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Doctors Will Test Gene Editing On HIV Patients

Soychemist writes "Some people have a mutation that makes them highly resistant to HIV, and scientists think that they can give that immunity to anyone with a new type of gene therapy. The first human trials will start at the University of Pennsylvania this week. Researchers will draw blood from people with drug-resistant HIV, clip the CCR5 gene out of their T-cells with a nuclease enzyme, grow the modified cells in a dish, and then return 10 billion of them to the patient's bloodstream. Those cells will be immune to the virus, and they will keep the patient's T-cell count up even if the rest are destroyed. 'We will see if it is safe and if those cells inhibit HIV replication in vivo,' said the lead researcher. 'We know they do in the test tube.'"

16 of 263 comments (clear)

  1. might as well guinea pig at that point by Clover_Kicker · · Score: 5, Insightful

    What's the worst that could happen, they screw it up and you die?

    1. Re:might as well guinea pig at that point by LaskoVortex · · Score: 5, Insightful

      What's the worst that could happen, they screw it up and you die?

      Or they could not treat you and you'd die.

      --
      Just callin' it like I see it.
    2. Re:might as well guinea pig at that point by Anonymous Coward · · Score: 5, Insightful

      This is what we need. Medicine is far too conservative in so many cases. Death would be a welcome accident in many diseases of terminal illness. Not saying the patient shouldn't decide, but they should certainly have the option to try experimental treatments if they want.

    3. Re:might as well guinea pig at that point by RelaxedTension · · Score: 5, Funny

      Or you volunteer, and be the one to get the placebo.

    4. Re:might as well guinea pig at that point by Mauzl · · Score: 5, Informative

      If you RTFA, its people who have drug-resistant HIV who are being experimented on.

    5. Re:might as well guinea pig at that point by TubeSteak · · Score: 5, Insightful

      These drugs are expensive and the majority of individuals who have HIV don't have access to these them.

      Those drugs are expensive in (mostly Western) countries that kowtow to pharmaceuticals and have not stood up and supported generics.

      India was the first country to start cranking out generics and is probably the sole reason that Big Pharma dropped prices from $10,000~15,000 per year to less than $1,000 on what are now considered second-line drugs.

      Generics were the only thing pulling down insane prices and the USA's response was to setup TRIPS (Trade-Related Aspects of Intellectual Property Rights) which enormously complicated the process of issuing compulsory licenses.

      Off the top of my head: India, Brazil, Thailand and South Africa have all issued compulsory licenses for AIDS drugs.
      AFAIK, Thailand and Brazil are the only two countries to do so post-TRIPS.

      In response to Thailand's compulsory license, the price of one drug dropped by half for many other developing countries. OTOH, a major pharmaceutical said they wouldn't be applying for licenses to sell new drugs in Thailand... and the USA put Thailand on a watch list of countries considered to be committing intellectual property theft. So it was something of a Pyrrhic victory for Thailand.

      Long story short: Big Pharma's bottom line is getting in the way of preventing major health catastrophes in developing countries.

      --
      [Fuck Beta]
      o0t!
    6. Re:might as well guinea pig at that point by jsse · · Score: 5, Funny

      What's the worst that could happen, they screw it up and you die?

      You'll lose all your body hair; become a human-biting-albino that're very sensitive to sunlight; start biting humans and then turn them into your kind; you'll keep your biting until the T-virus in your body because air-bourne infecting disease, when you'd find no human alive for you to bite.

      I didn't mean it'd happen, but since you asked for the worse...

    7. Re:might as well guinea pig at that point by CarpetShark · · Score: 5, Insightful

      Like it or not, being a dumbass is part of the human condition. Compassion is recognition of that.

    8. Re:might as well guinea pig at that point by GlassWhale · · Score: 5, Insightful

      I don't know what you'd achieve by telling HIV-infected children that many adults contract HIV through their own carelessness. Likewise I can't see the point of telling them there's no Santa Claus. Doesn't make it less true though.

