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Biotech Medicine Science

Doctors Will Test Gene Editing On HIV Patients 263

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.'"
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Doctors Will Test Gene Editing On HIV Patients

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  • by Clover_Kicker ( 20761 ) <clover_kicker@yahoo.com> on Wednesday February 04, 2009 @12:20AM (#26721077)

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

  • by LaskoVortex ( 1153471 ) on Wednesday February 04, 2009 @12:23AM (#26721091)

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

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

  • by Anonymous Coward on Wednesday February 04, 2009 @12:39AM (#26721167)

    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.

  • bravo (Score:5, Insightful)

    by Ritz_Just_Ritz ( 883997 ) on Wednesday February 04, 2009 @12:47AM (#26721225)

    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.

    With so many people in the developing world suffering from HIV, it would be nice to see something like this fast tracked. I am sure that some of those folks, now intimately familiar with their own mortality, would be eager to participate knowing that they could potentially help other people.

  • Re:bravo (Score:5, Insightful)

    by BlueParrot ( 965239 ) on Wednesday February 04, 2009 @12:55AM (#26721251)

    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.

  • by dunezone ( 899268 ) on Wednesday February 04, 2009 @01:01AM (#26721289) Journal
    Yeah, those modern anti-retroviral are not so perfect though. Most of them are combo drugs which means you take multiple pills at a time. As a result of that the side effects can be extremely painful and dangerous. And I know, its better then death if you don't take them but finding a better alternative is a must. These drugs are expensive and the majority of individuals who have HIV don't have access to these them.

    And just like anti-bioitics. After a certain period of time these drugs become ineffective. Hell, if you start missing doses or cant afford them it could easily be game over.

    The people in these studies are failing current treatment methods. There is nothing left at this point.
  • by MichaelSmith ( 789609 ) on Wednesday February 04, 2009 @01:17AM (#26721363) Homepage Journal

    This is what we need. Medicine is far too conservative in so many cases.

    I think we need more engineers involved in Medicine, particularly on the implementation side. I wish I could point to this but I remember reading about a scientist who had invented something quite revolutionary who spent the next seven years rebuilding his machine to be small enough for general use. That is the kind of job which should be given to an engineer, but it won't keep the scientist employed.

  • by ThrowAwaySociety ( 1351793 ) on Wednesday February 04, 2009 @01:34AM (#26721457)

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

    In which case, you are no worse off, and at least get careful observation and conventional treatments for your symptoms.

  • Mutations (Score:2, Insightful)

    by Spooon69 ( 758526 ) on Wednesday February 04, 2009 @01:41AM (#26721487)
    The HIV virus has a high rate of mutation, one of the reasons it sticks around in your body and your immune system has to keep attacking it, it's pretty much a "new" virus every time. What's to keep the virus from mutating and avoiding the CCR5 requirement it currently has? CCR5 doesn't seem to be a requirement for a normal human immune system (one of the many types of backups the immune system has), thus some percentage of the population being perfectly healthy without that receptor. I'd even go as far as to say that if HIV mutates into not requiring CCR5, then this new strain could spread and theoretically be worse than the current HIV strain in the wild.
  • by daveime ( 1253762 ) on Wednesday February 04, 2009 @01:46AM (#26721511)

    No, the only thing we need to rethink is how the pharma companies can ever justify selling a medication that costs $200 for a single dose.

    You know, Chris Rock summed it up nicely. No one will ever cure AIDS, the money isn't in the cure, it's in keeping you alive and helping you live with it. Just get your "fix" and live for one more day.

  • by TubeSteak ( 669689 ) on Wednesday February 04, 2009 @02:17AM (#26721647) Journal

    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.

  • Re:bravo (Score:1, Insightful)

    by Anonymous Coward on Wednesday February 04, 2009 @02:24AM (#26721691)

    That is not dead which can eternal lie.
            And with strange aeons even death may die.

  • I'm sure your stance would change if you were someone who was in need of treatment and didn't have the appropriate medical insurance.

    Probably would. But the way I see it now is, I smoke, I've smoked for 20 years, and I'm starting to get the first reduced lung function and I should quit but probably won't and I'll die from it, dragging myself and the rest of society down for a million bucks in chemo and treatment. What's up with that? Why should I keep 50 kids out of college because I was a dumbass? That's not right. Same thing with HIV. For the most part, you can prevent HIV. You can stay monogamous as much as you can. Don't be a slut, and use a rubber. Don't use needles. Sometimes you have to think about what your impact is on society.

    I think the real problem is the outrageous cost of treatment. Some drugs being manufactured have higher marketing costs then they do for research.

    Maybe the stuff is expensive because it is, well, actually complicated to research, make and produce. Science is a craft and crafts are expensive. I don't doubt that the workflow in a pharma company is probably retarded, and that makes it more expensive, but, at the same time, the problem is really that all the knowledge is completely new, so there's no automation in any of the research. It's not like McCoy can just drop a blood sample into the computer and whip up a batch of cure.

    Anyway, you seem to have a rather callous view on who should or shouldn't get treatment and I think its deplorable.

    That's Democracy. If you want to have private health insurance, have private insurance. Otherwise, accept that, there's going to be people who will make the argument that we should not blow too much medical money on sufferers of illnesses resulting from lifelong self indulgence or excessive risk taking.

  • by Xenna ( 37238 ) on Wednesday February 04, 2009 @02:37AM (#26721765)

    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.

    HIV is a bastard because it's relatively benign and very hard to transmit. A normal deadly virus like Ebola kills you quickly, HIV keeps you healthy and able to infect others for years. It's mainly transferred by sex, which is a big bummer for all of us who like to sleep around which includes lots of gay men.

    But once you have it, you won't get rid of it. Nothing special either, because most people live with the chicken pox virus for most of their lives. That usually doesn't kill you, though...


  • by wickerprints ( 1094741 ) on Wednesday February 04, 2009 @02:38AM (#26721771)

    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

  • Re:bravo (Score:4, Insightful)

    by The FNP ( 1177715 ) on Wednesday February 04, 2009 @03:06AM (#26721975)

    Actually, I figure this is exactly why /. has ACs. We put up with nearly every other AC troll post so that occasionally we can read 'Confessions of a HIV+ Slashdotter'. I don't need to know your identity to read your anecdote. Also, I respect your right to privacy and AC posting more in this case than if you were merely stating a preference for Windows over Linux.

  • by rrkap ( 634128 ) on Wednesday February 04, 2009 @03:43AM (#26722179) Homepage

    I should add that this isn't a reason not to pursue research for cures for HIV. It's a very interesting problem and this technique may have broad application if it works. Also in some parts of the world HIV is very common and hard to avoid.

    Just as we would treat a heart attack in a 400 lb man, so we should also treat someone with HIV as best we can.

  • by PatrickThomson ( 712694 ) on Wednesday February 04, 2009 @04:12AM (#26722295)

    HIV research is in danger of being halted because it's not seen as profitable; now that *would* be a major health catastrophe.

  • by MoellerPlesset2 ( 1419023 ) on Wednesday February 04, 2009 @04:20AM (#26722333)

    Chris Rock summed it up nicely. No one will ever cure AIDS, the money isn't in the cure, it's in keeping you alive and helping you live with it. Just get your "fix" and live for one more day.

    Yeah, because Chris Rock has a unique level of insight into the realities of drug development

    It's bullshit. Utter bullshit of the kind that can only come from the cynicism and arrogance that follows total cluelessness.
    Do you think bio-medical researchers are some kind of wizards? Do you think we know so much that we have the luxury of being able to pick and choose between solutions? Bullshit.

    Do we know how the human body works? No, we do not.
    Do we know how diseases work? In most cases, no.
    If we did - Cancer is a good example of an illness known in great detail - does that mean we know what to do about it? It does not.
    If you have an idea of what to do - does that mean it'll work? It doesn't.
    If it works in lab, does that mean it'll work in a live human? It does not. (see the first point)

    Do you know how many drug concepts (that is, ideas) that make it to becoming a finished, FDA-approved drug? It's not even one in ten thousand. With a situation like that, you do not have the luxury of picking and choosing. You go with whatever idea you have, and you take it as far as it'll go. If you're lucky, it might turn into a drug. And it most certainly won't work exactly as imagined from the start.

    Do you know how Viagra, that blockbuster bestseller of 'lifestyle' drugs came about? It was a side effect of a drug originally developed for angina and pulmonary hypertension - a heart drug. The main factor in drug development remains: luck.

    But if you still think there are plenty of good, uninvestigated options for AIDS drug development out there, I suggest you go do it yourself. Maybe then you'll gain some real insight into how complicated reality really is.

  • by N1AK ( 864906 ) on Wednesday February 04, 2009 @05:19AM (#26722589) Homepage

    That's Democracy. If you want to have private health insurance, have private insurance. Otherwise, accept that, there's going to be people who will make the argument that we should not blow too much medical money on sufferers of illnesses resulting from lifelong self indulgence or excessive risk taking.

    I prefer to look at it from the other angle. No country in the world has a social healthcare system that will give people unrestricted access to any and all available treatments. In the UK this is often most visibly seen when the NHS decides not to provide certain very expensive drugs that can extend the lifetime of Cancer victims.

    I don't have a figure available to say what providing truely top level healthcare to everyone so I don't want to make sweeping statements about how unaffordable it would be, but this is exactly what needs to be considered when saying everything should be on social healthcare.

    Is it moral for me to suggest that some treatments aren't provided socially, and then pay money into insurance that gives me those treatments? I'm not 100% sure, but I already pay money into a private pension scheme which will help me avoid being stuck on the tiny state pension when I retire and have mortgage insurance through work that protects my house if I become employed, something that everyone doesn't have. Life saving medicine is a more complex issue because one of the arguements ultimately boils down to saying it costs me too much to pay for healthcare that will save someone else's life.

  • Re:bravo (Score:1, Insightful)

    by Anonymous Coward on Wednesday February 04, 2009 @05:31AM (#26722643)

    You're going to die from AIDS and go to hell. Satan and his demons are going to make you their anal slut for all eternity. It's your fault for being a fag and taking it up the ass. Just realize your fate and know hell doesn't use condoms or give out breaks. 18 demon cocks in your faggot hole all the time. enjoy your little bit of time left here.

  • by Anonymous Coward on Wednesday February 04, 2009 @06:04AM (#26722789)

    So, HIV is mostly preventable, eh? Go tell that to one or two of the millions of kids infected at birth. Make sure you look them in the eyes when you do.

    How about the millions of Americans who weren't told smoking is BAD(tm) when they were young? For your second exercise in compassion, go visit a cancer ward one of these days and try to pick the old folk who deserve treatment for kidney cancer from the undeserving. Make sure you tell them what you decided.

  • by CarpetShark ( 865376 ) on Wednesday February 04, 2009 @06:35AM (#26722939)
    Like it or not, being a dumbass is part of the human condition. Compassion is recognition of that.
  • by daveime ( 1253762 ) on Wednesday February 04, 2009 @07:21AM (#26723167)

    Ah you poor blinkered fool. The pharmas spend all their time playing these kind of tricks, and very little time actually developing ANYTHING new.


    In Malcolm Gladwell's story in The New Yorker, Gladwell discusses the evolution of Prilosec, a popular heartburn medication that, in one five year stretch alone, earned twenty-six billion dollars. With the patent about to expire in 2001 a good thing was about to come to an end for the drug patent's owner, AstraZenneca. The company set out to create the "new and improved" version of Prilosec, one that would qualify for a new patent and keep the money machine rolling. Ultimately, the company discovered that by making a very subtle change in the chemical engineering of Prilosec, they would have an entirely new drug. But to be issued a patent, they had to prove that the new version was better than the old. They decided that their best opportunity was to focus their testing on something called erosive esophagitis, a condition where stomach acid bubbles up and harms the lining of the esophagus.

    For the first month of the study, the new drug's impact on this problem was identical to Prilosec's. Bad news for the drug company. But in the second month, tests showed that there was a 90% heal rate with the new drug versus 87% with Prilosec, a difference of only 3% for one month but good enough to qualify for patent protection. Thanks to this small and insignificant improvement, AstraZenneca gained FDA approval, was awarded a new patent and put the drug on the market under the name Nexium. The cost for a month's supply of Nexium is about $120. The cost for the now generic version of Prilosec? About $20 for a month's worth of pills.

  • by GlassWhale ( 1295841 ) on Wednesday February 04, 2009 @08:09AM (#26723365)
    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.
  • Re:Mutation? (Score:1, Insightful)

    by Squeeonline ( 1323439 ) on Wednesday February 04, 2009 @09:42AM (#26723907)

    The problem being that many cell receptors that these viruses bind to (eg CAR coxaci adenovirus receptor) are highly conserved in humans. If we could just mutate the gene(s) that code for that receptor and nothing else happened it would be fine.
    The reason that these genes and receptors are conserved is that they have essential functions, that may not yet be fully (if at all) understood.

  • by karlwilson ( 1124799 ) on Wednesday February 04, 2009 @10:17AM (#26724191)
    Now THAT'S insightful.

Research is what I'm doing when I don't know what I'm doing. -- Wernher von Braun