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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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  • i sure hope autism isn't something that is more-or-less cemented at birth, making drugs like these not very useful.
    • Re: (Score:2, Interesting)

      by Gerafix (1028986)
      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 @05:27AM (#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.
        • Re: (Score:2, Troll)

          by Gerafix (1028986)
          I never claimed that the drug was not targeted for fragile X, in fact only you mentioned the drug and autism in the same sentence. Perhaps you should reread the OP.
          • Re:Assumption much? (Score:5, Informative)

            by scapermoya (769847) on Monday January 11, 2010 @06:10AM (#30721408) Homepage
            here we go, buddy:

            the article is about a drug that targets a rare genetic trait. because the article appears in layman media and is remotely linked to autism, the submitter titled the /. story "Startup Tests Drugs Aimed at Autism," which is only mildly true.

            My original comment:

            "i sure hope autism isn't something that is more-or-less cemented at birth, making drugs like these not very useful."

            i was tacitly talking about the minority of autism cases linked to this fragile X syndrome, as evidenced by the fact that I was talking about "drugs like these." I was trying to make the point that, even though the drug has been shown to mitigate some of the symptoms of fragile X in adult animals, this tells us nothing about whether the drug will have an effect on autism. i.e. autism's link to fragile X could be completely unrelated to the symptoms of fragile X seen in the animal models (seizures, abnormal protein synthesis, etc). We have no idea what all the functions of FMRP are. anyone who says we do is a fool.

            it is entirely possible that mGluR5 has nothing to do with autism. it could simply be a receptor in a downstream pathway from FMRP, separate from whatever pathway(s) are involved with autism development. furthermore (getting back to my first post), even if this receptor is somehow involved with autism, it could be involved only at a very specific stage in development. thus, giving mGluR5 antagonists to people who have passed that stage would have no effect.

            thus your comment:
            "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."
            is practically worthless, even without the rude bit at the end that I left out. they have only shown that some non-autism symptoms of fragile X are mitigated in adult mice. it's poor form to extrapolate as you seem to be doing when there is no evidence to support it. might i recommend a biochemistry course?

            i sincerely hope these drugs do work. but even if they do it will only affect ~5% of the population of people with autism.
            • Re: (Score:2, Troll)

              by Gerafix (1028986)
              Never extrapolated as I never claimed that it worked on adult humans or that they even tested it on humans at all. Only that they claimed that it worked on the animals that they tested. Nice try at back peddling though.
              • Re:Assumption much? (Score:4, Interesting)

                by scapermoya (769847) on Monday January 11, 2010 @07:00AM (#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.
                • Biology (hard science) has a constructive approach going from the ground up - chemicals and physical operations.

                  Psychology (soft science) is about identifying trends and describing them clearly to other experts; something like anthropology or sociology does. Attempts to deconstruct these trends using other more "atomic" trends as well as finding interdisciplinary connections is extremely difficult and builds prestige; however, these often amount to being temporary fads that are later found to be weak or fa

  • $60m is pocket-money (Score:3, Informative)

    by PDoc (841773) on Monday January 11, 2010 @05:16AM (#30721202) Homepage
    Seriously, $60m isn't anywhere near enough to bring this to market. Most studies in pharma show that $1000m is far closer to the real figure these days, with some pushing that towards $1700m [drugresearcher.com]. Of course, this is an average figure, and the costs of drug development are highest towards the end (phase IIb, phase III). Any drug targeting the CNS is going to be expensive in trials, and with the condition apparently 'rare' (an ill-defined term), finding suitable patients willing to undergo the treatment in trials might be difficult. More realistically, $60m might get them to the point where a Big-Pharma will either buy the company or the drug.
    • Seriously, $60m isn't anywhere near enough to bring this to market. Most studies in pharma show that $1000m is far closer to the real figure these days, with some pushing that towards $1700m.

      One billion dollars to bring a product to market? Smells like BS to justify insane prices and legislation to stop generics being made. I see nothing much in the linked article to suggest the money is being used meaningfully, only commercially (which could be hookers and blow for the execs, or sales gigs for doctors to push their pills, etc...)

      Serious question: Where does the money go? What specifically makes the trials expensive?

