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Biotech Science

How A "Superbaby" Is Helping To Find Muscular Dystrophy Treatments 93

Posted by ScuttleMonkey from the hercules-hercules dept.
An anonymous reader writes to tell us that a baby boy with unusually big muscles — caused by a gene mutation — is helping scientists to discover new muscular dystrophy drugs. "Myostatin was discovered in mice in 1992 in Lee's Johns Hopkins lab. In 1996 he proved its importance by showing that mice without the myostatin-producing gene got twice as big. The next year he discovered that the bulging Belgian Blue cow was a myostatin mutant, the first of eight prized cattle breeds later found to have the mutation. The company he had co-founded, MetaMorphix, is working on manipulating myostatin to beef up livestock. Wyeth picked up the rights to develop a drug for humans. Its experimental antibody drug produced bulked-up mice in 2002, and results of a trial in adults with muscular dystrophy are expected as early as March."
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How A "Superbaby" Is Helping To Find Muscular Dystrophy Treatments More

How A "Superbaby" Is Helping To Find Muscular Dystrophy Treatments

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  • Cool, but no silver bullet. (Score:5, Informative)

    by Short Circuit ( 52384 ) * <mikemol@gmail.com> on Monday February 19, 2007 @10:46AM (#18067656) Homepage Journal
    This is actually pretty cool. I have a step-mother and a half-brother with muscular distrophy.

    It won't be a silver bullet, though. His disability is so bad that he's never been able to talk, much less walk on his own. Some of his joints have essentially locked up due to disuse. Even if the treatment were available today, he'd still have to learn how to talk. He might even need knee-replacement surgery before he could start learning to walk.

    At least he'd be able to feed himself, though.

    • Re: (Score:3, Funny)

      by garcia ( 6573 )
      But would it be a "silver bullet" (I keep imagining a Vampire drinking a Coors Light) for those that are treated early?

      • Re: (Score:2, Interesting)

        by Short Circuit ( 52384 ) *
        Possibly. And it would be effective for people like my step-mother, whose only problem is a general lack of strength.
    • coochee-coo - please don't squeeze my hand into mashed pulp.

    • Still a great relief for me (Score:5, Interesting)

      by BlackCobra43 ( 596714 ) on Monday February 19, 2007 @11:00AM (#18067804)
      I have an offshoot of muscular dystrophy. Any progress towards a cure brings me that much closer to being able to perform regular physical activities and... you know..not being guaranteed to die way earlier than anyone should. I've been following this possible cure since the tests in mice and I think it shows great promise. It's sad that it is already too late for many people (in fact, it may be too late for even myself before this cure is commercialized..my strength already wanes) but I'm just happy that there is a chance that others won't have to live my pain.

      • Re: (Score:3, Interesting)

        by ylon ( 656206 )
        I have a friend with some sort of offshoot it seems as well. I can't recall the name of the type that he has, but he lives a very difficult life in an assisted living facility. I really don't know what to do to help him as the doctors are essentially performing euthanasia by giving him high doses of lithium for his mental issues that are being caused by this disease and his difficult life. I really want to help him and I'm trying help get a site started that is pro alternate solutions/treatments for musc
      • Don't count yourself out yet. As a fan and student of the human body, I keep up with a lot of stuff like this, and it might surprise you to know that there is in fact a myostatin inhibitor that has recently undergone testing, and scientists are currently analyzing the results. [mda.org]

        Positive results could have this thing to market quickly, considering its intent is to treat terminal patients in the first place.

      • Re: (Score:1)

        by philwx ( 789834 )
        We're with you in spirit, I know how lonely it can feel when you are seriously ill. God bless, or if you prefer, may the cosmic number generator output favorably for you.
    • Schwarzenegger's love child?
    • See, it's stuff like that...that's why I'm of two minds about these "discoveries". On one hand, this kid totally deserves a normal life. On the other hand, so do people like your step-mother and half-brother. I'm mostly thinking out loud here....It would be wonderful if they could study this kid without constantly "studying" him. Find out what, if any, benefits his body gave him. On the other hand, his muscles may end up to be his own debilitation. Which would, in itself, be sad.
    • When is Captain America getting it?

  • Muscles are attractive (Score:5, Funny)

    by kestasjk ( 933987 ) * on Monday February 19, 2007 @10:49AM (#18067676) Homepage
    Muscles are attractive, but these days muscles aren't more likely to increase the survival of your children, so how long might it take until they're no longer found attractive? You could argue that they might even be an unnecessary waste of resources, so might their attractiveness diminish?

    (Cue the /. jokes..)

    • Re: (Score:3, Funny)

      by fishthegeek ( 943099 )

      You could argue that they might even be an unnecessary waste of resources


      You must be an MCSE.

