The first human trial for gene transfer therapy for the treatment of erectile dysfunction

Preliminary results

Arnold Melman, Natan Bar-Chama, Andrew McCullough, Kelvin Davies, George Christ

Research output: Contribution to journalArticle

51 Citations (Scopus)

Abstract

Objective: To test the safety of a single intracavernous injection of a plasmid vector (hMaxi-K) that expresses the hSlo gene, that encodes the α-subunit of the Maxi-K channel, for the treatment of erectile dysfunction (ED). Methods: Six men, thus far have fulfilled the entry criteria of the protocol and had gene transfer with hMaxi-K. Three received a dose of 500 μg and three received a dose 1000 μg of the gene product, injected intracavernously as naked DNA. Dosing at 5000 μg and higher is planned. Results: The primary end point of the phase I trial is safety. No drug-related adverse events or significant laboratory changes have occurred after the gene transfer. Moreover, there is no evidence of the gene in semen at the one copy per μg total DNA in any of the participants. Conclusion: Preliminary results indicate that, in a single dose escalation study, ion channel gene transfer with hMaxi-K can be administered safely to men with ED without adverse events.

Original languageEnglish (US)
Pages (from-to)314-318
Number of pages5
JournalEuropean Urology
Volume48
Issue number2
DOIs
StatePublished - Aug 2005

Fingerprint

Erectile Dysfunction
Genetic Therapy
Genes
Therapeutics
Large-Conductance Calcium-Activated Potassium Channels
Safety
DNA
Semen
Drug-Related Side Effects and Adverse Reactions
Ion Channels
Plasmids
Injections

Keywords

  • Erectile dysfunction
  • Gene transfer
  • hMaxi-K
  • Ion channel therapy
  • Maxi-K channel
  • Naked DNA
  • Potassium channels
  • Smooth muscle

ASJC Scopus subject areas

  • Urology

Cite this

The first human trial for gene transfer therapy for the treatment of erectile dysfunction : Preliminary results. / Melman, Arnold; Bar-Chama, Natan; McCullough, Andrew; Davies, Kelvin; Christ, George.

In: European Urology, Vol. 48, No. 2, 08.2005, p. 314-318.

Research output: Contribution to journalArticle

Melman, Arnold ; Bar-Chama, Natan ; McCullough, Andrew ; Davies, Kelvin ; Christ, George. / The first human trial for gene transfer therapy for the treatment of erectile dysfunction : Preliminary results. In: European Urology. 2005 ; Vol. 48, No. 2. pp. 314-318.
@article{79a5f4e12dd8443a8a68d39404761e4a,
title = "The first human trial for gene transfer therapy for the treatment of erectile dysfunction: Preliminary results",
abstract = "Objective: To test the safety of a single intracavernous injection of a plasmid vector (hMaxi-K) that expresses the hSlo gene, that encodes the α-subunit of the Maxi-K channel, for the treatment of erectile dysfunction (ED). Methods: Six men, thus far have fulfilled the entry criteria of the protocol and had gene transfer with hMaxi-K. Three received a dose of 500 μg and three received a dose 1000 μg of the gene product, injected intracavernously as naked DNA. Dosing at 5000 μg and higher is planned. Results: The primary end point of the phase I trial is safety. No drug-related adverse events or significant laboratory changes have occurred after the gene transfer. Moreover, there is no evidence of the gene in semen at the one copy per μg total DNA in any of the participants. Conclusion: Preliminary results indicate that, in a single dose escalation study, ion channel gene transfer with hMaxi-K can be administered safely to men with ED without adverse events.",
keywords = "Erectile dysfunction, Gene transfer, hMaxi-K, Ion channel therapy, Maxi-K channel, Naked DNA, Potassium channels, Smooth muscle",
author = "Arnold Melman and Natan Bar-Chama and Andrew McCullough and Kelvin Davies and George Christ",
year = "2005",
month = "8",
doi = "10.1016/j.eururo.2005.05.005",
language = "English (US)",
volume = "48",
pages = "314--318",
journal = "European Urology",
issn = "0302-2838",
publisher = "Elsevier",
number = "2",

}

TY - JOUR

T1 - The first human trial for gene transfer therapy for the treatment of erectile dysfunction

T2 - Preliminary results

AU - Melman, Arnold

AU - Bar-Chama, Natan

AU - McCullough, Andrew

AU - Davies, Kelvin

AU - Christ, George

PY - 2005/8

Y1 - 2005/8

N2 - Objective: To test the safety of a single intracavernous injection of a plasmid vector (hMaxi-K) that expresses the hSlo gene, that encodes the α-subunit of the Maxi-K channel, for the treatment of erectile dysfunction (ED). Methods: Six men, thus far have fulfilled the entry criteria of the protocol and had gene transfer with hMaxi-K. Three received a dose of 500 μg and three received a dose 1000 μg of the gene product, injected intracavernously as naked DNA. Dosing at 5000 μg and higher is planned. Results: The primary end point of the phase I trial is safety. No drug-related adverse events or significant laboratory changes have occurred after the gene transfer. Moreover, there is no evidence of the gene in semen at the one copy per μg total DNA in any of the participants. Conclusion: Preliminary results indicate that, in a single dose escalation study, ion channel gene transfer with hMaxi-K can be administered safely to men with ED without adverse events.

AB - Objective: To test the safety of a single intracavernous injection of a plasmid vector (hMaxi-K) that expresses the hSlo gene, that encodes the α-subunit of the Maxi-K channel, for the treatment of erectile dysfunction (ED). Methods: Six men, thus far have fulfilled the entry criteria of the protocol and had gene transfer with hMaxi-K. Three received a dose of 500 μg and three received a dose 1000 μg of the gene product, injected intracavernously as naked DNA. Dosing at 5000 μg and higher is planned. Results: The primary end point of the phase I trial is safety. No drug-related adverse events or significant laboratory changes have occurred after the gene transfer. Moreover, there is no evidence of the gene in semen at the one copy per μg total DNA in any of the participants. Conclusion: Preliminary results indicate that, in a single dose escalation study, ion channel gene transfer with hMaxi-K can be administered safely to men with ED without adverse events.

KW - Erectile dysfunction

KW - Gene transfer

KW - hMaxi-K

KW - Ion channel therapy

KW - Maxi-K channel

KW - Naked DNA

KW - Potassium channels

KW - Smooth muscle

UR - http://www.scopus.com/inward/record.url?scp=21844432423&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=21844432423&partnerID=8YFLogxK

U2 - 10.1016/j.eururo.2005.05.005

DO - 10.1016/j.eururo.2005.05.005

M3 - Article

VL - 48

SP - 314

EP - 318

JO - European Urology

JF - European Urology

SN - 0302-2838

IS - 2

ER -