Related Experiment Video

Updated: Feb 12, 2026

Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies
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Published on: September 30, 2016

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Effective anti-programmed death-1 therapy in a SUFU-mutated patient with Gorlin-Goltz syndrome

A Moreira1, M C Kirchberger1, F Toussaint1

  • 1Department of Dermatology, Universitätsklinikum Erlangen, Friedrich-Alexander-University Erlangen-Nuremberg (FAU), 91054, Erlangen, Germany.

The British Journal of Dermatology
|April 1, 2018
PubMed

Insights

A patient with over 50 basal cell carcinomas, resistant to multiple treatments, showed significant improvement with the immunotherapy drug pembrolizumab. This anti-programmed death-1 antibody offered a new therapeutic option for advanced basal cell carcinoma.

Area of Science:

  • Oncology
  • Dermatology
  • Immunotherapy

Background:

  • Basal cell carcinoma (BCC) is the most common human cancer.
  • Advanced BCC can be challenging to treat, especially when resistant to standard therapies.
  • The patient presented with numerous, treatment-refractory basal cell carcinomas.

Observation:

  • A 77-year-old male presented with over 50 basal cell carcinomas on the head and upper trunk.
  • The patient exhibited resistance to multiple treatment modalities, including surgery, imiquimod, retinoids, itraconazole, and vismodegib.

Findings:

  • Off-label administration of the anti-programmed death-1 antibody, pembrolizumab, was initiated.
  • Significant positive response was observed after four infusions of pembrolizumab.
  • Pembrolizumab demonstrated efficacy in a treatment-refractory case of extensive basal cell carcinoma.

Implications:

  • Pembrolizumab represents a potential novel therapeutic strategy for advanced and treatment-resistant basal cell carcinoma.
  • Immunotherapy targeting the PD-1 pathway may overcome resistance mechanisms seen with other treatments.
  • Further investigation into pembrolizumab's role in managing advanced BCC is warranted.

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