    • Re: (Score:3, Insightful)

      by account_deleted ( 4530225 )
      Comment removed based on user account deletion

    • Re:Muscles are attractive (Score:5, Interesting)

      by 0100010001010011 ( 652467 ) on Monday February 19, 2007 @11:02AM (#18067818)
      Get back to me when men aren't interested in breasts.

      That being said, I would say around 3-4 generations (90 - 120 years). There was once a time when a full figured woman with curves was 'attractive'. It showed you were child bearing and rich (since food wasn't as easy to come by.) In most cultures it still is in. It just seems to be in 1st world countries that 'thin is in'.

      Breasts however... are still in.

      • Get back to me when men aren't interested in breasts.
        That will be when we are extinct.

      • Re: (Score:2)

        by mrbooze ( 49713 )

        Get back to me when men aren't interested in breasts.

        You only have to look back to the 1920s for that. Flapper gals used to go to great lengths to minimize their breasts.

      • That being said, I would say around 3-4 generations (90 - 120 years). There was once a time when a full figured woman with curves was 'attractive'.

        You don't need to go back that far. Marylin Monroe was basically a size 16, many of her contemporaries were similarly sized. I think a size 14 is the cutover to being 'plus sized' or fuller figured.

        Hell, as recent as the last decade, Anna Nicole Smith was considered a sex kitten despite being of a more ample size than, say, Kate Moss. Obviously, Hugh Heffner s

        • Re: (Score:1, Informative)

          by Anonymous Coward
          Marylin Monroe was 118 pounds [wikipedia.org]. Even accounting for size inflation [wikipedia.org], she was NEVER a size 16. In fact, according to standardized clothing sizes [wikipedia.org] (which are completely ignored anymore, but were developed around the time Marylin was around), she would have worn at most a size 12 (based on her hips) and probably something closer to a 6 (based on her waist).
      • There was once a time when a full figured woman with curves was 'attractive'. It showed you were child bearing and rich (since food wasn't as easy to come by.) In most cultures it still is in. It just seems to be in 1st world countries that 'thin is in'.

        Personally...I find "full figured woman with curves" MUCH more attractive than any woman who's skinny. Part of this is that not only are men & women getting bigger...but by & large...anyone 35 & over is going to be "more curvy"...due to the

    • Re:Muscles are attractive (Score:4, Interesting)

      by vadim_t ( 324782 ) on Monday February 19, 2007 @11:14AM (#18067920) Homepage
      Actually, waste of resources is precisely what's considered to be attractive.

      Consider for example various competitions between animals: Who has the largest horns, who has the biggest and prettiest tail, etc. There's some breed of bird where the male creates a display of various pretty things to attract a female. Pretty much all that can be reduced to "If I could find the time and energy to haul all that stuff around, then obviously I'm a good mate, and you want my genes"

      In human society, expensive clothes are appreciated. When being fat meant that you had enough resources to waste, being fat was attractive. Now that effort is required to be slim instead (to the point of it costing more than being fat) being slim is attractive.

    • Re: (Score:2)

      by spun ( 1352 )
      A peacock's tail is also an unnecessary waste of resources, yet peahens seem to find it attractive. Sometimes, a waste of resources is a kind of brag that says, "See, potential mate, I am so fit that I can take on this handicap and still survive."
    • There's actually a fairly big trend in the fashion industry with thinner male models, especially runway. Look at a Prada or (some) Dolce & Gabbanna runway shows. Prada is a prime example and has been using the geeky-thin but kinda toned look for some time and it's been gaining popularity. Granted we may not see the thinness of male models equal the desired thinness of the female ones, but it's starting to balance out somewhat I think. Androgyny is pretty big.

  • Unbreakable (Score:2, Informative)

    by FredDC ( 1048502 )
    Reminds me of the movie Unbreakable [imdb.com].
  • Here [1fast400.com]. This is old news. There are even myostatin blockers sold as "dietary supplements" [bodybuilding.com].

    • Re: (Score:1, Insightful)

      by Anonymous Coward
      But the dietary supplements don't work anywhere near as well as whatever happened to the baby, and we're not allowed to go around punching random genes out of babies, which is why the baby is news.
      • The supplements don't actually work at all. There are currently no myostatin blockers available that actually work, or many, many more people would know about them.

        And he's right about the baby not being news. It was born in 2000 and people in the industry have been keeping an eye on it since near-birth.

        This is just a case of Forbes picking up on an old story that not many people knew about and sensationalizing it.
      • The dietary supplements are, afaik, fake. The kid was big news years ago and I think that was what spawned the market for them.