  2. Re:Sounds expensive... by dunezone · · Score: 5, Informative

    Every time someone posts about HIV we get a jackass like you. There are people who have HIV and didnt contract it through sex or drugs.

    There are people who were born HIV+ because their mother was a carrier, there are people who have been raped and now carry the virus, there are people in the medical field that contract it because some drug addict freaks out while they tend to them. Hell, even though we test blood now many people contracted it through blood transfusions before they tested it.

    Oh and by the way. Condoms don't give 100% protection against HIV its about 80-85%.

    HIV is a bastard of a virus. Our immune systems can usually handle most viruses without intervention. You cant win on your own against HIV. It will destroy the immune system eventually.

    If this treatment is successful at this level. At least we can give life to those who didn't have the choice.

  3. M-x genotype-mode by Anonymous Coward · · Score: 5, Funny

    You *know* there's going to be an Emacs mode for gene editing.

  4. Re:bravo by BlueParrot · · Score: 5, Insightful

    While strides have been made in HIV treatment, it's still a death sentence. Doctors can keep the patient alive longer, but they can't prevent the inevitable.

    Life itself is a death sentence. We all die sooner or latter, it's merely a matter of how long it takes and many HIV positive patients die from unrelated causes ( such as car accidents ). If you catch HIV when you're 70 there's a good chance you will die from some completely unrelated condition, such as stroke, heart failure, or lung cancer from a lifetime of tobacco abuse. Not saying an HIV infection is to be taken lightly, but believe it or not, it is far from the worst diagnosis you can get.

  5. Potential Failure RIsks: by LonghornXtreme · · Score: 5, Informative

    There are 3 big risks / problems I see with this approach:

    #1: The modified T-Cells attack the host after they are reintroduced. Think of it like auto-immune disease or transplanted-organ rejection. This could cause effects ranging from a mild food allergy to death. Anyone know how much damage 10 billion rogue T-cells could do? I sure don't; however, I do know that they aren't a straight 1 T-Cell used up for each 1 antigen.

    #2: Unmodified T-cells attack the modified T-Cells because the surface of the modified T-Cells (i.e. the CCR5 protein) could possibly trigger an immune response. This would render the modified T-Cells kind of pointless. Seems like this would have better chance of working on patients with full blown AIDS rather than merely HIV+.

    3: Modified T-Cells survive and are unaffected by HIV; however, these surviving modified T-Cells are just clones of the one original T-Cell that the lab modified. So in essence, you have injected the test subject with 10 billion of the same T-Cell. Unless the doctors have a way of massaging the genes on a representative sample of T-Cells, then this is kind of useless to the patient. What good are 10 billion T-Cells if they are each only good for tagging one antigen? Meaning, that the 10 billion T cells could only respond to a single stimulus, i.e. they could all only fight one strain of the common cold, but not anything else.

    Disclaimer: I have a BA in bio from a public ivy; however, my GPA wasn't that great, and I didn't pursue a career in the field. I very well could be overlooking something substantial in immunology etc.

  6. Re:bravo by Anonymous Coward · · Score: 5, Informative

    Actually, this is far from the truth. I am an HIV-positive individual and based on the fact that my HIV was detected early and also that I started on HIV medications within the first year of contracting it my lifespan has been extended to the point where I will most likely live a full life. Before contracting HIV I thought it was a terrible thing to have and that I would kill myself if I ever got it. I have news for you: it is a terrible thing, but just like everything else in the world you get over it and with HIV medications having nearly NO side effects and once-to-twice daily dosing it makes life worth living.

    While some may speculate on whether or not I really know what I'm talking about, I do. I am a healthcare professional. For those of you who are wondering why I didn't post this under my account, I would rather not taint Google's results with gossip for my next employer. :)

  7. Re:CANCER by Wain13001 · · Score: 5, Informative

    For the most part, T-cells die off naturally.
    People infected with HIV do not have to deal with a problem of too many t-cells, so in this case it's not much of a concern.

