Dr Steve
09-19-2009, 07:09 AM
Well, I've always said that genetic therapy will be the "cure" for many illnesses in the future...cancer (shut off the growth gene), cardiac disease (turn on the good cholesterol gene), maybe even aging (turn on the cleanup genes and shut off the programmed death genes), but I always couched this in "we'll never see it in our lifetime". It was always something that was tantalizingly out of reach.
With all these momentous and important diseases, who knew one of the first things that may hit the shelves in gene therapy would be for Limpcock Syndrome?
I was searching "novel treatments for erectile dysfunction", always wanting to stay on top of things for the Weird Medicine listener (and 202/197 fan in general) when I came across this article:
Urol Clin North Am. 2007 Nov;34(4):619-30, viii.
Gene therapy for male erectile dysfunction.
Melman A.
Department of Urology, Albert Einstein College of Medicine, Bronx, NY, USA.
ameman@aecom.yu.edu
The potential clinical advantages of a gene transfer therapy-based approach to
treatment of genitourinary smooth muscle-based disorders are several: potential
single therapy for restoration of normal bladder or erectile function;
eliminating the need for daily medication; use in combination with other
therapies to reduce dose requirements and side effects; and development of
mechanism-based, patient-specific treatment approaches. With the safe
administration of hMax-K to men with erectile dysfunction in the first human
phase 1 trial and the initiation of the phase 1 trial of hMaxi-K for patients who
have detrusor overactivity, we have entered an exciting new era in the
development of safe enduring therapies for genitourinary disorders.
Usually, you have to use a virus to insert foreign DNA into your genome...these horny bastards figured out a way to do it with "naked" DNA:
Int J Impot Res. 2006 Jan-Feb;18(1):19-25.
Gene transfer for the therapy of erectile dysfunction: progress in the 21st
century.
Melman A.
Department of Urology, Montefiore Medical Center/Albert Einstein College of
Medicine, Bronx, NY 10467-2400, USA. amelman@montefiore.org
Gene transfer represents the next potential era of advancement in medicine for
the prevention of the effects of aging or for treatment of genetic or acquired
disease. For gene transfer to be a practical successor to today's oral and
minimally invasive therapies, the product must have a high safety profile and a
long duration of effectiveness to correct the need for on-demand administration.
Several types of vectors have been used in preclinicals studies, but because of
widely publicized adverse events, progress using viral vectors in humans has been
limited. There is a current phase I human trial using naked DNA as the vector
with the maxi-K gene to modify cellular contractility. Preliminary results in the
safety trial thus far have shown no treatment-related adverse events, no transfer
to the semen, and the possibility of efficacy in one participant.
So I looked to see what I could find in the more recent literature to see what I could find on this...are we nearly in the era of practical genetic therapy?
Curr Opin Urol. 2009 Sep 8. [Epub ahead of print]
Gene transfer for erectile dysfunction: will this novel therapy be accepted by
urologists?
Melman A, Rojas L, Christ G.
aDepartment of Urology, Albert Einstein College of Medicine, Bronx, New York, USA
bDepartment of Urology and Physiology and Pharmacology, Wake Forest Institute for
Regenerative Medicine, Winston Salem, North Carolina, USA.
PURPOSE OF REVIEW: The purpose of this review is to update the results of the
only phase 1 erectile dysfunction gene transfer trial and based upon those
results present the outcome of a web-based survey that studied whether or not
knowledgeable in the field urologists would use gene transfer in their patients
once approved for use by the US Food and Drug Administration. RECENT FINDINGS:
The results of the clinical trials showed no transfer-related serious adverse
events. The response to 10 questions of a web-based survey indicates that gene
transfer as a first or second-line therapy for practicing urologists would be
well accepted. SUMMARY: Practicing, experienced urologists, after the US Food and
Drug Administration approval, are willing to employ gene transfer therapies in
their patients, be it men who have failed or dissatisfied with other treatments
or as shown in up to one-third of men as a first therapy. That outcome portends
for rapid adaptation and active participation into the medical practice for
maxi-K or other specific gene transfer, stem cell, or combinations that will be
developed in the future.
Manufacturers don't do this kind of study (physician acceptance surveys) unless they are READY TO BRING THE DRUG TO MARKET.
Stay tuned.
I do think it's funny that since most of these researchers are MEN, that one of the first gene treatments in the history of the world is going to be for erectile dysfunction. Not that I'm complaining.
