Is Gene Therapy a Safe and Effective Treatment for HIV?

[G01]

Wow. It's a brutal route, but it got him there:

http://gizmodo.com/5713498/man-officially-cured-of-hiv

http://www.nytimes.com/2008/11/14/health/14iht-14hiv.17818039.html?_r=1

MSNBC is skeptical:

http://www.msnbc.msn.com/id/40666443/ns/health-aids/

CNN:

http://www.cnn.com/2010/HEALTH/12/14/hiv.infection.cure/index.html

 

[The legend]

Awesome.
I think we'll soon be rid of this disease. Millions can be saved..wonderful!

 

[Lacy33]

This is really great. But wouldn't it have been breaking news all over?

 

[G01]

This is really great. But wouldn't it have been breaking news all over?

Yeah, I can't believe this isn't headline news!

 

[Proton Soup]

2008 seems to make it old news. heck, would you ever know, tho? when you get over chicken pox, it still never goes away. it just sits there dormant, waiting for you to get old so that it can bring you to your knees with shingles.

 

[nismaratwork]

I remember when this case first emerged... it's unique, but not a herald of a new treatment any more than discovering vaccines in general cured all viruses. His immune system had a freak-chance to recognize the coating on that particular strain of HIV, and all it took was metastatic cancer.

Immune therapies are nothing new, and the immune system having odd reactions to the neoplasms produced by some cancers is nothing new either.

So, not BS, but not really the light at the end of the tunnel either.

 

[Lievo]

The paper is here:
http://bloodjournal.hematologylibrary.org/cgi/content/abstract/blood-2010-09-309591v1

 

[bobze]

The trade off of getting a bone marrow transplant and having to take immune suppression drugs the rest of your life to avoid host-graft disease (not to mention all the pain associated with a undergoing such a transplant) in exchange for HIV isn't much a trade-off--Or anything to get that excited about.

We've known for a quite awhile that certain populations have alterations to their CD4 or CCR5 receptors which make them incredibly resistant, if not immune, to HIV infections. It would be little surprise then that someone who receives a bone marrow transplant from one of these individuals would be bequeathed the legacy of mutant receptors.

Ideally, if the staunch conservatives in the NIH would lighten up on gene therapy, we could take someone who has HIV; take some of their bone marrow, infect those cells with mutant CCR5 receptor genes, wipe out their immune system and retransplant their own bone marrow back into them. Since the "bubble-boy" (SCID) incident though, we've been overly and detrimentally (IMO) cautious with gene-therapies (which really wasn't an incident as far as cost benefit is concerned; I mean you either die when you're 5 or live to your late teens or 20s and risk getting cancer, which can be treated--Not a hard choice IMO).

Of course the cost to this procedure would certainly be limiting to who could get treated for HIV--But its a start.

 

[Lievo]

I agree with you, bobze, but for the part where you minimize the side effect of getting a cancer. To me it was not an incident. It was a hugh failure in need to be fixed.

 

[bobze]

I agree with you, bobze, but for the part where you minimize the side effect of getting a cancer. To me it was not an incident. It was a hugh failure in need to be fixed.

Well again, I think its a matter of "cost-benefit". You can't control where a viral vector inserts genes, ergo it is possible it will insert genes into places that may not beneficial.

In the case of "bubble-boy", he had SCID. A disease in which you have literally no immune system--None. People with this disease, without transplants or gene therapy live to the ripe old age of "a few years old". When they underwent the first gene therapy trials, some of these children (a decade later) developed leukemias. I think for the time being, the trade-off of leukemia (something much more treatable than a total lack of an immune system) is worth the risk. At least for this disease, maybe not others.

The point was though, that this severely set back gene therapy treatments available to other people with the disease, because of an over-reaction (IMO). Given the choice between "imminent" death and dealing with leukemia (though it should be noted that only 4 or the 10 patients developed leukemia) a decade down the road (a decade of life you wouldn't have)--I'll take my chances with the leukemia any day.

I think it is incredibly wrong to not put the choice into those families hands if given informed consent. I know as a parent, if my child had SCID--I'd again, take the chances with leukemia down the road.

I'm not saying the situation is ideal, but there are a lot of disease (and families) that gene therapy could be helping where the pay off (not imminent death) is worth the current risk, IMO. And I'm not minimizing the potential side of effects of current gene therapy--Of course, this is something we need to be putting lots and lots of research dollars into, such that we can learn to control the position of gene insertion, but the point I maintain is that the decision for current gene therapy should be in the hands of individuals and families afflicted by diseases where the option of "no therapy" is imminent death. Is the situation (at current) ideal? Certainly not, but when you have nothing else to loose--You have nothing else to loose. Know what I mean?

 

[Lievo]

I think it is incredibly wrong to not put the choice into those families hands if given informed consent.

I can agree that. But I will stress that for an informed consent to take place one must provide accurate information:
-First, we should mention that there exists alternatives such as allogenic bone transpantation.
-Second, we should talk about what it is to have a leukemia -dying is one thing, but we have to think about pain, fatigue, and crash of the false hopes we'll have induce.
-Third, we should mention that we presently cannot evaluate the cost, as the risk of oncogenesis in successful gene therapy trials will be known only after treating more patients and closely following them long term.*. We know Leukemia is a risk, that's not to say this is the only one risk. It's actually possible that other side effects exist that we still don't know about. In other words, we should make clear that up to know we're still talking about experimenting new treatment. Families must be well aware of that, or they can't provide informed consent in the first place.


*Here I stole a sentence so let's put the http://download.journals.elsevierhealth.com/pdfs/journals/0091-6749/PIIS0091674904009212.pdf".