YAP1 is essential for malignant mesothelioma tumor maintenance
Loreley Calvet1, Odette Dos-Santos2, Emmanuel Spanakis3
1Department of Oncology, In Vivo Pharmacology, Sanofi Research Center, Vitry-sur-Seine, France. loreley.calvet@sanofi.com.
Abstract:
Malignant pleural mesothelioma, a tumor arising from the membrane covering the lungs and the inner side of the ribs, is a cancer in which genetic alterations of genes encoding proteins that act on or are part of the Hippo-YAP1 signaling pathway are frequent. Dysfunctional Hippo signaling may result in aberrant activation of the transcriptional coactivator protein YAP1, which binds to and activates transcription factors of the TEAD family. Recent studies have associated elevated YAP1 protein activity with a poor prognosis of malignant mesothelioma and its resistance to current therapies, but its role in tumor maintenance is unclear. In this study, we investigate the dependence of malignant mesothelioma on YAP1 signaling to maintain fully established tumors in vivo. We show that downregulation of YAP1 in a dysfunctional Hippo genetic background results in the inhibition of YAP1/TEAD-dependent gene expression, the induction of apoptosis, and the inhibition of tumor cell growth in vitro. The conditional downregulation of YAP1 in established tumor xenografts leads to the inhibition of YAP1-dependent gene transcription and eventually tumor regression. This effect is only seen in the YAP1-activated MSTO-211H mesothelioma xenograft model, but not in the Hippo-independent HCT116 colon cancer xenograft model. Our data demonstrate that, in the context of a Hippo pathway mutated background, YAP1 activity alone is enough to maintain the growth of established tumors in vivo, thus validating the concept of inhibiting the activated YAP1-TEAD complex for the treatment of malignant pleural mesothelioma patients.
Insights
Targeting the YAP1-TEAD complex inhibits malignant pleural mesothelioma growth. Downregulating YAP1 in established tumors caused regression, validating this as a potential therapeutic strategy for this cancer.
Area of Science:
- Oncology
- Molecular Biology
- Cancer Signaling Pathways
Background:
- Malignant pleural mesothelioma (MPM) frequently involves genetic alterations in the Hippo-YAP1 signaling pathway.
- Aberrant YAP1 activation, due to dysfunctional Hippo signaling, is linked to poor prognosis and therapy resistance in MPM.
- The precise role of YAP1 in maintaining established MPM tumors remains unclear.
Purpose of the Study:
- To investigate the dependence of established malignant pleural mesothelioma tumors on YAP1 signaling in vivo.
- To determine if YAP1 activity is essential for tumor maintenance in a Hippo-mutated background.
Main Methods:
- Investigated the effect of YAP1 downregulation in vitro and in established MPM xenografts (MSTO-211H).
- Utilized a Hippo-independent colon cancer xenograft model (HCT116) for comparison.
- Assessed YAP1/TEAD-dependent gene expression, apoptosis, and tumor growth.
Main Results:
- Downregulation of YAP1 inhibited YAP1/TEAD-dependent gene expression, induced apoptosis, and reduced tumor cell growth in vitro.
- Conditional YAP1 downregulation in established MSTO-211H xenografts led to tumor regression.
- No tumor regression was observed in the Hippo-independent HCT116 xenograft model.
Conclusions:
- YAP1 activity alone is sufficient to maintain established tumors in a Hippo-mutated background.
- Inhibiting the YAP1-TEAD complex is a validated therapeutic strategy for malignant pleural mesothelioma.
- This study supports targeting the YAP1-TEAD complex for MPM treatment.
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