First HIV Gene Therapy Test Encouraging

[J220]

First HIV Gene Therapy Test Encouraging
 
By THE ASSOCIATED PRESS
Published: November 6, 2006
Filed at 9:15 p.m. ET

WASHINGTON (AP) -- The first test of a potential new gene therapy for HIV -- the virus that causes AIDS -- was encouraging enough for researchers to launch a more extensive trial.

''The goal of this phase I trial was safety and feasibility, and the results established that,'' said lead researcher Dr. Carl June. ''But the results also hint at something much more.''

In addition to showing that the treatment was possible and didn't endanger the patients, the amount of virus in the subjects remained steady or decreased during the study, which involved just five people with chronic HIV infection.

One patient had a sustained decrease in the amount of virus, and immune cells and strength of the immune system increased in four patients during the nine-month study.

However, ''just because this has produced encouraging results in one or two patients doesn't mean it will work for everyone. We have much more work to do,'' said co-author Dr. Bruce Levine.

June and Levine are researchers at the University of Pennsylvania's Abramson Family Cancer Research Institute. Their findings are reported in the online edition of Proceedings of the National Academy of Sciences.

The study team also included researchers from the VIRxSYS Corp. of Gaithersburg, Md., which is involved in developing the new treatment and helped fund the study. Other funding came from the National Institutes of Allergy and Infectious Disease and the Abramson institute.

The researchers removed immune cells from the patients and introduced a virus called a lentivirus into the cells. This change prevents HIV from reproducing and, in the laboratory, has the ability to fight HIV in cells that have not been treated, June explained in a telephone interview.

The idea, he said, was that unlike most HIV medications that have to be taken daily or several times a day, this treatment can be done once and will keep fighting the infection.

This was the first human test to see if it could be done safely, he said. It was done on patients whose HIV infections have resisted treatment.

Now, the team has launched a phase II test that will involve more patients, including some whose HIV is controlled by drugs. In this test the patients will get more than one transfusion of the treated cells. Those on standard drug treatment, following the new therapy, will be asked to interrupt their drugs to see if the infection returns.

''This paper should make quite some noise,'' commented Dr. Martin Haas, a professor at the University of California San Diego School of Medicine.

''I think this is very important work and they have doggedly continued it,'' said Haas, who was not part of the research team. ''I think they have really significant prospects to develop this into serious anti-HIV approaches for those patients in whom HIV cannot be kept under control by chemical means.''

On the Net:

Proceedings of the National Academy of Sciences: http://www.pnas.org

[kentb]

Thanks for posting this.  I am calling the Clinical study nurse in charge tomorrow to see if there is still enrollment for the new study.. Will keep all posted.

Kent

[kentb]

For those that are interested the new gene therapy study at the University of Pa. requires weekly visits to the University,  so it might be a good opportunity for anyone who is local.

thanks,

kent

[J220]

Thanks for that info. Did you find out what the inclusion criteria for the trial is? Is the trial listed in clnicaltrials.gov?

J.

[Gilles]

have read this in number of reliable papers and websites including The Economist. I like when hiv news pass more than HIV specific magazines and papers, it usually indicates that research is worth the attention.

[J220]

Yep it's always encouraging to see news like this widely disseminated.

If this trial continues to do well this may become THE standard treatment, one shot (or a series of shots) and that's it, your body does the rest, fighting off the virus so it doesn't multiply and infect other cells. As a result your viral load will drop and your cd4 will increase, most likely to undetectable in the former, and to near-normal levels in the latter.

And even if there are latent infeced cells, when they do wake up and start to churn out copies of the virus, they have nowhere to go, since more and more cd4 cells are immune.

Did you see the animation from VIRxSYS? This is the link from the website, it explains how VRX496 works:

http://64.176.10.223/pages/human-therapies/first-clinical-application/how-vrx496-works.php

Onward!!! J.

[bobino]

This is amazing stuff.  Let's all hope that the trials are successful and that this becomes available soon.  It would mean an end to all the drugs and their inevitable toxicities and side-effects.  I'm keeping my fingers crossed.

[J220]

It occurs to me that this would serve as a preventive vaccine as well, wouldn't it? If your CD4 cells are immune to infection then, infection cannot take place.

In any case, yes, this looks very good. This may very well be the next treatment revolution. Like youn said, no meds, no side effects. And the more time passes the more effective it is, because immunized CD4 cells will continue to divide, while those infected die off. J.

[blondbeauty]

This is going to make us HIV-proff. Not only corn is going to be transgenic. Humankind as well!

[J220]

I was reading a bit closer the from Virxsys's website and read this:

"Some patients that have reached the three year post-infusion safety visit continue to exhibit persistence of modified cells, decreased viral load and increased CD4 counts over baseline."

I didn't know that the doses had been given that long ago, and this was with a single small testing dose. With results like this I guess the media interest is warranted. I think we may have a winner here...

My main concern is with availability of this technology to developing worlds...sure, for most of us here in the US and Europe we'll have access to this, but given the complexity of the process, it will be difficult to disseminate. Or maybe not, if the WHO and other organizations get in gear on this. The other possibility is that Virxsys can develop this into injectable form (not requiring drawing blood, modifying it, and reinjecting it.) They mention this in the website, but it's not clear if it applies to this case.

Anyway, all in all it looks good. Apparently parts of the phase two are already under way and results should be announced in 2007. I expect great  news from it. They are testing multiple dosages and a single larger dose, that ostensibly will be more effective. The fact that they are determining optimum dose clearly indicates this is working as planned. J.