        Here's [msn.com] a story from 2004 with a pic of the kid from when he was 7 months old.
      • You are allowed to randomly slap genes together, but you're supposed to do it using conventional IVF or sex, not recombinant technology.
    • Yes, old news. And we also know that the research is going on fast because of the many billion$ from the BodyBuilding world (just note that trying to win a pro BB contest costs something like a small car, and a *lot* of amateur BBers spent a *lot* of money every year just to be big)
  • "In 1996 he proved its importance by showing that mice without the myostatin-producing gene got twice as big."

    • A later study will find that Gamma rays somehow affected the myostatin mutations.

      2 new breeds of super-cow await... German Grey and Georgian Green...

      • Re: (Score:1)

        by Barryke ( 772876 )

        A later study will find that Gamma rays somehow affected the myostatin mutations.

        2 new breeds of super-cow await... German Grey and Georgian Green...
        Green? That'd be the Georgian Hulk.

  • How long.. (Score:5, Insightful)

    by zyl0x ( 987342 ) on Monday February 19, 2007 @10:57AM (#18067766)
    ..until this kind of gene therapy is available on the black market or to the general public? Maybe the Olympics will have to start doing genetic tests for enhanced performance genes. Kinda weird to think of it that way.
    • It's already on the table, I just wrote a paper on this topic last year. Various "gene doping" techniques have not been very effective for athletic performance as of yet, but it's not far away.
      • Ok. Since the baby [slashdot.org] occurred naturally (the mutation is a natural occurance), will anti-doping agencies eventually be forced into a position of testing and disqualifying athletes because their natural genetic makeup gives them an 'unfair' advantage? Will this child, and his progeny, be barred from NCAA / Olympic / whatever athletic competition? Is such a ban even legal under US/EU anti-discrimination laws? Could they survive losing the subsequent lawsuits?
        • The way past this is to monitor someone's levels of various things throughout their entire athletic career. If something gets too far out of whack beyond medical tolerances, then they're a doper. The other, even easier way that still works right now is that artificial genes seem to leave a different um, "fingerprint" I guess. Of course, that's something that could go away over time I'm sure, as the technique is still in its infancy.
          • Athletic competitions are about the performance and physical skill humans can achieve through natural means. That includes inherent genetic advantages.
          • Nope...

            I'm referring to the anti-doping agencies banning people because of their natural genetic makeup, not because of something they've taken / injected / eaten / whatever.

            Imagine a near future world where the german child (or any of his progeny) are told, "You cannot compete in our games because you were born with forbidden genes. They give you an unfair advange".

            The mind boggles.

    • People have been talking about this in athletic newgroups for several years now. The scam artists are already selling "myostatin inhibition" herbs. And the real biochemists are working furiously on a product, since it will useful for various dystrophic diseases too.

    • Re: (Score:1, Informative)

      by Anonymous Coward
      What is really scary is you wanna know who was really interested in myostatin? Victor Conte...the BALCO guy who was involved in the whole Barry Bonds steroid scandal and did jail time for it. He actually published papers on the topic. Not saying that they ever came up with anything useful, but these were the people who came up with "The Clear" which was undetectable by steroid screens.

      http://en.wikipedia.org/wiki/Tetrahydrogestrinone [wikipedia.org]
    • I can provide some insight into this. The article is pretty outdated. This "superbaby" has been around for a couple of years and has gained attention in the bodybuilding communitiy. There is currently a market for myostatin inhibitors http://www.bodybuilding.com/store/myostat.html [bodybuilding.com].

      Most people know that these products are scams, but it shows that the news has been around.
  • amazing!

    Bonus to whoever figures out what I am referencing :P

  • Unintentional Comedy? (Score:3, Interesting)

    by baldass_newbie ( 136609 ) on Monday February 19, 2007 @11:01AM (#18067814) Homepage Journal
    From the synopsis:

    ...is working on manipulating myostatin to beef up livestock
  • When he grows up, that baby might be the one who'll finally be able to take on Chuck Norris [chucknorrisfacts.com].
  • FLASH: Steroid Market Craters

  • Moo (Score:1)

    by Chacham ( 981 )
    His name isn't clark, is it?
  • If a lack of myostatin, whether natural or due to some type of therapy, allows rapid muscle growth what effect does it have on your heart or your tongue? An enlarged tongue could cause all kinds of breathing and eating problems. An unusually large heart could cause any number of problems resulting in death. From the little bit of reading I have done so far none of the research data I have seen addresses these issues.

    • Re: (Score:3, Informative)

      by eli pabst ( 948845 )
      what effect does it have on your heart or your tongue?
      It's my understanding that myostatin is only expressed in skeletal muscle, so cardiac muscle would be unaffected by a myostatin inhibitor.

      I'm not an expert on it, but my lab has done a lot of research on myostatin and has identified some of the mutations in humans (including some cool papers looking at mutation prevalence in world class body builders).

