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Pazopanib elicits remarkable response in metastatic porocarcinoma: a functional precision medicine approach
Sharon Pei Yi Chan1, Chen Ee Low2, Chun En Yau2
1Cancer Science Institute of Singapore, National University of Singapore, Singapore.
Abstract:
Metastatic porocarcinomas (PCs) are vanishingly rare, highly aggressive skin adnexal tumors with mortality rates exceeding 70%. Their rarity has precluded the understanding of their disease pathogenesis, let alone the conduct of clinical trials to evaluate treatment strategies. There are no effective agents for unresectable PCs. Here, we successfully demonstrate how functional precision medicine was implemented in the clinic for a metastatic PC with no known systemic treatment options. Comprehensive genomic profiling of the tumor specimen did not yield any actionable genomic aberrations. However, ex vivo drug testing predicted pazopanib efficacy, and indeed, administration of pazopanib elicited remarkable clinicoradiological response. Pazopanib and its class of drugs should be evaluated for efficacy in other cases of PC, and the rationale for efficacy should be determined when PC tumor models become available. A functional precision medicine approach could be useful to derive effective treatment options for rare cancers.
Insights
Metastatic porocarcinomas are rare, aggressive skin cancers. Functional precision medicine identified pazopanib as an effective treatment when standard options failed, showing promise for rare cancer therapies.
Area of Science:
- Oncology
- Dermatology
- Precision Medicine
Background:
- Metastatic porocarcinomas (PCs) are rare, aggressive skin tumors with high mortality.
- Effective systemic treatments for unresectable PCs are lacking due to their rarity.
- Understanding PC pathogenesis and conducting clinical trials are challenging.
Purpose of the Study:
- To demonstrate the clinical implementation of functional precision medicine for metastatic PC.
- To identify effective treatment options for metastatic PCs lacking actionable genomic alterations.
- To evaluate the efficacy of pazopanib in a patient with metastatic PC.
Main Methods:
- Comprehensive genomic profiling of the tumor specimen.
- Ex vivo drug sensitivity testing.
- Clinical administration of predicted effective therapy (pazopanib).
Main Results:
- Genomic profiling revealed no actionable aberrations.
- Ex vivo testing predicted pazopanib efficacy.
- Pazopanib administration resulted in a significant clinicoradiological response.
Conclusions:
- Functional precision medicine can identify effective treatments for rare cancers like PC.
- Pazopanib shows potential efficacy in metastatic PCs, warranting further investigation.
- Development of PC tumor models is crucial for understanding treatment rationale.
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