).

Results: Using spatial clustering analysis, primary driver mutations were identified asEGFR in localized SCC and CDH1 in metastatic SCC. ERBB4 and STK11 were found to be significant co-occurring mutations in localized SCC. Pathway analyses showed the RTK/RAS, TP53, TGF-b, NOTCH1, PI3K, and cell cycle pathways to be highly relevant in all high-risk SCCs with the Wnt pathway enhanced in metastatic SCC only.

Conclusions: This study compared gene mutations between localized and metastatic SCC with the intent of identifying key differences and new potential targeted treatment options. To our knowledge, the co-occurrence ofERBB4 and STK11 mutations has not been previously reported. Targeted inhibition of CDH1 and the Wnt pathway should be further explored in metastatic SCC.

Keywords: Cutaneous squamous cell carcinoma; Metastasis; Mutations; Next-generation sequencing; Targeted-therapy.

MeSH terms

  • AMP-Activated Protein Kinase Kinases
  • Adult
  • Aged
  • Aged, 80 and over
  • Antigens, CD / genetics
  • Antineoplastic Agents, Alkylating / pharmacology
  • Antineoplastic Agents, Alkylating / therapeutic use*
  • Biomarkers, Tumor / antagonists & inhibitors
  • Biomarkers, Tumor / genetics*
  • Cadherins / antagonists & inhibitors
  • Cadherins / genetics
  • Carcinogenesis / drug effects
  • Carcinogenesis / genetics
  • Carcinoma, Squamous Cell / drug therapy
  • Carcinoma, Squamous Cell / genetics*
  • Carcinoma, Squamous Cell / secondary
  • Cohort Studies
  • DNA Mutational Analysis
  • Female
  • High-Throughput Nucleotide Sequencing
  • Humans
  • Male
  • Middle Aged
  • Molecular Targeted Therapy / methods
  • Mutation
  • Protein Serine-Threonine Kinases / genetics
  • Receptor, ErbB-4 / genetics
  • Skin / pathology
  • Skin Neoplasms / drug therapy
  • Skin Neoplasms / genetics*
  • Skin Neoplasms / pathology
  • Wnt Signaling Pathway / drug effects
  • Wnt Signaling Pathway / genetics

Substances

  • Antigens, CD
  • Antineoplastic Agents, Alkylating
  • Biomarkers, Tumor
  • CDH1 protein, human
  • Cadherins
  • ERBB4 protein, human
  • Receptor, ErbB-4
  • Protein Serine-Threonine Kinases
  • STK11 protein, human
  • AMP-Activated Protein Kinase Kinases