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Stem Cell Treatment Found Effective For Rare Brain Disorder

Posted by samzenpus on from the we-can-grow-him-better dept.

sciencehabit writes "Four young boys with a rare, fatal brain condition have made it through a dangerous ordeal. Scientists have safely transplanted human neural stem cells into their brains. Twelve months after the surgeries, the boys have more myelin—a fatty insulating protein that coats nerve fibers and speeds up electric signals between neurons—and show improved brain function, a new study in Science Translational Medicine reports. The preliminary trial paves the way for future research into potential stem cell treatments for the disorder, which overlaps with more common diseases such as Parkinson's disease and multiple sclerosis."

28 of 43 comments (clear)

  1. Question We're All Afraid To Ask by Anonymous Coward · · Score: 1

    show improved brain function,

    Will this treatment ever be effective on politicians?

  2. Once again by azcoyote · · Score: 3, Interesting

    Adult stem cells FTW. Embryonic stem cells are not necessary.

    --
    Incipiamus, fratres, servire Domino Deo, quia hucusque vix vel parum in nullo profecimus.
    1. Re:Once again by kribby · · Score: 5, Insightful

      That might be true, but the scientific community should never have their hands tied behind their back because of religious superstitions.

    2. Re:Once again by EvolutionInAction · · Score: 1

      Except for the whole 'only works for a rare brain disorder' bit you are correct!
      We can't learn how to fix ourselves if we use crippled cells instead of known good ones.

    3. Re:Once again by gr8_phk · · Score: 1

      Adult stem cells FTW. Embryonic stem cells are not necessary.

      Yes, but ever since "fetal stem cell research promotes abortion" there is really no distinction in politics. It will take a long time to brake the "stem cells = evil" connection in peoples minds - republicans in particular if you really want to stir the pot.

    4. Re:Once again by reverseengineer · · Score: 3, Informative
      These happen to only be "adult stem cells" in the sense that they are not totipotent embryonic stem cells (cells from the very earliest stages after fertilization that can differentiate into any cell type), but I'd like to point out that doesn't mean they come from an adult brain. They're only "adult" by the meaning of having matured to the multipotent stage (neuronal stem cells can differentiate into neurons or glia, but not muscle cells or liver cells, for instance). The biotech company that provided these cells, StemCells, Inc. cultures them from donated fetal brain tissue.

      The first production step comprises a proprietary method for purification of HuCNS-SC cells from donated fetal brain tissue procured from an FDA-registered, not-for-profit agency, in compliance with Good Tissue Practice (GTP) and all other applicable state and federal regulations. As part of the purification process, cells from the tissue are “tagged” with a monoclonal antibody that recognizes human neural stem cells. High-speed Fluorescence Activated Cell Sorting (FACS) is then used to isolate the cells tagged by the monoclonal antibody. The FACS-isolated HuCNS-SC cells are then placed in cell culture.

      --
      "FDA staff reviewers expressed concern about the number of patients who were left out of the study because they died."
    5. Re:Once again by Chrutil · · Score: 4, Insightful

      And the government should never take money from people to pay for something they're religiously opposed to.

      NOTE: I'm not arguing with parent, I'm saying that both his statement and mine are how it should be.

      So no tax can fund school where girls attend for a Taliban?

    6. Re:Once again by DNS-and-BIND · · Score: 1

      "The future must not belong to those who slander the prophet of Islam."
      -- President Obama, addressing the United Nations General Assembly

      --
      Shutting down free speech with violence isn't fighting fascism. It IS fascism!
    7. Re:Once again by Yoda222 · · Score: 1

      I'm religiously opposed to the concept of government, to roads, TSA, CIA, NSA, FBI, ...

    8. Re:Once again by the+gnat · · Score: 1

      How about the full quote?

      "“The impulse towards intolerance and violence may initially be focused on the West, but over time it cannot be contained. The same impulses toward extremism are used to justify war between Sunni and Shia, between tribes and clans. It leads not to strength and prosperity but to chaos. In less than two years, we have seen largely peaceful protests bring more change to Muslim-majority countries than a decade of violence. And extremists understand this. Because they have nothing to offer to improve the lives of people, violence is their only way to stay relevant. They don’t build; they only destroy. [...] The future must not belong to those who slander the prophet of Islam. But to be credible, those who condemn that slander must also condemn the hate we see in the images of Jesus Christ that are desecrated, or churches that are destroyed, or the Holocaust that is denied.”

