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Startup Tests Drugs Aimed at Autism

kdawson posted more than 4 years ago | from the whose-x-you-callin'-fragile dept.

Medicine 171

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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Do I have it (-1, Offtopic)

simstick (303379) | more than 4 years ago | (#30721142)

If I have the urge to get a first post?

Re:Do I have it (3, Informative)

MichaelSmith (789609) | more than 4 years ago | (#30721150)

If you were really autistic you would lurk and never post.

Re:Do I have it (1, Insightful)

Anonymous Coward | more than 4 years ago | (#30721172)

Or would he post but never lurk?

Re:Do I have it (5, Informative)

DangerFace (1315417) | more than 4 years ago | (#30721250)

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 [wikipedia.org] 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.

Re:Do I have it (1)

Hieronymus.N (865735) | more than 4 years ago | (#30721882)

Thank you. Seriously.

Re:Do I have it (0)

Anonymous Coward | more than 4 years ago | (#30721946)

Agreed. Its hard to articulate something like that. I particularly like your reversal of the curing, this is something I will use in the future.

Re:Do I have it (2, Informative)

morgan_greywolf (835522) | more than 4 years ago | (#30722192)

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.

Re:Do I have it (2, Interesting)

tigre (178245) | more than 4 years ago | (#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:Do I have it (2, Insightful)

uglyduckling (103926) | more than 4 years ago | (#30723002)

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.

when I read about "drug"... (1)

Adolf Hitroll (562418) | more than 4 years ago | (#30721144)

...I fear this is more about big pharma milking scared people than about saving people.

Assumption much? (1)

scapermoya (769847) | more than 4 years ago | (#30721152)

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

Re:Assumption much? (-1, Troll)

Ethanol-fueled (1125189) | more than 4 years ago | (#30721194)

Autism is what happens when an American marries a Gook, often in a military marriage, and then both of them party and don't pay any attention to the developing baby.

The baby, never having been nursed with real breast milk, shuts themself out of life. They get up at all hours in the morning and just SCREAM, with no rhyme or reason, and inconveniencing everybody else in the neighborhood.

Parents of autistic kids have nothing to blame but themselves. You injected them with all kinds of crap because the news told you to. You never sang songs to them because you hired a maid to watch them while you partied all night. Yes, your kid was born autistic...because you fucked up.

Re:Assumption much? (0)

Anonymous Coward | more than 4 years ago | (#30721212)

And here I was thinking it was just vaccines. Now I can blame a nationality, a race, and parties!

Re:Assumption much? (1)

Hal_Porter (817932) | more than 4 years ago | (#30723578)

And here I was thinking it was just vaccines. Now I can blame a nationality, a race, and parties!

Ladies and Gentlemen! I have just signed the legislation that will abolish scourge of autism forever. The GOP is banned as of five minutes ago. Any questions? Not from you, Mr Hannity.

Re:Assumption much? (0, Troll)

scapermoya (769847) | more than 4 years ago | (#30721214)

yawn. is this supposed to be sarcasm? wrong forum, buddy. don't feed the trolls kids. it's what they want you to do.

Re:Assumption much? (0)

Anonymous Coward | more than 4 years ago | (#30721236)

"scapermoya" posting anonymously so as to not piss you all off (tee-hee!) but -- Lets be boring and predictable kids. How about another safe, sterile Soviet Russia joke!

Re:Assumption much? (0)

Anonymous Coward | more than 4 years ago | (#30721364)

"scapermoya" posting anonymously so as to not piss you all off (tee-hee!) but --
Lets be boring and predictable kids. How about another safe, sterile Soviet Russia joke!

In Soviet Russia, nobody likes Ethanol-fueled.

How did I do?

Re:Assumption much? (1, Interesting)

Gerafix (1028986) | more than 4 years ago | (#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? (3, Interesting)

scapermoya (769847) | more than 4 years ago | (#30721232)

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:Assumption much? (1, Troll)

Gerafix (1028986) | more than 4 years ago | (#30721300)

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? (4, Informative)

scapermoya (769847) | more than 4 years ago | (#30721408)

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:Assumption much? (1, Troll)

Gerafix (1028986) | more than 4 years ago | (#30721512)

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? (3, Interesting)

scapermoya (769847) | more than 4 years ago | (#30721648)

"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:Assumption much? (1, Insightful)

Anonymous Coward | more than 4 years ago | (#30722458)

I'm starting to think you two have a personal stake in this article (whether you know it or not)...

