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Medicine

Startup Tests Drugs Aimed at Autism 171

Posted by kdawson
from the whose-x-you-callin'-fragile dept.
An anonymous reader sends in this link from Technology Review about a startup company testing drugs that may help those with autism-spectrum disorders — even adults. "Seaside Therapeutics, a startup based in Cambridge, MA, is testing two compounds for the treatment of fragile X syndrome, a rare, inherited form of intellectual disability linked to autism. The treatments have emerged from molecular studies of animal models that mirror the genetic mutations seen in humans. Researchers hope that the drugs, which are designed to correct abnormalities at the connections between neurons, will ultimately prove effective in other forms of autism spectrum disorders. ... The company is funded almost entirely by an undisclosed family investment of $60 million, with $6 million from the National Institutes of Health. [A spokesman] says that Seaside has enough funding to take its compounds through clinical testing and approval."
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Startup Tests Drugs Aimed at Autism

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  • Re:Assumption much? (Score:2, Interesting)

    by Gerafix (1028986) on Monday January 11, 2010 @06:15AM (#30721196)
    Whether it's cemented at "birth" is beside the point of this drug as it attempts to correct a current state not prevent one. They claim it works on adult animals they have tested. RTFA? Nah this is /. lets just make assumptions.
  • Re:Assumption much? (Score:4, Interesting)

    by scapermoya (769847) on Monday January 11, 2010 @06:27AM (#30721232) Homepage
    lol. people on here can be such punks sometimes...
    i probably should have elaborated my point. what I meant was that it is entirely possible that autism is the result of a developmental process that occurs before birth. the animal models you mention are not of autism itself, but of fragile X syndrome. TFA says that the syndrome is associated with less than 5% of autism.
    the key point is, "While it's not yet clear if there is a critical window during development for giving the drug, adult animals still benefit from the treatment." There is no evidence yet that this will translate to any effect on autism, even in those with fragile X.
    so before you mouth off next time, RTFA yourself.
  • Side effects? (Score:5, Interesting)

    by Lord Lode (1290856) on Monday January 11, 2010 @06:53AM (#30721328)

    If someone has some form of autism making him extremely good at something (music, math, extreme memory, collecting stamps, ...), would this medicine affect his ability to do that?

  • Backward what-if (Score:3, Interesting)

    by macraig (621737) <mark.a.craig@g[ ]l.com ['mai' in gap]> on Monday January 11, 2010 @07:11AM (#30721414)

    You asked the question backward, buddy: if autistic traits as a package are all so very bad, then why weren't they weeded out of the gene pool millennia ago? Why is there a persistent trail of autistic achievers from Archimedes to Grigory Perelman and Craig Newmark? Why have the traits not only persisted but seem to be increasingly prevalent? If the multiple reports showing a statistical increase in autistic traits have any merit at all, that would seem to suggest that indeed there is an INCREASING value or merit to at least some of the traits, if not the whole. Natural selection may in fact have been working slowly to weed out the (currently) neurotypical. Perhaps a congested world of 6.5 billion people with an altered environment is favoring a package of mutations that are called autism, and accelerating the prevalence of those mutations?

    Bye-bye, neurotypicals... it's about time. We're tired of you disturbing our circles!

  • Re:What if (Score:2, Interesting)

    by Anonymous Coward on Monday January 11, 2010 @07:11AM (#30721418)

    Speaking as someone with Asperger im inclined to say:
    Im not sick. I just have different set of tools by which i precieve and communicate with.
    Problems arise when my tools try to interface with "normal" tools.

  • Re:What if (Score:5, Interesting)

    by Paul Jakma (2677) <paul+slashdot@jakma.org> on Monday January 11, 2010 @07:35AM (#30721534) Homepage Journal

    We know what would happen: Far more people would suffer from complications of diseases, such as male sterility from rubella, some would even die. No cases of autism would be prevented however, because there is no known link between vaccines and autism. This is what happened in the UK when MMR vaccination rates dropped dramatically after an idiot made up evidence and the study was published in the Lancet.

    See the link in my reply to your parent.

  • Re:What if (Score:5, Interesting)

    by bcmm (768152) on Monday January 11, 2010 @07:48AM (#30721588)

    If that were the case then natural selection would have taken its course long ago and we'd all be autistic. But it's an amusing question to philosophize nonetheless.