      This might be informative reading for you: http://www.ncbi.nlm.nih.gov/entrez/dispomim.cgi?id [nih.gov]
      • Thanks for the link. I also found This Article [mesomorphosis.com] which discusses myostatin expression in other tissues including cardiac muscle. It seems that depending on the species and the test subject's stage of development myostatin can have positive or negative effects on not only cardiac muscle but also completely non-muscular tissues within the body.
  • Great. Exactly what we need.
    More 'miracle' foods to "solve" our 'problems.'

    When will people stop trying to invent the damned golden rice and actually focus on the real problems?
    • Man, if someone made golden rice that you could eat and tasted good and made you build healthy muscle while not being full of calories, I think that might actually solve the real problem, unless you're thinking something else is "the real problem".

      Would you look down on people who ate the golden rice to be healthy because they didn't have to deal with diet and exercise?

    • Re: (Score:3, Insightful)

      by DrKyle ( 818035 )
      Real problems? I don't think we geneticists can solve the world's real problems, so we will just try and do what little we can to help out. Making more nutritious food won't fix fucked up foreign policies that deal with food aid or make the supply chain more efficient, that's the problem of people who work in those industries.

      It's like saying OLPC shouldn't exist because it doesn't solve the core problems of the third world, it will only help children get more education. It may not fix the problem, but

  • Baby pic! (Score:4, Informative)

    by bgarcia ( 33222 ) on Monday February 19, 2007 @11:45AM (#18068232) Homepage Journal
    Picture of the baby: link [msn.com]
  • Insert here some random joke about mutant superheroes.

  • Superbaby (Score:2, Funny)

    by Wiarumas ( 919682 )
    I, for one, welcome our new superbaby overlord.
  • This boy is just one of countless individuals who belies the creationist refrain that mutations never observably add "information" to a genome, or positive effects. This is a particularly large effect mutation, which is not the general case for what I'm talking about, but like the family with unusually dense bones, tetrachromatism in women, immunity to LDH cholesterol toxicity, and so on, its just one more example of how there are countless changes going on in the human gene pool even in very recent histor

    • Re: (Score:2)

      by d3ac0n ( 715594 )
      Except that this mutation does NOT add new information to the genome.

      If you had RTFA you would have seen that the mutation occurs when the gene that is responsible for producing the chemical Myostatin is NOT present in the genome. So, not only is your post rude, inflammatory and trollish, but it's just plain WRONG.

      • Re: (Score:2)

        by plunge ( 27239 )
        You don't know what information is, do you? Adding and subtracting genes is not the same thing as adding or subtracting information.
  • It's just AH-Nold in a diaper....

    The things a guy will do to get some attention... :-P
  • I have read some of these comments on this website and truthfully, I find them upsetting. You see, my 6 year old son has a diagnosis of Duchenne muscular dystrophy. Do you know what he has to look forward to in his future? Let me just tell you about some of the few. He gets to have a one on one aid in school because he won't have the strength to toilet and wipe himself. He probably will never have a relationship with a woman much less marry one and have children. He will end up needing someone to dress a
    • Birthdays have a whole new meaning. Truthfully, these are kind of sad days for us. It is just another year closer to becoming whellchair bound.

    • Re: (Score:1)

      by tqphan ( 1066234 )

      He probably will never have a relationship with a woman much less marry one and have children.

      Sounds like he'll fit in the /. community just fine. On a serious note, I don't find the comments here offensive at all. Who am I to say? Well, what you just describe is me after 27 years of SMA Type 2 http://www.mda.org.au/specific/mdasma.html [mda.org.au]. Be thankful your son could walk for the first decade of his life. Anyway, /. wouldn't be the same without the "In the Soviet Russia, MD cures you," "Can you imagine a Beowulf cluster of these?" "I have MD, you insensitive clod." Haha it's funny because it's t

      • Sorry, I don't mean to sound clinically depressed but none of this is funny. "sounds like he'll fit in the community just fine." Don't you think I want to dance with my son at his wedding? He will be robbed of one of the many beautiful things that life has to offer and this includes growing up to be a tall handsome man( srteroids stunt the growth), meeting the girl of his dreans and marrying her one day and producing children. "Be thankful your son could walk for the first decade of his life?" Hey, I better
  • This reminds me of the central premise of Unbreakable... that for every degenerative disease, there can be a (much rarer) mutation which is the opposite.
    • FTFA:

      He's strong but no longer abnormally so. It may be that myostatin's effects are most dramatic during fetal development

      What's weird is with Belgian Blue and Piedmontese cattle(2 breeds with the genetic "defect") the calves are born no more muscular than other breeds. However, as they begin to mature the extra muscle then packs on. Apparently opposite from this human example.

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