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Exportin(g) precision oncology beyond genomics with XPO1 inhibition

Mohamed A Gouda1, Vivek Subbiah2

  • 1Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.

Med (New York, N.Y.)
|November 12, 2022
PubMed

Insights

Researchers found abnormal XPO1 activity in pediatric Wilms tumor (WT) and malignant rhabdoid tumor (MRT). Early tests show inhibiting XPO1 may be a new treatment strategy for these rare childhood cancers.

Area of Science:

  • Oncology
  • Molecular Biology
  • Pediatric Medicine

Background:

  • Pediatric cancers like Wilms tumor (WT) and malignant rhabdoid tumor (MRT) require novel therapeutic targets.
  • Identifying druggable targets is crucial for advancing treatment options in pediatric oncology.

Purpose of the Study:

  • To identify and validate novel therapeutic targets for pediatric WT and MRT.
  • To investigate the potential of targeting XPO1 (Exportin 1) in these specific pediatric malignancies.

Main Methods:

  • Utilized public genomic and proteomic databases to analyze gene expression and protein activity.
  • Performed in silico analysis to identify aberrant molecular pathways.
  • Conducted early-stage in vitro/in vivo studies to assess the efficacy of XPO1 inhibition.

Main Results:

  • Aberrant XPO1 activity was identified and validated in patient samples of WT and MRT.
  • Demonstrated a proof-of-concept for XPO1 inhibition as a therapeutic strategy.
  • Showcased a bench-to-bedside translation pathway for XPO1-targeted therapies.

Conclusions:

  • XPO1 is a validated druggable target in pediatric Wilms tumor and malignant rhabdoid tumor.
  • XPO1 inhibition represents a promising therapeutic avenue for treating these pediatric cancers.
  • Further clinical investigation of XPO1 inhibitors is warranted for pediatric WT and MRT patients.

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