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Medicine Science

Doctors Used HIV To Develop Cure For 'Bubble Boy' Disease (bbc.co.uk) 103

Posted by BeauHD from the first-of-its-kind dept.
An anonymous reader quotes a report from the BBC: U.S. scientists say they used HIV to make a gene therapy that cured eight infants of severe combined immunodeficiency, or "bubble boy" disease. The babies, born with little to no immune protection, now have fully functional immune systems. Untreated babies with this disorder have to live in completely sterile conditions and tend to die as infants. The gene therapy involved collecting the babies' bone marrow and correcting the genetic defect in their DNA soon after their birth. The "correct" gene -- used to fix the defect -- was inserted into an altered version of one of HIV, the virus that causes AIDS. Researchers said most of the babies were discharged from the hospital within one month. Dr Ewilina Mamcarz of St Jude, an author of the study, said in a statement: "These patients are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives. This is a first for patients with SCID-X1 (the most common type of SCID)."

The study was published in the New England Journal of Medicine.
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Doctors Used HIV To Develop Cure For 'Bubble Boy' Disease More

Doctors Used HIV To Develop Cure For 'Bubble Boy' Disease

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  • Cool! (Score:5, Insightful)

    by Evtim ( 1022085 ) on Friday April 19, 2019 @08:05AM (#58458930)

    Don't know much about this topic, but it sounds reasonable and also very cool....using something deadly to save lives.

    Congrats to the researchers!

    • Re:Cool! (Score:5, Interesting)

      by StormReaver ( 59959 ) on Friday April 19, 2019 @08:11AM (#58458950)

      That's very similar to the story that ran a few weeks ago about using the Measles virus to cure a form of cancer in patients with no Measles antibodies (they tended to kill the Measles virus).

      These are very cool developments!

    • But what about natural selection? Do we just throw that out the window now?

      • Re: (Score:3, Insightful)

        by TimothyHollins ( 4720957 )

        Natural selection *is* genetics. These babies survived due to natural selection of intelligence in the scientists that developed the fix. Survival of the fittest doesn't refer to each singular individual in a vacuum, it includes such things as social skills, capabilities of the group/clan/society in which the individual lives, etc. The selection here was related to babies being cute as fuck, thus motivating older and smart people to help them.

      • Directed evolution (Score:5, Interesting)

        by Okian Warrior ( 537106 ) on Friday April 19, 2019 @09:14AM (#58459186) Homepage Journal

        But what about natural selection? Do we just throw that out the window now?

        That's a very good question to ask, but it has a direct answer: We take responsibility for our own evolution.

        There's no reason we should allow ourselves to die over a random genetic change any more than we should allow ourselves to die over a random tornado.

        And for those who think that the gene is there for a reason, and may be useful in the future to combat some unforeseen environmental event: if we gain enough genetic knowledge then it won't matter. We can repair the gene in people today and reintroduce the gene (or engineer some other fix) in the future if it becomes a problem.

        It's all balled up into the idea of making ourselves better, which seems to be the goal of evolution.

        • Re: (Score:1)

          by bozzy ( 992580 )

          Evolution has no "goals". Evolution, at a fundamental level, is a change in gene frequency, in a given population, over time.

          Whether genes stay around in the population are only dependent on whether there is selective pressure on the traits they manifest as it relates to reproduction. Genes need not have a "reason" and may be neither helpful nor injurious to reproduction. In that case, they tend to stay in the genetic line.

          Does old-age related male pattern baldness or attached vs detached earlobes have an

      • But what about natural selection? Do we just throw that out the window now?

        Natural selection can be cruel to watch. We gave it up with welfare decades ago. Now people unable to pay care for kids get paid to have more. Surely you've seen how society is getting better year after year?

      • We've been thwarting 'natural selection' progressively for thousands of years now, using something called 'medical science'. Know that strep infection, or mononucleosis, or {insert illness you had when you were a kid, *here*} that you took antibiotics for? Congratulations, you 'cheated' natural selection. If you're really a big fan of natural selection and have not passed your genes on to a new generation yet, you can always commit suicide and restore natural selection to it's rightful place in your life. ;

    • The basic idea of modifying an immunovirus to in turn modify a defective immune system into a functional state is indeed brilliant.

      I question the wisdom of using HIV specifically though - the disease is so dangerous precisely because it is extremely genetically unstable, with a far higher mutation rate than all but a few other viruses. That makes it virtually impossible to develop a cure since the virii you're fighting today are all different than the ones you were fighting last year (and from each other)

      • I question the wisdom of using HIV specifically though - the disease is so dangerous precisely because it is extremely genetically unstable, with a far higher mutation rate than all but a few other viruses.

        Hmm, sounds like you need to send an email, at least, to the researchers. They obviously don't know this, and never considered the possibilities inherent in their "solution".

