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Startup Tests Drugs Aimed at Autism

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."

6 of 171 comments (clear)

  1. Re:Assumption much? by scapermoya · · Score: 3, Interesting

    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.

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  2. Side effects? by Lord+Lode · · Score: 4, Interesting

    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?

  3. Re:What if by Paul+Jakma · · Score: 4, Interesting

    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.

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  4. Re:What if by bcmm · · Score: 4, Interesting

    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.

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  5. Re:Assumption much? by scapermoya · · Score: 3, Interesting

    "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.

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  6. Re:Do I have it by infinite9 · · Score: 4, Interesting

    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.

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