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An Orthotopic Bladder Cancer Model for Gene Delivery Studies
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An Orthotopic Bladder Cancer Model for Gene Delivery Studies

Published on: December 1, 2013

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Cationic amphiphilic drugs as potential anticancer therapy for bladder cancer

Geertje van der Horst1, Arjanneke F van de Merbel1, Eline Ruigrok1

  • 1Department of Urology, Leiden University Medical Center, The Netherlands.

Molecular Oncology
|September 8, 2020
PubMed

Insights

Repurposed drug penfluridol shows promise for bladder cancer. This cationic amphiphilic drug effectively reduced tumor growth in mice and patient-derived tissues, offering a potential new therapy for urothelial carcinoma of the bladder.

Area of Science:

  • Oncology
  • Pharmacology

Background:

  • Muscle-invasive and high-risk non-muscle-invasive urothelial carcinoma of the bladder (UCB) present an unmet clinical need for effective therapies.
  • Drug repositioning offers a faster route to introduce novel anticancer treatments due to established safety and dosing profiles of existing drugs.

Purpose of the Study:

  • To investigate the potential of cationic amphiphilic drugs (CADs) as a therapeutic strategy for UCB.
  • To evaluate the efficacy of the CAD penfluridol in preclinical models of UCB.

Main Methods:

  • In vitro assessment of CADs on human UCB cell lines, observing effects on viability and lysosomal integrity.
  • In vivo evaluation of intravesical penfluridol in an orthotopic mouse xenograft model of human UCB.
  • Ex vivo treatment of patient-derived UCB tissue cultures with penfluridol.

Main Results:

  • CADs demonstrated dose-dependent cytotoxicity against UCB cell lines and induced lysosomal leakage.
  • Intravesical penfluridol significantly inhibited tumor growth and metastasis in a mouse model.
  • Penfluridol treatment resulted in significant partial or complete antitumor responses in 97% of patient-derived ex vivo UCB tissues.

Conclusions:

  • Penfluridol exhibits significant antitumor activity in preclinical models of UCB.
  • The drug warrants further clinical investigation as a potential treatment for bladder cancer.
  • Drug repositioning of CADs is a viable strategy for developing new urothelial carcinoma therapies.
Keywords:
bladder cancercationic amphiphilic drugsex vivo culturepenfluridolpreclinical in vivo model

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