    9. Re:Once again by DNS-and-BIND · · Score: 1

      That's nice, but does nothing for "the scientific community should never have their hands tied behind their back because of religious superstitions." This quote is anti-Islamic, and Obama is quite clearly supporting religion. So...go ahead and try to reconcile the two. I'll wait.

      --
      Shutting down free speech with violence isn't fighting fascism. It IS fascism!
    10. Re:Once again by parkinglot777 · · Score: 1

      Unlike DNS-and-BIND who gave only a partial quote for a purpose (similar to many political ads), you did a great job on giving the whole truth. Should mod yours up and mod the GP down (as flamebait).

    11. Re:Once again by interkin3tic · · Score: 1

      Except for research into cellular differentiation, where there is no substitute for ESC yet. You want to heal a sick person's liver, yeah, ESC were never the way to go. Unless you're cloning the patient, you'd never find a suitable harvest embryo that their body wouldn't reject. You want to study how a liver cell gets MADE, you should be studying ESC. Or aborted fetuses. But many more people have ethical qualms about that.

    12. Re:Once again by parkinglot777 · · Score: 1

      This quote is anti-Islamic

      Which quote? You mean the "religious superstitions"? Excuse me, your political mind makes your mind so narrow. To me, it is just a plain comment to "any religions" that threatening science community and is not specific to Islamic.

      Obama is quite clearly supporting religion

      And another comment of yours that is blinded by your political view. You are looking at the circumstance and interpret it the way your politic is -- with your half-glass-empty attitude. One question to you in general, does this country (U.S.) have laws that said being an Islamic illegal? Or the laws said everybody in this country must not be an Islam? I don't say that what happened to the world from those lunatics from the religion is appropriate. What I want people to see is that the whole group of people should not be painted by only a portion that is bad -- stereotyping.

    13. Re:Once again by SleazyRidr · · Score: 1

      Shh. As long as we keep calling them "adult" the fundies will keep off our backs.

      --
      Is 1563649 a prime number?
    14. Re:Once again by GameboyRMH · · Score: 1

      Since the cover's blown maybe we can call them "muslim fetus stem cells" this time.

      --
      "When information is power, privacy is freedom" - Jah-Wren Ryel
  3. Flowers For Algernon by steelfood · · Score: 2

    These boys were said to have taken immunosuppressants for nine months before beinig injected with the stem cells. Given this, and that the disorder is genetic, I'm assuming the stem cells are from an external source.

    Since the stem cells are turning into neurons, I wonder how this will affect them in the future. Would the neurons remain without immunosuppressants? Or would the boys slowly lose these foreign cells growing up, and ultimately revert back to their original selves.

    The nervous sytem is a dangerous thing to manipulate. The effects could range from nothing to the boys taking on traits of their donor. While it's great stem cells can provide relief for this disorder, I hesitate to call it a cure. And if things go south later in their lives, it may very well be a curse.

    --
    "If a nation expects to be ignorant and free in a state of civilization, it expects what never was and never will be."
    1. Re:Flowers For Algernon by ColdWetDog · · Score: 4, Informative

      These boys were said to have taken immunosuppressants for nine months before beinig injected with the stem cells. Given this, and that the disorder is genetic, I'm assuming the stem cells are from an external source.

      From the Fine Linked Article, the stem cells were allogenic - ie, not from the patient.

      Since the stem cells are turning into neurons, I wonder how this will affect them in the future. Would the neurons remain without immunosuppressants? Or would the boys slowly lose these foreign cells growing up, and ultimately revert back to their original selves.

      Good question, likely they will be on suppressants the rest of their lives.

      The nervous sytem is a dangerous thing to manipulate. The effects could range from nothing to the boys taking on traits of their donor. While it's great stem cells can provide relief for this disorder, I hesitate to call it a cure. And if things go south later in their lives, it may very well be a curse.

      That's why they are doing this on an invariably fatal disease. They are going to die of this disease (and quite early on IIRC) without treatment. So it is considered a 'compassionate' protocol (not withstanding philosophical discussions on whether or not this really is a compassionate thing to do). So you get to do things that are much more dangerous than your average clinical trial. But this really is the only way to approach it - well, the only way our consistent with current ethical guidelines in the US.