      • by Gerafix (1028986)
        Just buy the Chinese version when it comes out, that's probably where the drugs will be made in the first place anyway.
      • by MrMr (219533)
        Those figures are roughly correct. They are computed by dividing the research expenditure of a company by the number of new drugs going to market in a specific time-frame.
        The reason the expenses are so high is the number of high-level employees feeding from the trough (IP, legal, management, and even a handful of scientists) and the absurd amounts some doctors get paid for experimenting on their patients (tens of thousands per data point is not uncommon)
        • by Abcd1234 (188840)

          Those figures are roughly correct. They are computed by dividing the research expenditure of a company by the number of new drugs going to market in a specific time-frame.

          Oh please, that's a bullshit calculation. That doesn't represent the cost of taking any one drug to market. The represents the cost of putting drugs through trials plus the cost of wasted research into dead-end areas plus all the organizational overhead of those research units plus god knows what else.

          • by paiute (550198)

            That doesn't represent the cost of taking any one drug to market. The represents the cost of putting drugs through trials plus the cost of wasted research into dead-end areas plus all the organizational overhead of those research units plus god knows what else.

            True, the cost to bring a single drug to market is far less. Now all you have to do is go back ten years to the benchtop chemist and tell him or her which one of the 500 structures they are working on is that one so they can ignore all the rest.

            • by Abcd1234 (188840)

              True, the cost to bring a single drug to market is far less. Now all you have to do is go back ten years to the benchtop chemist and tell him or her which one of the 500 structures they are working on is that one so they can ignore all the rest.

              Uhuh. And what's your point, exactly? You see, mine is that, despite what the libertarian slashbots here would have to believe, the overhead created by government regulation to bring a drug to market is not, in fact, 1 to 1.7B, as the OP would have us believe.

              Is th

              • by paiute (550198)

                But once a promising compound has been found

                That was my point.

                Once I pick the right numbers, the Maga Millions will be mine.

                • by paiute (550198)

                  But once a promising compound has been found

                  That was my point.

                  Once I pick the right numbers, the Maga Millions will be mine.

                  Of course I meant Mega Mullions.

      • by Yewbert (708667)

        I work at a Big Pharma, and I was going to make the same comment as the one to which you're replying. $750M to $1000M is much more realistic a range for the cost to bring a NEW API to market. (API = active pharmaceutical ingredient)

        This cost is the end result of high, demanding standards for quality, safety, documentation and a zillion other details governed by the FDA. If you want to know why FDA-approved drugs cost so much more than "dietary supplements" and all the other alterna-crap, it's because the

      • by nedlohs (1335013)

        Then your sense of smell and bullshit detection is faulty.

        What makes trials expensive is that the vast majority of drugs don't work or have too severe side effects. And you don't find that out until after you've spent some money.

        So yes, the actual dollars spent on "wonder drug X" is not $1 billion. But once you also count the other 1000 drugs that didn't make it (and were indistinguishable from "wonder drug X" at the start) you've spent that much.

        But in this case $60 million might do it. Of course if it fai

  • Side effects? (Score:5, Interesting)

    by Lord Lode (1290856) on Monday January 11, 2010 @05:53AM (#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?

    • Re: (Score:3, Insightful)

      This is a drug that may alleviate some of the symptoms of fragile X syndrome, many of these symptoms are not reversible, and one of them can be some forms of Autism

      This is not a "Cure for Autism", it is a possible, partial cure for a genetic disorder that has as one of it's effects in some patients some forms of Autism

      Autism is not simple...it has no one cause, and has no one cure ....

      • I just find the idea of a cure for autism hard to accept, because I think the cure is incompatible with what autism "enables" in some people. If someone is socially inept because he spends all his time with mathematics, the cure would make him socially active, but he'd stop doing math. What is cured then? You've made a person normal instead of special.

        Of course in the above I'm not talking about people with autism who can't do anything at all except sit in a corner, I guess for many people with autism you c

        • Re: (Score:3, Interesting)

          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 ma

          • by sjames (1099)

            I think you missed the point. Imagine magic pill X that immediately and perfectly cures autism. 100%, not a trace of physiological abnormality left. Arguably, giving that to an adult would be akin to wiping their mind empty and starting over. They would be left unable to appreciate any of the things they enjoyed before and would have no idea what they might enjoy now. Having never experianced normal socialization, they would still be on the outside looking in, but now with no special abilities and enjoymen

        • The dangerous case with your note is that it's result dependent.

