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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!
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.
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.
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.
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.
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.