    More seriously, what if high-functioning autism was a somewhat beneficial trait for a few individuals, provided not everybody in a community was like that, and natural selection has formed the balance we see now? After all, science and technology has been advanced significantly by people who now seem autistic more than once.

  • Re:Assumption much? (Score:4, Interesting)

    by scapermoya (769847) on Monday January 11, 2010 @08:00AM (#30721648) Homepage
    "Whether it's cemented at "birth" is beside the point of this drug as it attempts to correct a current state not prevent one. They claim it works on adult animals they have tested. RTFA? Nah this is /. lets just make assumptions."

    looks like we're going to have to do a close reading here, for the sake of your education.

    In your first clause of your first sentence, you directly refer to my comment about the possibility that autism may be "cemented at birth," (meaning that regardless of which small molecules you give to someone with autism caused by fragile X syndrome, there will be no effect). You therefore made it clear that you were also talking about autism caused by fragile X, and not simply fragile X itself.
    In your second clause (where your main misunderstanding of the facts/developmental biology seems to lie), you state that there is a distinction between 'correcting a current state' and 'preventing one.' The main mistake you are making here is connecting the mGluR5 receptor with autism. This connection appears nowhere in the article, and is likely the result of you reading too fast. Your second sentence continues with this incorrect idea. You correctly point out that the mGluR5 inhibitors appear to have reduced some non-autistic symptoms in adult mice. However, because your original statement was about autism, not fragile X (because my statement was about autism, not fragile X generally, and you were responding to me), you committed a logical mistake.

    i'll state it again, just for you. there is no evidence that the seizures and protein synthesis abnormalities seen in animal models of fragile X are causationally related to autism. a small fraction of autism cases in people appear to be linked to a gene that is upstream of the mGluR5 receptor, but that definitely doesn't mean that the drugs that antagonize the receptor will have any effect on autism. again, even if this receptor does play a role in autism, it could be at a specific developmental stage, making the drugs useless for treating the disease in people. that is what i meant by "cemented at birth."

    and you did extrapolate. let's detail it for you. you made the assumption that because these mGluR5 antagonists reduced some neurological symptoms in animal models, that autism would be similarly affected. granted, you never said this explicitly (perhaps you were too busy insulting me?). the context of your comment makes it crystal clear though. by responding to my post about autism, you made your comment about autism too. and you mistakenly said that the drugs mentioned in the article, "attempts [sic] to correct a current state not prevent one." In the context of autism, this is not true in the least.
    feel free to keep it coming though. me and my degree in molecular and cell biology have all night.
  • Re:What if (Score:1, Interesting)

    by Anonymous Coward on Monday January 11, 2010 @09:35AM (#30722132)

    And squalene too...funny how no one ever complains about the squalene in their olive oil or naturally occurring in the human liver [who.int], but when there's squalene in vaccines the anti-vaxxers can't shut up about it. Kind of funny when you think that the anti-vaccination crowd and the alternative medicine crowd seem to have strong connection, and the alt-med folks love all things 'natural and wholesome,' including, of course, olive oil. That's the arrogance of ignorance for ya.

    It's not the squalene, or the mercury, it is the vaccines. Get rid of squalene, and whatever else, and they'll find a new 'problem.' That's what happened with mercury in the MMR vaccine. They ditched the mercury, and people found some other chemical to complain about (aka moving the goalpost). The anti-vax crowd won't be happy until the syringe is filled with water, and even then, they'll only be satisfied so long as you don't call it something scientific (because scientific=scary), like dihydrogen monoxide.

  • Re:Side effects? (Score:3, Interesting)

    by JasterBobaMereel (1102861) on Monday January 11, 2010 @10:19AM (#30722676)

    As I said Autism is not simple, assuming the range of Autism Spectrum disorders are all part of the same thing (which not everyone agrees on) then the range of causes is huge, and the range of effects is also huge

    This is a possible cure for one actual genetic disease (Fragile X) in some people along with the normal symptoms it can cause some autism spectrum symptoms, this may if it works at all alleviate some of the symptoms and it may alleviate autism if that was one of them.... note the large number of maybe's possibly's in that, and this is only one relatively rare cause of autism and they are not really trying to cure autism in this case, it is just one of a number of symptoms ...

    If you count the full spectrum of Aspergers (right down to so mild it is almost impossible to diagnose) then a large proportion of the population has autism ....

