Gene therapy for overactive bladder: A review of bk-channel α-subunit gene transfer

Karl Erik Andersson, George Joseph Christ, Kelvin P. Davies, Eric S. Rovner, Arnold Melman

Research output: Contribution to journalReview articlepeer-review

Abstract

A need exists for local (ie, bladder-specific) interventions to treat overactive bladder (OAB) with low risk of unwanted postprocedural outcomes. Gene therapy targeted to leverage endogenous physiology in bladder cells may assist in restoring normal cell and organ function. Herein, we review the potential promise of gene therapy for treating OAB, focusing on gene transfer of URO-902, a non-viral naked plasmid DNA expressing the big potassium (BK) channel. We searched PubMed for articles concerning functional aspects of the BK channel and its potential use for gene transfer as local OAB treatment. Results from preclinical, phase 1, and phase 2 studies of URO-902 for erectile dysfunction and phase 1 studies of URO-902 for OAB are included. The BK channel has been extensively studied; however, URO-902 is the first gene therapy used in clinical trials directed toward treating OAB via the BK channel. In both URO-902 studies, there were no serious adverse events considered treatment related and no adverse events leading to early withdrawal. Both studies included secondary efficacy endpoints with promising results suggesting improvement in OAB symptoms, and quality of life, with use of URO-902 versus placebo. Gene therapy involving the BK channel, such as gene transfer with URO-902, has demonstrated promising safety and efficacy results in women with OAB. Findings warrant further investigation of the use of URO-902 for OAB treatment.

Original languageEnglish (US)
Pages (from-to)589-599
Number of pages11
JournalTherapeutics and Clinical Risk Management
Volume17
DOIs
StatePublished - 2021

Keywords

  • Big potassium channel
  • Gene expression
  • Ion channels
  • Urinary bladder

ASJC Scopus subject areas

  • Safety Research
  • Pharmacology, Toxicology and Pharmaceutics(all)
  • Pharmacology (medical)
  • Chemical Health and Safety

Fingerprint

Dive into the research topics of 'Gene therapy for overactive bladder: A review of bk-channel α-subunit gene transfer'. Together they form a unique fingerprint.

Cite this