          "Spends all his time with math" ... and writes semi-pro papers, tutors a kid, and writes little freeware puzzles to amuse the net hordes. Win.

          "Spends all his time with math" ... and tries to figure out the numerology of life, but crucially, gets the worst of new age with none of the science of emergence. Then he's total bait for medical pigeonholing. Too smart for "ordinary" types, but too flawed to play the "strange genius" card.

          • by ArsonSmith (13997)

            Or possibly even worse

            "Spends all his time with math" ... and sits in a corner calculating and reciting pie for 12 years before dieing due to lack of movement. It would be better to reduce the maths ability in order to give the person the ability to experience life.

    • Very possibly.

      If the ability comes together holistically/gestalt, I believe there is a "critical mass" that needs to occur to get it right. When the subject falls below that threshold, they get GarbageOut that proves very frustrating.

    • by ShakaUVM (157947)

      >>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?

      In the study of "idiot" savants, studies have shown that curing their extreme social inability also "cures" their ability to be exceptional at math, or whatever.

    • Re: (Score:3, Interesting)

      by netsavior (627338)
      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 ha
  • The company is funded almost entirely by an undisclosed family investment of $60 million, with $6 million from the National Institutes of Health. Carpenter says that Seaside has enough funding to take its compounds through clinical testing and approval. "We are prepared to do it ourselves," he says. "But if there is a partnership that allows us to more rapidly advance compounds, then we would embrace that opportunity."

    So they basically get to develop a drug and bring it to market for free.
    How much do you think they'll charge for it?
    The cynic in me suspects the answer won't be "slightly over cost"

  • creepy in the figurative sense, but also creepy in the literal sense too: it creeps up through the generations

    the basic idea is that you have a region of genetic code that, with every generation, gets a little longer with repeats. such that, after a few generations, it results in mental deficiencies. of course, its not so straightforward: your child's number of copies of repeats may dramatically jump, or it may hold relatively stable with an unchanged number of copies at a borderline level for many generati

  • Seeing as how the average cost to a major pharma to bring a drug from the bench of the medicinal chemist to the bottle in your medicine cabinet is approaching a billion dollars, having only $60 million to work with seems like running on a shoestring.

  • How about finding the cause of autism or a cure, rather than a drug to manage it?

    My guess? The Pharmaceutical companies make more money selling drugs to manage a condition rather than curing it, so that is where their researchers look.

    Additionally, the human genome has not changed much. So, either diagnostics have gotten better so more cases of autism are being noticed or there is actually more autism. If the latter case is true it has to be an environmental change as the cause. Discovering that would me

  • The article is pretty good, actually, in that it doesn't try very hard to claim that they're curing the world of its ills. There's a little in there, but mostly it deals with Fragile X.

    Randi Hagerman (the researcher quoted extensively in the article) is one of the leading lights in Fragile X research. She and her husband, Paul, described the gene, developed the RFLP that we now use to diagnose the illness, and did much of the fundamental work to explain the genetic-expression behavior of the gene. It is not

  • I notice a number of other industries are copying Apple's iPhone App commercials now. This would work perfectly for the drug industry: "Have this disease? theres a drug for it!".

    This paradigm is so deeply ingrained in the American psyche now that I automatically wonder about some new medicine for all the new disease syndromes we hear about. There are substances for mental health and physical fitness. Even herbal remedies are squished into pill shapes to make them seem more "medical".
  • by RoFLKOPTr (1294290) on Monday January 11, 2010 @11:14AM (#30724088)

    Autism vaccine is linked to flu in children. News at 11.

  • I'm a test subject in a trail.

    From what I understand (from my doctor) autism is thought to be genetic but it does have certain environmental triggers that affect whether or not you develop symptoms and to what extent. One of these triggers is exposure to the measles virus (the vaccine is an attenuated form of said virus). Apparently a person's body develops inappropriate antibodies that attack certain parts of the brain (Amygdala) after exposure to one of these triggers.

    The neurotransmitter Oxytocin (which

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