What if (1, Insightful)

MrMista_B (891430) | more than 4 years ago | (#30721164)

What if it's the people who don't have Autism who are sick? What if it's 'normal' that's wrong?

Re:What if (5, Insightful)

Gerafix (1028986) | more than 4 years ago | (#30721178)

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.

Re:What if (0)

Anonymous Coward | more than 4 years ago | (#30721252)

What constitutes autism is merely a definition. If today's autism was normal, then today's normal would be called autism. Objectively, we are all autistic and normal at the same time really.

Re:What if (2, Funny)

Gerafix (1028986) | more than 4 years ago | (#30721292)

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.

Re:What if (1)

TubeSteak (669689) | more than 4 years ago | (#30721310)

"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.

You know, we could settle this once and for all by asking the cat.

Scientists: Meow once if you're alive, meow zero times if you're dead.
Cat: Meow.
Scientists: Eureka!

Re:What if (1)

mwvdlee (775178) | more than 4 years ago | (#30721822)

Yes, but the boxed cat is all about the state being unknown until observation.
I'm sure hearing counts as observation.

Re:What if (0)

Anonymous Coward | more than 4 years ago | (#30721712)

I posted anon because I'm too lazy to maintain yet another account on some site.

Life and death are pretty good examples in this case. Which is THE correct definition of life and death? A medical one? A legal one? A religious one? A philosophical one?

In the end they are just words of which there exists dozens if not hundreds of definitions. And each definition is just some arbitrary opinion from one or more people.

The definition of death has changed over the centuries (http://en.wikipedia.org/wiki/Death#Problems_of_definition).

Re:What if (1)

AHuxley (892839) | more than 4 years ago | (#30721356)

Its not 1980s Moscow and your not a human rights worker, political type or democrat. Then your "today's normal" would hold.
Autism seems to be on the rise and some families seem to point out patterns - pre vaccines, happy, post vaccines dolphin-esque.
Squalene and mercury based preservative let drug companies extend expensive drug stocks over many more shots.
Until the cost of law suits impacts the bottom line ... normal can be called autism.

Re:What if (4, Informative)

jonaskoelker (922170) | more than 4 years ago | (#30721424)

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.

Re:What if (4, Interesting)

Paul Jakma (2677) | more than 4 years ago | (#30721534)

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.

Oh yes, the horror of disease too (1)

jonaskoelker (922170) | more than 4 years ago | (#30721600)

We know what would happen: Far more people would suffer from complications of diseases

Ah yes, of course. I probably should have mentioned that too.

You should be modded up for pointing this out. It's rather important that people see your post and are aware of this point.

Re:What if (1)

mwvdlee (775178) | more than 4 years ago | (#30721840)

I think "death" is pretty effective prevention of "autism". In absolute numbers, there would be less autistic people. In percentages, it would be about the same, but you know how statistical studies are created.

Re:What if (1)

toomanyairmiles (838715) | more than 4 years ago | (#30722104)

I don't think it's a belief that MMR is bad that people are clinging to so much as taking a risk judgement. The world has been subjected to so many drugs which, while approved for use by scientists and government, turn out to be killers that many perfectly rational people refuse to believe the scientific evidence and don't want to take any risk with their children's long term heath. To name a few:- Thalidomide (birth abnomalaties), Redux (heart valve disease), Vioxx (increased the risks of heart attack and stroke), Seldane (fatal heart-rhythm irregularities), Posicor [Mibefradil] (caused toxic levels of 25 different drugs to build up in the body), Rezulin (sudden liver failure), Duract (liver toxicity), Baycol (rhabdomyolysis, which can lead to kidney failure), Zelnorm (increased risk of heart attacks and stroke), Just one of these, Vioxx is suspected of causing 27,000 deaths.

Re:What if (2, Informative)

Jason Levine (196982) | more than 4 years ago | (#30722888)

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.

Re:What if (1)

rkhalloran (136467) | more than 4 years ago | (#30723074)

This is what I find infuriating about the anti-vaccination crowd; their "risk assessment" that decides to avoid protecting their children relies on the rest of us taking said risk for ours and providing "herd immunity" on behalf of their offspring. The recent, needless pop-ups of preventable diseases is a result of not enough parents feeling the need to protect their own children because "everyone else's kids are immunized, mine don't need to be".

Selfish gits.

SCOX(Q) DELENDA EST!!

Re:What if (4, Informative)

Paul Jakma (2677) | more than 4 years ago | (#30721502)

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 [timesonline.co.uk] . Stop repeating it.

Re:What if (1)

bcmm (768152) | more than 4 years ago | (#30721624)

some families seem to point out patterns

Anecdotes, made more common by the fact that autism almost always gets diagnosed at about the age kids get vaccinated, (even in unvaccinated kids - perhaps the absence of vaccines causes autism too, hmm?). Large studies do not show such patterns.

Re:What if (1)

starbugs (1670420) | more than 4 years ago | (#30721778)

How about stopping all this crap with so many vaccines?

Vaccines are good when they're needed but how many of them are not needed?

Do you remember how many vaccines you got growing up?
Well, check how many more vaccines children now get.

Vaccines are a hallmark of how great and beneficial our Medicine can be, but please:

Stop putting all this chemical crap into vaccines (preservatives, etc.) or at least give us (those who worry about what's injected into our kids) an alternative that we can trust. We're more than willing to pay extra for it.

Make those with a vested interest in the sale of vaccines have no say in which and how many vaccines we get.(Just check how much the H1N1 vaccine companies made, they love pandemics).

Stop with the "vaccines are the best things in the world and you are part of the 'axis of evil' if you think otherwise" BS. You're just as crazy as those who think that all vaccines should be banned. Just think critically and be skeptical both about promoters of vaccines and those who oppose them.

Many think that vaccines contribute to autism, I think vinyl floors [scientificamerican.com] could be worse. But in rare situation, maybe vaccines are a contributing factor, maybe they are not. Show me truly impartial research.

Now I'm off to research if all the money spent vaccinating children against polio in a country that hasn't had a "wild case" since 1979 would have been enough to have already eradicated the disease globally. It's frightening that this vaccine currently causes more harm than good.(In the US 8-9 cases/year caused by the vaccine, 0 cases are caused by the actual disease since 79, but the vaccine companies are laughing all the way to the bank)

Re:What if (1)

mwvdlee (775178) | more than 4 years ago | (#30721990)

Stop putting all this chemical crap into vaccines (preservatives, etc.)

Vaccines are pretty much 99% artificually created. Remove the chemicals and you're left with something that has little to no useful effect. Besides, many chemicals are created in ways that just immitate nature.

or at least give us (those who worry about what's injected into our kids) an alternative that we can trust.

Trust is highly subjective. What would need to be changed in order for you to trust vaccines?

Re:What if (-1, Troll)

Anonymous Coward | more than 4 years ago | (#30722650)

"Trust is highly subjective. What would need to be changed in order for you to trust vaccines?"

Maybe if you remove the mercury which is one of the most toxic substances known to man. There are plenty of animal studies that actually found evidence of thimerosal inducing damage to the neurons. Also i would like to see a long term study done with a large control group.
Such a study does not exist on humans, so there is no way to know what the cumulative and long term effects are from all these vaccines.

Re:What if (1)

bcmm (768152) | more than 4 years ago | (#30722194)

Stop putting all this chemical crap into vaccines

This is Slashdot. News for Nerds. You should already know that you're made of chemicals.

Stop with the "vaccines are the best things in the world and you are part of the 'axis of evil' if you think otherwise" BS.

I did not say that, it doesn't look like I said that, and I don't believe you actually think I said that.

Many think that vaccines contribute to autism, I think vinyl floors could be worse. But in rare situation, maybe vaccines are a contributing factor, maybe they are not. Show me truly impartial research.

I think kittens are the sole cause of autism. I don't know why, but I do. No matter how much research you collect to the contrary, I will consider it all to be insufficiently impartial for me, despite having no research that backs up my own opinions.

Re:What if (1)

starbugs (1670420) | more than 4 years ago | (#30723310)

I did not say that, it doesn't look like I said that, and I don't believe you actually think I said that.

The four points I made about vaccines were not directed at you or anything you wrote, but you did mention that autism gets diagnosed around the age a child gets vaccinated. Vaccines in children are so frequent now that (I think) this statement does not make much sense. Children where I live are scheduled for an average of two or more vaccines per year.