      --
      Faster! Faster! Faster would be better!
    2. Re:Flowers For Algernon by reverseengineer · · Score: 3, Informative

      The stem cells aren't turning into neurons, actually, despite coming from "neuronal stem cells." The intent of the treatment is for the stem cells to differentiate into oligodendrocytes, which are a type of glial cell (which in turn are several types of cells that provide support functions to neurons). Oligodendrocytes are interesting cells because they wrap around neurons like insulation around a wire (which is exactly their purpose). These cells play an important role in nerve conduction and in overall brain function, but they're just tubes filled with fat.

      --
      "FDA staff reviewers expressed concern about the number of patients who were left out of the study because they died."
    3. Re:Flowers For Algernon by bluefoxlucid · · Score: 1

      In other words, we're potentially letting the retard babies breed, and in the mean time instead of dying they get to have medically induced AIDS (sans an actual HIV infection).

      I know Eugenics isn't popular in this country, but honestly, when it comes to "you have an inheritable genetic disorder that turns your life into an expensive shitfest," can we impose physical sterilization as a condition of treatment? I mean really, these people are a drain on the monetary economy by way of insurance (insurance pays out, meaning premiums must go up to cover risk--more money flowing out means higher risk in the business overall, which is covered by having a bigger cash reserve); they consume the resource of medical care (there isn't enough of it to cover everyone's needs if we made it free); and by breeding they are passing on genetic code that results in these same disorders, making their children carriers or worse (and because most people breed, it gets around and infects an ever-growing portion of the population).

      Think about it. Remove these people from the breeding pool.

      --
      Support my political activism on Patreon.
  4. FYI by Anonymous Coward · · Score: 2, Interesting

    The disease is early-onset Pelizaeus-Merzbacher disease.

    If you were new here, you might have thought that would have made it into the summary.

    1. Re:FYI by ColdWetDog · · Score: 1

      OK, just to be fair to the editors (this one time). If TF summary had mentioned early onset Pelizaeus-Merzbacher disease, would that have helped?

      I suppose they could have included the Wikipedia Link to it, but really, if you were at all interested, you could have done this yourself (I hope).

      The Internet - it's a wonderful thing.

      --
      Faster! Faster! Faster would be better!
    2. Re:FYI by Rich0 · · Score: 1

      The name of the disease with the link would have been helpful.

      Otherwise why don't we just use this summary for 90% of the articles out there:

      Researchers backed by an institution have just discovered a new technology. This technology promises to make lives better, and may be available to ordinary consumers soon. The story was published by a real news site with more details, but we're going to link to some guy's blog which you can read here...

      This might do for the other 10%:

      A politician just said something that is guaranteed to make you upset. Please discuss how this development is going to ruin your life. Oh, and try to forget that this is a news for nerds site...

  5. Re:Other uses... by Penurious+Penguin · · Score: 3, Funny

    You are hereby cordially invited to join me for a bucket of warm beer and wanton drooling. We will wear not but ducktape diapers and pressed Spam-helmets whilst watching the Nature Channel on mute. In the background will be Aphex Twin, all albums simultaneously played through a Marshall stack on max output. We will hold hands and use google-translate in attempt to dictate a Universal Access Linux box in a foreign tongue, such as Magyar or Uyghur. Our first task will be to nmap army.mil, followed by trolling on slashdot. Then the midgets will arrive, with cheap Scotch whiskey and starving badgers.

    If this sounds like your cup of tea, please make strange and unusual noises. I have very sensitive hearing and will approach you from behind.

    - Sgt Septic,
    Head Chief of many tanks, including T 34s, Panzers, and Septics.

    --
    Forward! -- Emperor Norton, 2012
  6. Is there anything they can't do?! by Infestedkudzu · · Score: 1

    I mean besides convince people to stop hindering research with them.

  7. Re:This Also Paves the way for Neural Upgrades by jamesh · · Score: 1

    And yes, I'm going to be cryonically suspended so that if the technology is not ready by the time I'm gone, I'll still be "around" so-to-speak to revive and apply the upgrades.

    Good luck with that. In the overcrowded world of tomorrow, i'll be voting no on "wake up Press2ToContinue" :)

  8. stem cell treatment by RedCharlie · · Score: 1

    My spouse is a genetic counselor at a children's hospital and actually has one patient with Pelizaeus-Merzbacher. It's "one of those really bad ones" as she put it. It's hopeful that such a treatment now exists, even if it's still very experimental.

  9. Can they grow me a Hypothalamus? by boltik · · Score: 1

    No cure for Aspergers? I'm back to my meaningless repetitive tasks.