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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."
If you were really autistic you would lurk and never post.
http://michaelsmith.id.au
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.
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. 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.
Give a man a fire, and he's warm for a day. Set a man on fire, and he's warm for the rest of his life. (Terry Pratchett)
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.
Beware the Jubjub bird, and shun the frumious Bandersnatch.
Then again, if you had fragile X syndrome you wouldn't actually have autism. This is a deeply misleading article title and summary, since Autistic Spectrum Disorder (ASD) covers a wide range of psychological profiles and is deeply misunderstood by most people - even plenty of people who work with it every day. You will notice, reading the article (yeah, I must be new here) that none of the scientists mention ASD. The guy who wrote this piece just thought that would give him an angle, since no one has heard of fragile X syndrome, but everyone loves a good autism story, despite (because of?) most people never having met someone with serious levels of ASD.
Just to clear things up, fragile X syndrome is a chromosomal abnormality that causes various physical deformities and some forms of mental retardation. This is acceptable of you want to know more. There is some limited evidence that correlation exists between some forms of ASD and fragile X syndrome, but causality is far from demonstrated.
Additionally, ASD is defined as being a "pervasive developmental disorder", meaning that a) symtoms must be present from fairly early on in life and b) autism is an innate part of the person suffering from it, and a cure not only doesn't exist - the concept of a cure is nonsensical. Don't get me wrong, I would love there to be a cure for ASD, but medical science currently defines it as uncurable. As an analogy, it would be like trying to 'cure' someone of having social function and being capable of imaginitive play - you could teach them limited functions to appear like they had no grasp of the abstract, but you couldn't turn them autistic.
The media, and people in general, need to cease this endless obsession with autism - it's an incredibly complex subject, and studying it for years only allows you to scratch the surface (trust me on this). 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.
Be smart, help people!
what if what you think is red is actually blue to me lol!
Beware the Jubjub bird, and shun the frumious Bandersnatch.
The issue is one of impairment of functioning. This, of course, means that many self-proclaimed 'Aspies' are not what this drug targets.
A child that is not capable of communicating with its parents is autistic. A child that leads his parents by the hand to something instead of pointing to what he wants is autistic. Even dogs are capable of shared attention (pointing at an object is a concrete example) while autistic children are not. Shared attention is of course necessary for language communication as verbally expressing ideas is based on shared attention of ideas and concepts - and these kids can't even point to a toy or cookie.
An 8 year old that learns to program in C++ at the age of 8 is exceptionally bright, not autistic. At this same age an autistic kid will be spinning the wheels on a car (as opposed to playing with it) or stacking blocks for hours on end. They may play in the same vicinity as other children, but almost never with them. You see these same tendencies in normal children up to a certain age - an 18 month old will play in the proximity of other children but not with them. A 3 year old plays with other children instead of simply being in the same vicinity. Autistic children never reach this stage.
Autistic adults social and communication issues are simply an extension of these milestones that were reached significantly (or never) later than other people because of neurological problems. A geeky guy that enjoys chatting on Ventrillio while raiding in World of Warcraft for hours on end is very likely not autistic given how social using voice chat and raiding is. An autistic adult isn't very likely to frequently visit comic book or Star Wars conventions either. Just because these activities are stereotyped for males with social phobias or other social issues doesn't mean that they're indicative of autism.
True autism is a very real and very impairing condition, not a matter of having odd interests and being a bit socially awkward.
No wonder you posted anon, that's an incoherent statement! You might as well say, "If today's dead was normal, then today's living would be called dead. Objectively, we are all dead and living at the same time really." Because life and death is merely a definition. I'm sure you understand that only works for cats in boxes.
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?
here we go, buddy:
/. story "Startup Tests Drugs Aimed at Autism," which is only mildly true.
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
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.
Beware the Jubjub bird, and shun the frumious Bandersnatch.
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!
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.
Autism seems to be on the rise and some families seem to point out patterns - pre vaccines, happy, post vaccines dolphin-esque.
I think it would be interesting to see what would happen if everybody stopped vaccinating their kids until well after autism's typical age of onset.
Although I think I know the answer: we would have just as many autistic kids, which would suggest that it isn't the vaccines causing the autism, yet a few people will cling to their belief no matter the evidence against it.
some families seem to point out patterns - pre vaccines, happy, post vaccines dolphin-esque.
This probably originates from a single study in the UK more than ten years ago that linked the MMR vaccine with increased incidences of autism. That study has been since been thoroughly debunked and discredited. Stop repeating it.
I use Friend/Foe + mod-point modifiers as a karma/reputation system.
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.
I use Friend/Foe + mod-point modifiers as a karma/reputation system.