There is a difference between chemical 'crap'(as I called it earlier) in a vaccine and substances that are benign. If I order food from a restaurant I will request "No MSG" but "extra salt"(my BP is fine). Both make the food taste better.

I think kittens are the sole cause of autism. I don't know why, but I do. No matter how much research you collect to the contrary, I will consider it all to be insufficiently impartial for me, despite having no research that backs up my own opinions.

There are people who don't believe that smoking is harmful, some of those are smokers who will die of lung cancer. It's not my job to change their mind. We know that there are cases of children who have never received any vaccines but developed autism. There also are people who have "life-threatening" reactions to pet dander. If consistent contact with an allergen creates an auto-immune reaction that contributes to autism, then maybe you are right about kittens. There is the risk of toxoplasmosis in pregnancy, who's to say that something cats carry might not be the cause of other problems. Not long ago it was thought that stress caused most stomach ulcers.

But you have made me more suspicious of Cats [amazon.ca] .

Re:What if (1)

bcmm (768152) | more than 4 years ago | (#30721640)

And sorry for the doublepost, but the amounts of mercury are minute - about the same as is present in a tin of tuna. Oh help, should we listen to the unscientific claims about consumption of fish making children into geniuses, or the ones about mercury giving them autism?

Re:What if (1, Interesting)

Anonymous Coward | more than 4 years ago | (#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.

Backward what-if (2, Interesting)

macraig (621737) | more than 4 years ago | (#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:Backward what-if (2, Insightful)

Gerafix (1028986) | more than 4 years ago | (#30721484)

Because people haven't stopped reproducing, even if they have certain latent or expressed genetic variations. I suppose I should have said that we would be mostly autistic, not all. It appears you're begging the question as to whether or not it is valuable. Whether autistic traits are increasing or not is debatable, especially as to the cause - genetically valuable or simply better clinically recognized? Keep in mind Homo sapien DNA is not perfect, it is prone to variation and mutation, even variations and mutations which are harmful in one area and beneficial in another. Although I am sure you're aware of that already.

Re:Backward what-if (1)

macraig (621737) | more than 4 years ago | (#30721594)

The species might yet become "mostly autistic", though even our great-great grandchildren won't be around to comment on it, much less us. All we can do is speculate. The point of my comment was specifically to challenge your implication that all autistic traits are consistently detrimental and thus inconceivable that they might actually be increasing in prevalence and replacing what we now call neurotypical traits. I doubt that many people would argue that ALL autistic traits are beneficial, but some most definitely are, and many of the rest are fairly benign in truth... unless "being different" is by itself a death sentence. Are all of what we consider autistic traits actually related, stemming from a common genetic or environmental stimulus? Probably not, and I have no doubt there will be dramatic refinements to diagnoses in the future. We can only talk about it using the lousy definitions we have now, which aren't terribly objective.

Re:Backward what-if (0)

Anonymous Coward | more than 4 years ago | (#30721638)

Traits that are not purely single-allele dominant are rarely bread out completely. There are fatal congenital diseases with prevalance similar to full-blown autism (as compared to asperger's).

Not to mention that full autism (as opposed to Asperger's) is often low-functioning, at a much higher prevalance than the neurotypical population.

Claiming Archimedes as autistic is a bold move. He is known to have eccentric behaviours, but then, there's more than one term for social disorders (or atypicalities if you'd rather), and records that far back are necessarily spotty and translated. I'll give you Perelman. I'm not convinced Craig Newmark's single notable achievement is anything special. I'm not saying he's a bad person or a dumb person, just a lucky person with a website that I'm fairly convinced could be done far better. At lest make the last entry in a two-column list with an odd number of entries appear in the left column instead of the right. Yikes.

Also, you seem to have a Pokémon view of evolution. Call me back when the world has been congested with 6.5 billion people for thousands of years, OR there's a cataclysm that kills the large majority of those 6.5 billion but spares the Autistic; because we certainly haven't seen autism rise from an environment that didn't even exist when some currently-living people started breeding. If indeed Autism has been displacing the present neurotypicality, it's from environmental factors that have existed in steady-state before modern society, and may or may not still be active.

Disclaimer: I may or may not be neurotypical. I was diagnosed with autism at a young age, multiple times, but each time they eventually decided it wasn't really autism. I'm very very bad at social situations (eg. afraid of approaching the checkout counter because I'll have to deal with a person there -- thank god for the self-checkout kiosks at grocery stores) but I lack many of the strongest hallmarks of autism, so I'm going to do the opposite of what half of slashdot does and self-diagnose as neurotypical (but odd).