Next: a TOPICAL Viagra (i.e., Viagra that you rub on your dick, avoiding most or all of the side effects of Viagra tablets. :clap: :clap: :clap:
With all these momentous and important diseases, who knew one of the first things that may hit the shelves in gene therapy would be for Limpcock Syndrome?
I was searching "novel treatments for erectile dysfunction", always wanting to stay on top of things for the Weird Medicine listener (and 202/197 fan in general) when I came across this article:
Urol Clin North Am. 2007 Nov;34(4):619-30, viii.
Gene therapy for male erectile dysfunction.
Melman A.
Department of Urology, Albert Einstein College of Medicine, Bronx, NY, USA.
ameman@aecom.yu.edu
The potential clinical advantages of a gene transfer therapy-based approach to
treatment of genitourinary smooth muscle-based disorders are several: potential
single therapy for restoration of normal bladder or erectile function;
eliminating the need for daily medication; use in combination with other
therapies to reduce dose requirements and side effects; and development of
mechanism-based, patient-specific treatment approaches. With the safe
administration of hMax-K to men with erectile dysfunction in the first human
phase 1 trial and the initiation of the phase 1 trial of hMaxi-K for patients who
have detrusor overactivity, we have entered an exciting new era in the
development of safe enduring therapies for genitourinary disorders.
Usually, you have to use a virus to insert foreign DNA into your genome...these horny bastards figured out a way to do it with "naked" DNA:
Int J Impot Res. 2006 Jan-Feb;18(1):19-25.
Gene transfer for the therapy of erectile dysfunction: progress in the 21st
century.
Melman A.
Department of Urology, Montefiore Medical Center/Albert Einstein College of
Medicine, Bronx, NY 10467-2400, USA. amelman@montefiore.org
Gene transfer represents the next potential era of advancement in medicine for
the prevention of the effects of aging or for treatment of genetic or acquired
disease. For gene transfer to be a practical successor to today's oral and
minimally invasive therapies, the product must have a high safety profile and a
long duration of effectiveness to correct the need for on-demand administration.
Several types of vectors have been used in preclinicals studies, but because of
widely publicized adverse events, progress using viral vectors in humans has been
limited. There is a current phase I human trial using naked DNA as the vector
with the maxi-K gene to modify cellular contractility. Preliminary results in the
safety trial thus far have shown no treatment-related adverse events, no transfer
to the semen, and the possibility of efficacy in one participant.
So I looked to see what I could find in the more recent literature to see what I could find on this...are we nearly in the era of practical genetic therapy?
Curr Opin Urol. 2009 Sep 8. [Epub ahead of print]
Gene transfer for erectile dysfunction: will this novel therapy be accepted by
urologists?
Melman A, Rojas L, Christ G.
aDepartment of Urology, Albert Einstein College of Medicine, Bronx, New York, USA
bDepartment of Urology and Physiology and Pharmacology, Wake Forest Institute for
Regenerative Medicine, Winston Salem, North Carolina, USA.
PURPOSE OF REVIEW: The purpose of this review is to update the results of the
only phase 1 erectile dysfunction gene transfer trial and based upon those
results present the outcome of a web-based survey that studied whether or not
knowledgeable in the field urologists would use gene transfer in their patients
once approved for use by the US Food and Drug Administration. RECENT FINDINGS:
The results of the clinical trials showed no transfer-related serious adverse
events. The response to 10 questions of a web-based survey indicates that gene
transfer as a first or second-line therapy for practicing urologists would be
well accepted. SUMMARY: Practicing, experienced urologists, after the US Food and
Drug Administration approval, are willing to employ gene transfer therapies in
their patients, be it men who have failed or dissatisfied with other treatments
or as shown in up to one-third of men as a first therapy. That outcome portends
for rapid adaptation and active participation into the medical practice for
maxi-K or other specific gene transfer, stem cell, or combinations that will be
developed in the future.
Manufacturers don't do this kind of study (physician acceptance surveys) unless they are READY TO BRING THE DRUG TO MARKET.
Stay tuned.
I do think it's funny that since most of these researchers are MEN, that one of the first gene treatments in the history of the world is going to be for erectile dysfunction. Not that I'm complaining.
Next: a TOPICAL Viagra (i.e., Viagra that you rub on your dick, avoiding most or all of the side effects of Viagra tablets. :clap: :clap: :clap: