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Gene Therapy May Thwart HIV

sciencehabit writes "Over the past few years, a man living in Berlin, Timothy Brown, has become world famous as the first — and thus far only — person to apparently have been cured of his HIV infection. Brown's HIV disappeared after he developed leukemia and doctors gave him repeated blood transfusions from a donor who harbored a mutated version of a receptor the virus uses to enter cells. Now, researchers report promising results from two small gene-therapy studies that mimic this strategy, hinting that the field may be moving closer to a cure that works for the masses."

23 of 171 comments (clear)

  1. Man if it cures HIV by Anonymous Coward · · Score: 3, Funny

    Then you know what it will do? Put thousands of people out of work!

    Think of the Pharmacists!

  2. Google delta CCR5. This is old. by wierd_w · · Score: 4, Informative

    The delta CCR5 mutation was already well known, and the subject of several (at least 4) different experimental receptor blocking and gene therapy medications, all of which were blocked by the FDA citing safety concerns.

    This is not meant to be a conspiracy theorist bottom feeding post, but simply intended to inform. There have been many studies of this mutation for thereputic uses conducted in Europe over the past decade, including seeveral promising phase 2 trials.

    Like most life saving medications though, any prospective cure for HIV will probably be developed in the US, and approved in Europe. (Then approved in the US after decades of routine use overseas.)

    While this particular gene therapy might be new, the mechanism is not novel.

    1. Re:Google delta CCR5. This is old. by wierd_w · · Score: 2

      What I am getting at, is that this mechanism has been the subject of clinical trial medications in the past, and refused acceptance every time.

      While the delta CCR5 mutation appears benign in humans under most circumstances, the burden of proving such benality imposed by the FDA will likely be insurmountable, and as such, even if this stuff kills off HIV infection in 24 hours of application, it will probably never get approval in the US.

    2. Re:Google delta CCR5. This is old. by wierd_w · · Score: 2

      In the cases of receptor blocking medications, there were sticky issues involving elevated liver enzymes, iirc. This caused the fda to reject. (Essentially the blockers are large protiens that bind to the receptor and plug it up like a cork, so the virus cannot dock. The body has to break down these foreign bodies in the liver to eliminate them, which causes elevated liver enzymes. A condition known to induce liver failure. Yes, several distinct medications were blocked by this decision.)

      The gene therapy trials were halted, because of issues involving reliability of the gene therapy's proper integration into the host genome in a reliable way. (EG, that the vector was consistent in how it integrated the new gene, and that the process did not increase risks for cancers in already immunosuppressed patients with aids.)

      Overshadowing both thorny issues was the question of if the actual deactivation of CCR5 (the mutation deletes a good portion of the gene that produces this receptor, making the cell produce nonfunctional versions) would itself be safe over the long run. Several studies have been comissioned to address this very subject, and initial results indicate that the delta32 CCR5 mutation increases risks of several nasty infectious agents, such as west nile.

      Until these issues are addressed, it is unlikey that the US FDA will approve any CCR5 blocking/disrupting medications.

      This is why I said that it is likey to be approved in Europe, and not the US.

    3. Re:Google delta CCR5. This is old. by ArsenneLupin · · Score: 2
      There's a huge difference:
      • thalidomide was a medication intended to cure a trivial condition (morning sickness) with gruesome side-effects on a third party (the kid).
      • this new AIDS treatment is intended to cure a deadly disease, with side-effects that only affect the patient himself.

      So, it looks like the risk/reward relationship is slightly different in both cases, doesn't it...

  3. Re:Cure for the masses by damicatz · · Score: 2, Interesting

    The difference is, with this, all someone has to do is patent the gene and then they can charge whatever they want.

    At least with spaceships and computers, you actually have to create something.

  4. Re:No cure, ever. Not in America. by bluemonq · · Score: 2

    Well shit, I guess I still have malaria.

  5. Time for Captain Obvious by erice · · Score: 2

    I'll trade in the pharmacists for unfettered, unprotected sex for all. A world without STDs would be an awesome world, indeed. Seinfeld's dream of an intercourse hello would be realized.

    Um. HIV is one that gives people the chills today but there are other STD's. Some, like genital herpes are highly contageous and incurable. Hepatitis C is less contageous but also incurable and potentially lethal. There are even antibiotic resistance forms of gonorrhea.

    1. Re:Time for Captain Obvious by Demonoid-Penguin · · Score: 2

      Genital herpes is a non issue for the most part

      Uh what? Because it doesn't kill you - just makes you wish you were dead.... fuck that! If I had a choice between syphillis or herpes (and no other choice) - I'd take syphillis. That I can get treated - and I could be honest about having it, use protection, and still get laid. You do not want to fuck with someone who would fuck you, if you had herpes.

    2. Re:Time for Captain Obvious by antifoidulus · · Score: 2

      It's a non-issue in the developed world where(outside of the US of course....) HIV is quite rare. However genital herpes, in either partner of a discordant couple, increases the odds of catching HIV considerably. The sores, often not even visible, defeat the skin barrier that keeps HIV out of the system most of the time. Furthermore, HIV tends to pool in very high concentration in herpes sores, significantly increasing the chance that it will be spread.

      If we cannot develop an affordable HIV vaccine/treatment, we could still go a long way towards preventing more cases by coming up with vaccines for HSV(as well as other STDs). We already have a vaccine that is effective against certain HPV strains, and it looks like a herpes vaccine may be possible. However, there is little interest in vaccines against chlamydia and gonorrhea since they are so easily(and cheaply) treatable in the rich world. However, completely eradicating them without a vaccine would be difficult if not impossible.

  6. Re:Cure for the masses by thehodapp · · Score: 3, Insightful

    I doubt not. Think of all the vaccines you're covered by now. Influenza, Polio, Hepatitis, etc. Those don't cost very much. Most Americans can afford those now. The deal with this is that it is probably only going to end up being a few treatments before you're fully cured so medical bills won't rack up. The other thing about this is that HIV is so widespread there will be plenty of donation money for those who can't afford it in the case that the cure turns out to be expensive.

  7. Majic Johnson? by Nidi62 · · Score: 2

    I thought South Park had definitively proven that Magic Johnson was able to cure his AIDS through all the money he's earned?

    --
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  8. Re:Just wait by xstonedogx · · Score: 2, Insightful

    You're completely forgetting the religious aspect. Only fornicators destined for hell are going to get AIDS. Clearly we need to bomb the free clinic.

    Also, it causes autism.

  9. Re:Science wins again. by xstonedogx · · Score: 2

    Jesus! It's not that I don't agree with you more or less, but why don't you and science get a room?

  10. Brain-dead. by westlake · · Score: 2

    Doctors do not cure. Not in America. They treat. They can't make a money off of you for as long as you live if they cure you. If they treat you, they can milk you until you die.

    Why does something so stupid always modded up to "Insightful?"

    The cure means that your patients have a real shot at rebuilding their lives and finances. It means that they will be a candidate for other medical services for perhaps the next half century or more.

    The cure opens the door to the understanding and treatment of other diseases.

    The cure is elusive. The cure may have side effects. The cure may dangerous. The surgical procedure that a weakened patient may not survive.

    The geek doesn't want medicine.

    What he wants is magic and miracle at a discount price.

  11. Re:No cure, ever. Not in America. by Lokitoth · · Score: 2

    And all those Polio treatments.

  12. Re:Small catch by rthille · · Score: 2

    No, the treatment they are working on is gene therapy, not the transplant that seems to have cured the man. They are related, but not the same.

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  13. Re:Small catch by ceoyoyo · · Score: 2

    "The survival rate of a bone marrow transplant is around 40% even in the best hospitals in the world."

    The survival rate for people dying of nasty forms of leukemia who get a bone marrow transplant from an unrelated donor when they're already on death's door might be about 40%, but the procedure itself isn't nearly that dangerous. Autologous hematopoietic stem cell transplant has a mortality rate around 5% on average, and groups who specialize in it are getting quite a bit better than that. Presumably that's what you'd do to treat - extract a patient's HSCs, genetically modify them, immunoablate, then inject the HSCs to reconstitute the immune system in HIV resistant form.

  14. Re:No way! by Electricity+Likes+Me · · Score: 3, Informative

    Uh, the HPV vaccine IS for the types of HPV that cause cancer. That's the entire point of the vaccination program. It is not a cure for all possible types of cervical cancer (only ~60% of them) but the HPV strains it vaccinates against are those linked to cancer + some other common ones (to encourage men to get the vaccine too and thus promote herd immunity).

  15. Re:No way! by Electricity+Likes+Me · · Score: 2

    Not really. To breed resistant strains of anything you need to have survivors. For example, the big problem with antibiotic resistant tuberculosis is people being non-compliant with their antibiotic regimes. So they get it, take the course for a few weeks or so and feel better, and then decide to stop taking the pills or start being lazy about keeping up to the schedule (which is 6-24 months).

    As a result, they haven't actually cleared the entire infection, instead they've neatly selected for the slightly more antibiotic resistant strains of TB, which they may end up passing off into the general population if they become infectious again.

    TB is particularly prone to this since it's difficult to treat in the first case (somewhat antibiotic resistant by nature, requiring prolonged treatments). It is much, much harder to develop resistance when you wipe out the entire population of something in one hit since there are no survivors to reproduce a new resistant strain.

  16. Re:A cure already exists: by Electricity+Likes+Me · · Score: 5, Insightful

    Or are a victim of a sexual assault. Or suffer a needlestick injury as a doctor treating someone with HIV (or have incidental blood-blood contact through say broken skin). Or if your partner of 10 years cheats on you. Or if the condom fails.

  17. Re:Obligitory Bill Hicks quote by backslashdot · · Score: 2

    Umm, on the incurable front there's still Hepatitis C, HPV (thanks to Michele Bachmann), and good ol' Herpes to worry about.

  18. Re:Just wait by Ginger+Unicorn · · Score: 2

    All I said was "that piece of halibut was good enough for jehovah"

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