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

Posted by ScuttleMonkey on 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."

12 of 93 comments (clear)

  1. Cool, but no silver bullet. by Short+Circuit · · Score: 5, Informative

    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.

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  2. Muscles are attractive by kestasjk · · Score: 4, Funny

    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..)

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    1. Re:Muscles are attractive by fishthegeek · · Score: 3, Funny

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


      You must be an MCSE.
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    2. Re:Muscles are attractive by 0100010001010011 · · Score: 5, Interesting

      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.

    3. Re:Muscles are attractive by vadim_t · · Score: 3, Interesting

      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.

  3. I bow down to the new super baby overlord by spicydragonz · · Score: 3, Funny

    coochee-coo - please don't squeeze my hand into mashed pulp.

  4. How long.. by zyl0x · · Score: 4, Insightful

    ..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.

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  5. Still a great relief for me by BlackCobra43 · · Score: 5, Interesting

    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.

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    1. Re:Still a great relief for me by ylon · · Score: 3, Interesting

      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 muscular dystrophy. Let me know if you'd like to help out at all by replying and I'll see what we can do to get things shifted into a higher gear.

  6. Unintentional Comedy? by baldass_newbie · · Score: 3, Interesting
    From the synopsis:

    ...is working on manipulating myostatin to beef up livestock
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  7. Re:Some concerns by eli+pabst · · Score: 3, Informative

    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 =601788

  8. Baby pic! by bgarcia · · Score: 3, Informative

    Picture of the baby: link

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