As for Autism, it's not ideal and can cause a lot of problems for people but it can also bring some big pluses too. Do we really all want to be clones? looking the way celeb culture says we should and having perfect personalities? Hell no! Rejoice in the difference.
Enlighten me, please explain what the pluses are of this severe disability.
Would you say the same about rheumatism, heart failure, emphysema, Parkinson or any other disability? I think it's probably best to let patients or their caregivers decide whether their condition is worth treating.
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.
# cat
Damn, my RAM is full of llamas.
"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.
Beware the Jubjub bird, and shun the frumious Bandersnatch.
Yeah, he probably wouldn't be able to do such useful things as remembering the whole phone book or recalling which baseball player did what in each year.
Or design and implement bittorrent, and run a company around it; see http://www.businessweek.com/magazine/content/08_43/b4105046863317.htm
Or win the Nobel Prize in economics; see http://en.wikipedia.org/wiki/Vernon_L._Smith and http://www.econtalk.org/archives/2008/03/vernon_smith_on_1.html
(Okay, that's Asperger's Syndrome; but I think that's within the scope of this discussion)
He might be able to button his own shirt or wipe his own ass, though.
Or it might be that he is better able to communicate with other people; he might have an easier time stumbling unto the idea that if he asks someone a question and silence is the answer, it might be because of an internal struggle between not wanting to lie and not wanting to admit the truth. And that he can gain something by not putting people in that situation again.
One of the reasons that the media, and people in general, have seemingly become obsessed with autism is that there has been a very significant rise in diagnosis of autism spectrum disorders. Furthermore, autism spectrum disorders are often misdiagnosed as other problems. Finally, the public is not aware that autism spectrum disorders cover a range of different, distinct disorders, from very low functioning varieties to very high functioning varieties.
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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.
I think the bigger skew to the risk judgment is that vaccines have been too successful for their own good in some respects. Do you remember when polio paralyzed people regularly, measles killed and a cough might being fears of being Whooping? If you're around my age (34) or even a bit older, you don't. Neither do I. Of course, I've read many accounts, but haven't seen it first hand. So it would be easy to discount the threat that these diseases pose.
Some people think: "I haven't seen anyone I know of die or be maimed by polio/measles/whooping cough, so how bad can it be?" (Yes, one antivax nut in Australia even said that whooping cough has never killed anyone!) If they had actually had a cousin die from whooping cough, had a sibling scarred for life from measles and had a friend who barely survived polio but will be in a wheelchair the rest of his life, I doubt they'd be so anti-vaccine.
My sci-fi novel, Ghost Thief, is now available from Amazon.com.
I disagree that "the concept of a cure is nonsensical". Your reversal is a bit like saying 'imagine someone can play guitar, and you had to teach them to be incapable of playing it...' - people who can play guitar can't un-learn it, but people who can't play guitar are capable of learning to play it to a greater or lesser degree. Someone with a severe physical disability may never be able to play guitar, depending on that disability.
In one sense I do understand what you mean, that the disorder is an intrinsic part of a person's makeup and not just a bolted-on impairment. However, ASD is a spectrum, and people sit in different places on that spectrum. For some, their ASD would make certain types of social interaction difficult, but not impossible, to process and understand - with patience, it is possible to extend the understanding and mastery of situations that would previously have been too distressing or just incomprehensible. It is therefore possible to 'cure' certain ASDs to the extent that a person is able to function 'normally' (whatever that means) in society and have a better quality of life than they would otherwise have had.
Your sentence "As a rule of thumb, if you can put together a fully formed sentence, you almost certainly don't have meaningful levels of ASD" indicates to me that you have a very little - or a very distorted - understanding of ASD (or you have constructed that sentence poorly and didn't really mean it). Lots of people are able to speak very well and enjoy conversation yet have significant ASD that affects every part of their life.
(Okay, that's Asperger's Syndrome; but I think that's within the scope of this discussion)
Uh, no, it's probably not.
When people talk about searching for cures for autism, they aren't typically talking about Asperger's. They mean actual, severe autism. You know, the kind where the individual is virtually non-functional.
As an aside, I don't suppose you're a self-styled Asperger's sufferer, are you? Because around here, the slashbots seem to think it's kinda cool to blame all their social problems on Asperger's (probably because the follow-up assumption is that, along with having an excuse for being socially awkward, they can also be comforted by the fact that they must obviously be brilliant, too). Hell, it's the new ADD among the Slashdot crowd, as far as I'm concerned.
Autism vaccine is linked to flu in children. News at 11.
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.
Disconnect your television. Do your own research. Draw your own conclusions. They're probably lying. Don't be a sheep.
Someone else would have made the same discoveries. There are plenty of people that are just as smart.
(1.21 gigawatts) / (88 miles per hour) = 30 757 874 newtons