    Most importantly though, T-cells do not replicate to create more T-cells. They come from a type of lymphocyte starter cell (a stem cell essentially), which reproduces in the bone marrow.

    Taking t-cells from your body, and then reintroducing them to your body will not give you leukemia (literally meaning "White Blood", refers to various cancers of white blood cells).

    More than likely this is just a test, not *the cure*. The point being to see if the modified t-cells survive long enough to keep the count from dropping (as it would via a normal HIV infection). If it does work, then we can start developing methods to modify bone-marrow in order to make the new t-cells your body creates have the altered gene.

    Of course, I'm not an expert, and the article is unfortunately slim on details, so this is basic speculation.

  8. Not to dampen hopes, but... by wickerprints · · Score: 5, Insightful

    Instead of just making the typical /. armchair commentary about the zillion ways in which this proposal would be foolish or at best useless, I'm going to give a different angle on this.

    First, on a global scale, the most sophisticated HIV treatments are administered to the relatively wealthy. Only when such a treatment is deemed effective does it start to spread down to the poor, due to economics. The old anti-retrovirals of yesterday are today's low-cost options for the millions of HIV+ individuals in developing nations. That's just the reality of the technological development of disease treatment. However, this "trickle-down" mechanism, combined with natural geographic and genetic variations, has led to the evolutionary branching of HIV into significantly distinct strains, with characteristically different disease modalities.

    Second, we have as yet no drug that is able to eliminate HIV in the body. The currently available treatments are at best able to turn HIV into a chronic, managed condition. This has some very interesting (some would say alarming) socioeconomic implications. What we are finding is that over time, HAART therapy has evolved from a multiple-dose-per-day regimen that was difficult to maintain, to a more easily managed schedule, leading to better therapy adherence in patients. However, some of these drugs are poorly tolerated in many individuals, and over time, HIV is known to develop multiple resistances due to poor adherence or tolerance. The more disturbing situation, however, is that in many gay communities, the practice of "pre-exposure prophylaxis" has become alarmingly common. What is happening is that some HIV- gay men are obtaining anti-HIV drugs and taking them prior to knowingly exposing themselves to potentially HIV+ individuals through unprotected sex.

    From a scientific standpoint, it is fascinating that this development is as successful as it has been. But from the standpoint of a gay man who takes every precaution to educate myself and follow safer sex practices and does everything in my power to serve as a role model for responsible behavior, I find it totally abhorrent that there are guys who expose themselves willingly to HIV in such an unethical manner, in light of all the AIDS deaths that have come before us and all the tireless work of our most brilliant scientists, medical care providers, and public health advocates. They have even given this "PrEP" cute names and euphemisms to disguise the utter insanity of what they are really doing (like they have done with the term "barebacking" to refer to unprotected anal intercourse). If there is anyone on the face of this earth that deserves to die of this terrible disease, it is them. And I don't say that lightly. Some of you might say that these people would have had unprotected sex with or without the drugs, but you have to realize that it is partly through the action of these individuals that drug-resistant HIV is spread. It is for this reason I dare stand in judgment against them.

    So this brings me to my third point. The CCR5 discovery is notable in that it confers strong resistance against HIV-1. Two copies of the gene are required for this resistance. However, the transmission of other strains of HIV may not be blocked by the presence of this gene. Even if this therapy were to work, I doubt it would be effective on a large scale. Some of these patients, if you cure them, will simply go out and have more unprotected sex. If you don't believe me, reread the previous paragraph.

    The only way human civilization will ever rid itself of the scourge of HIV is if we discover a vaccine or outright cure for all its strains. No chronic management or piecemeal therapy will be sufficient, because there are always people who will do things that will enable the virus to mutate and survive. Ever since the discovery and announcement of the virus in the 80s, this simple fact was apparent to me. But the untold billions of dollars in revenue that HIV research and managemen