        Luckily, your insight into the process will, no doubt, save many lives, and possibly even win you a Nobel....

        • An appeal for caution when dealing with a dangerous, rapidly mutating virus is totally out of line and worthy of sarcasm.

          “Your scientists were so preoccupied with whether or not they could, they didn’t stop to think if they should.” -- Weird rockstar-wannabe math guy from that dinosaur movie.

        • I have no question of their competence. However *wisdom* is rarely of any direct benefit in the scientific fields, and its lack is often prominently seen years or generations later.

      • Re: (Score:2)

        by ceoyoyo ( 59147 )

        The HIV virus in question is used in a culture dish on extracted immune stem cells. It's also disabled so it can't replicate.

        They're not injecting HIV into children.

    • I also like the idea that this can de-stigma HIV a little bit. Some religious fanatics liked to use HIV and AIDS as excuse to be mean to people. Because "God made this virus just to kill off the sinners". Seeing this being used for good, puts a bit of a dent in this argument.

  • moops!

    • Heard the Seinfeld slap bass* riff in my head as I was reading "Bubble Boy".

      * - Actually from a synthesizer played by Jonathan Wolff

  • ...for John Travolta!

  • Retrovirus. (Score:5, Informative)

    by Gravis Zero ( 934156 ) on Friday April 19, 2019 @08:24AM (#58458988)

    What they really did was modify a retrovirus [wikipedia.org], a virus that rewrites part of your DNA. Of retroviruses, HIV has been studied the most and thus it's mechanisms are best understood. Expect more DNA patches to be made for people with one shitty gene that is making them sick.

    • Any reason you would continue to use retrovirus techniques over CRISPR once the latter is more understood?

      • Could be many many reasons. From Patents to, off-target mutations, to simplicity, to who knows.

      • Re: (Score:3, Insightful)

        by Ingenium13 ( 162116 )

        My guess is the delivery mechanism. Retroviruses can be very specific, and affect just the targeted cells. Whereas with CRISPR it will also modify cells unnecessarily. Seems to be a lower risk profile.

        CRISPR I believe is more for editing existing genes, so if you have a "dominant" mutation causing the disease, then CRISPR is a better course of action because you need to modify that copy. But if the disease is caused by a recessive mutation (ie, usually non-functioning), then all it needs is a working copy i

      • Any reason you would continue to use retrovirus techniques over CRISPR once the latter is more understood?

        A retrovirus is used to modify the DNA of an existing organism while CRISPR is only good for modifying a single strand of DNA. You would need to modify the DNA of the child before it was born.

  • Can they pass it on? (Score:3)

    by Opportunist ( 166417 ) on Friday April 19, 2019 @08:30AM (#58459012)

    What if they donate blood or fuck someone?

  • It's heartless, but... (Score:3, Interesting)

    by sanosuke001 ( 640243 ) on Friday April 19, 2019 @08:42AM (#58459058)

    It's heartless, but is this actually a good thing? Passing along very bad genetic disorders that are rare now but, if more people live with them and have children of their own, will cause it to become more commonplace?

    I'm glad these parents' children didn't die from their disease, don't get me wrong, but a future where nobody is born with an immune system seems problematic...

    • If the gene is fixed on the germline cells the bad gene wonâ(TM)t be inherited.

      • That is what I didn't catch (maybe I missed it in the summary or article?). It just sounded like they fixed the bone marrow, not the DNA itself.

    • Come now - just think of the profits available in such a future! Why do you hate capitalism?

      Seriously though - if this were a common thing I'd be concerned. However, the genetic fan-out from such a small population, who will presumably know that their children are likely to be born with an expensive deadly disease, will probably be quite low.

      As the number of such treatments for various genetic diseases increases though it is certainly worth considering. There are a few obvious solutions though - first, i

  • So one question I have about this therapy that maybe someone can answer for me is where do these genes end up? I know in some gene editing techniques the replacement DNA is targeted to a specific region, but does HIV actually do this or does it just inject the genes wherever it gets a chance? I'm just curious if these sequences end up on the "usual" chromosomes or if they just get placed randomly, which in this kind of therapy isn't important (if I understand correctly) but could make a difference with fut

    • Re: (Score:2)

      by ceoyoyo ( 59147 )

      Retroviruses don't stick their genes in at random, they have definite preferences. There are off target effects with any gene editing technology though.

      Note that they didn't just inject modified HIV into a person. They extracted immune stem cells, modified them in a dish, then injected them back into the kids.

  • That's what interests me the most here: since they used a modified HIV to accomplish this, are these kids now immune or at least resistant to HIV?

  • This is for Leukaemia -- which is another blood based illness -- but the technique is the same:
    https://xkcd.com/938/ [xkcd.com]

  • I was thinking that Herpies might be worth looking into ?

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