  • Re:Do I have it (Score:3, Interesting)

    by tigre (178245) on Monday January 11, 2010 @10:26AM (#30722776)

    I think you have a high bar for what a "meaningful" level of ASD would be. My son is (as far as the spectrum goes) very high functioning, but it's impact is tremendously meaningful. And my experience is that this end of the spectrum is not a small group at all. It seems to be the broader end of the spectrum, at least as current diagnostic trends seem to me to indicate. As for the clinical relevance, certainly odds are small that this particular drug will be of use for a wide range of ASD sufferers, but I think progress on one aspect of the spectrum at least fills out the picture of this poorly understood class of disorders.

    I don't think medical science can "define" autism as uncurable, though it might currently list it in that category. I disagree that the concept of curing it is nonsensical, but it would certainly be along the lines of "curing" amputation, i.e. it would take some serious neurological rewiring to accomplish what could reasonably be considered a cure. And that is certainly beyond the pale of current medicine, but at least for the milder cases like my son's, I have some hope that (should he need and desire it) such a treatment would be available within his lifetime.

  • Re:Side effects? (Score:3, Interesting)

    by netsavior (627338) on Monday January 11, 2010 @12:11PM (#30724054)
    I have been "autistic" my whole life (diagnosed, by a real doctor (1983), confirmed by several therapists(1986, 1990, 2005) )
    I also have a very high IQ and an eidetic memory for relative location (like puzzles and spatial problems) and conversational dialog (also tested by actual professionals).
    I struggled with depression as a teenager, and I can tell you that Prozac is my kryptonite. I struggled at math and even basic reading while on the happy pills. I decided I would rather be sad and smart, then happy and dumb, and then by the time the awful joke that humanity plays on itself "adolescence" was over, the depression was resolved anyway.
    I can't write code or solve problems when I am drunk or on happy pills or on pain pills (have tried on all 3) and it is infuriating. People always assume I am some moralist because I abstain from alcohol, and I have found that "I don't drink because it makes me feel stupid" does not go over well.
    I have extreme fear of that exact scenario, because everything I have ever done to alter my mental state has resulted in profound temporary loss of unusual abilities that I use like a survival crutch, make my living off of them, build my relationships around them, etc.
  • Re:Do I have it (Score:5, Interesting)

    by infinite9 (319274) on Monday January 11, 2010 @01:40PM (#30725336)

    Being crap with people suggests some form of social, behavioural, or anxiety disorder. ASD is a serious disorder with serious consequences. Rainman does not exist. As a rule of thumb, if you can put together a fully formed sentence, you almost certainly don't have meaningful levels of ASD. If you can read facial expressions without spending years actually consciously memorising what faces mean what, you don't have meaningful levels of ASD. Okay, if you've gotten this far you might have comparatively mild Asperger's or something on that end of the spectrum, but it'll be clinically relevant only in a small fraction of a percent of that already small group.

    I have Asperger's Syndrome. Two of my biological children also have it. And a son we adopted from Russia has high functioning Autism. All of us have an autism spectrum disorder. All of us can put together fully formed sentences. The ability to form sentences is largely irrelevant with respect to autism spectrum disorders. High functioning autism kids have a speech delay that they overcome fairly early in childhood. But that's about it.

    Here's a metaphor what learning social skills is like for people with AS : The entire world communicates by playing the piano. 99% of the people out there are born knowing how to play the piano and can simply walk up to a piano at age 2 and start playing. Some are better than others. But most people can play the piano very well. I was born without being able to play the piano. I can learn it. But it's going to take me years. And a lot of variables will affect how fast I learn it and whether I learn it correctly. Am i an introverted shut-in who never seeks piano lessons? If so, I'll never learn it. Am I extroverted (but constantly making mistakes) and always trying to learn from as many piano teachers as possible? I may learn it faster and eventually play very well. Innate ability matters also. Some normal people are naturally good at the piano (social skills) while other normal people are not. What would the AS person have been had they not had AS? This affects things as well.

    It's not so much about memorizing facial expressions, but that's part of it. It's more about memorizing prerecorded behaviors and verbal responses for every possible social situation imaginable. Smile at a wedding... don't smile at a funeral... crying at a wedding doesn't equal sad... someone can be sad but not crying... there's a million combinations.

A motion to adjourn is always in order.

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