Gene Therapy Approach 'Completely' Protects Mice From HIV Infection

Pierre Bezukhov writes "Scientists from the California Institute of Technology have come up with a gene therapy approach that has proven effective in protecting mice (with humanized immune systems) against HIV infections. They used a genetically altered virus to infect muscles cells and deliver DNA codes of potent antibodies isolated from the blood of human HIV victims (abstract). The muscle cells then began to manufacture the antibodies in quantities that proved 'completely protective' against HIV infection. By contrast, traditional vaccines have not worked against HIV, as scientists have failed to find a molecule that induces the immune system to produce enough potent antibodies. The difficulties stem from the fact that HIV disguises some of its external structures from the antibodies."

How to conduct human trials

[scorp1us]

How do you conduct a proper trial for HIV? "Here, this is either a drug that will work, won't work, or a placebo which works a surprising amount of time. At best you have a 50/50 shot of getting HIV" Who is going to participate in that trial?

Turning off Gene Therapy?

[mathmathrevolution]

The article expresses a concern that once the gene therapy is started it can't be turned off if the person has an allergic reaction to the antibodies. Maybe somebody more informed can explain why:

1) You couldn't test for an allergic reaction in advance of the gene therapy.

2) You couldn't just do more gene therapy to turn off your original gene therapy.

Re:How to conduct human trials

[scamper_22]

You'll never get a proper scientific trial. There are whole areas of medicine we only know because evil regimes like the Nazi Germany conducted experiments without care.

But you can certainly make a best effort. Inject 1000 random people with either drug, placebo...

They do whatever they do. They can engage in risky behavior. They can wear condoms. Whatever is. As an experiment, you just assume their behavior is randomly distributed among the 1000 people.

At the end of the trial, you call them back and see what percentage has AIDS/HIV.
If all those who received the drug don't have it, and SOME of the ones without it did have HIV/AIDS, then you can say there is a high probability the drug works.

As I said, statistically you just have to assume that behaviors were distributed evenly among the 1000 person sample.

If no one comes back with HIV/AIDS... by some miracle that 1000 person trial... everyone suddenly behaves nicely and stops having unprotected sex, no one gets raped... well then the experiment is a failure, but at least 1000 people are not living a good life... so it's a human success.

Promising, but...

[FlavaFlavivirus]

This experiment will probably not produce an actual human drug, as it suffers from the same drawback as most previous gene-therapy studies: the Adenovirus transduction system will kill a significant number of patients. However, the results do seem to indicate that a monoclonal antibody has protective effects. The gene therapy vaccine may not work, but you could inject purified antibody into someone who had a known exposure, or is going to be in a high-risk situation, and prevent infection. Unfortunately, these types of therapies will never be able to cure an established infection, as HIV integrates its genome into host T-cells.

Re:How to conduct human trials

[Anonymous Coward]

As I understand it, the way to do a trial with this is to try it in an area where AIDS infection/transmission is common. For instance, test it on a group of prostitutes who don't have AIDS. When you come back a few years later, you check to see if any of your group have acquired the disease, and how their infection rate compares to others in their cohort.
 
Also, it's not uncommon to consent to acquiring a disease in the name of research (though they're not typically the lethal variety). I have a couple friends who work at Walter Reed designing malaria vaccines. If they ever feel like taking a couple weeks off, they consent to testing out one of the vaccines (which almost never work). They temporarily get malaria out of the deal, but they also get some time away from work, a few thousand dollars, and some free medical care.

Re:Gay Mice

[gyaku_zuki]

I love how you decided to keep this comment anonymous. 'It's as if' you are scared to actually back up your opinion. Seriously, in the 'playground' version of being a fag, you are the biggest one I've seen.

Gravity - 'It's as if nature doesn't think' humans should fly, but we do (planes).

Fun fact - nature isn't CONSCIOUS. It doesn't really give a crap what you think or do. Nature didn't wake up one day and think 'hey, I think I hate queers today!'. Unless you're religious, then of course why I even waste my breath is beyond me.

"Scientists from Caltech"?

[Christianson]

Maybe this is a silly, minor thing, but it bothers me these sort of blurbs always just talk about faceless "scientists." Does it really take that much work to find out who the principal researchers were? Maybe more people would be inspired to get into science if it actually seemed to come with some measure of face rather than anonymity in a lab coat.

Re:Can't Wait For The Peer Review

[cpricejones]

So the goal of the adenovirus is to introduce the broadly neutralizing antibody into T cell lines. These are the immune cells that are going to be ultimately fighting HIV infection, but they lack the right antibodies. In the normal situation, your body raises antibodies, but they cannot bind to the right spot on the virus envelope. Instead, they bind to a spot that the virus naturally varies, and the virus escapes via mutation (i.e., mutated virus replicates, other virus doesn't). In the new situation, the adenovirus provides an antibody that is better, which binds a spot on Env that the virus needs (so virus with mutation at this spot replicate poorly).

So muscle and heart cells are likely not getting the vector, nor would they be expressing antibodies. Similarly, your body wont continue to raise the antibody if the infection is gone (it will not be ad infinitum).

This is my understanding ... perhaps others could add key points.