Re:Backward what-if (1, Insightful)

Anonymous Coward | more than 4 years ago | (#30721694)

Why is there a persistent trail of autistic achievers from Archimedes ...

Do you seriously think that he could be diagnosed over 2,000 years after the fact? Anyone you see making historical claims of any given diagnosis is pushing an agenda.

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.

You are aware that autism is based on an arbitrary criteria established by the American Psychiatric Association or World Health Organization, depending on which country you live in. Diagnostic criteria change regularly and diagnosis are solely based on the subjective opinion of the practitioner. Also note that 10 years ago no one was talking about Asperger's, which suggests that this disorder is a fad.

Standards for diagnosis have changed, Asperger's has come into fashion with geeks and nerds, and an increase in the number of diagnosis does not mean that what ever causes autism is becoming more common. Additionally, you seem to be mistaken on how natural selection and evolution occur. First, evolution does not occur within 10, 50, or even 100 years. Second, natural selection relies on genetic fitness, which includes being the most fit to reproduce. If you have ever been around a developmentally delayed adult (autism is a specific type of developmental disorder) you can quickly estimate how many women would have sex with them. This doesn't even factor in their ability to earn a suitable living or properly care for their children. By the way, inappropriate or severely impaired self-care skills is one of the diagnostic criteria of autism.

Natural selection may in fact have been working slowly to weed out the (currently) neurotypical.

No it isn't.

Re:Backward what-if (1)

TubeSteak (669689) | more than 4 years ago | (#30721766)

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?

1. For autism to have been weeded out of the gene pool, it would have to be solely genetic and hereditary. The best research we have to date says that autism has a genetic/hereditary component and is also caused by "unidentified environmental factors" [google.com]

2. The people you are talking about (assuming they have autism) are what's called "high-functioning". The type of autism that leaves you banging your head against the wall isn't very conducive to reproduction or intellectual success.

3. You need to prove that "the traits not only persisted but seem to be increasingly prevalent". I can't find the article, but some scientists went and did a survey of adults and their conclusion can be boiled down to 'better diagnosing and increased reporting is the main reason that autism rates have been rising.'

Re:Backward what-if (0)

Anonymous Coward | more than 4 years ago | (#30722082)

Or perhaps decreased barbarism in our societal structures leads to improved survivability for people who run up and scream in your face, flip out because a bus drove by, or get violent due to seeing a picture of a fish? These people would probably have struggled to survive in the past because provocative autistic behaviors would be selected against with violence and institutionalization.

Now that we don't kill people for yelling at us, maybe the screaming autistics are more prevalent?

Re:What if (4, Interesting)

bcmm (768152) | more than 4 years ago | (#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:What if (1)

zwei2stein (782480) | more than 4 years ago | (#30721854)

Well, nowadays, you can argue that families with some members on spectrum have advantage; I am quite sure that some autistics are more than responsible for getting people laid (By taking care of communication chanells that allow hookups, yay for explaining how to use im and for setting up accounts!).

You'd also have "a bit better factor" - simply put, easiest way to pick up opposite sex is to be accompanied by someone who is similar to you but slightly less attractive (Ever noticed that girl on boyfriend hunt is usually accompanied by slightly fatter friend?). Sibling that is a bit socially akward is perfect "wingman". And while he might have less tham optimal change of getting laid, part of genes will still get around through his siblings he helped.

Re:What if (1)

Pharmboy (216950) | more than 4 years ago | (#30721890)

This is what worries me about genetic testing before birth. What if Stephen Hawking had been determined to be "unfit for life" before he was born, in order to save him from horrors of ALS?

Re:What if (0)

Anonymous Coward | more than 4 years ago | (#30722382)

What are the stats on little autists in families where both parents are in a field like engineering, math, or science? Don't they have a higher tendency to having a little austist. I wonder if it's sort of like schizotrophic/schizophrenic. A schizotrophic person could do well as a village's priest/medicine man/witch doctor. But when two schizotrophic individuals breed, the odds of schizophrenic offspring is significantly higher.

Re:What if (1)

LanMan04 (790429) | more than 4 years ago | (#30722522)

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?

The exact same thing has been postulated about color-blindness, which affects 10% of the male world population.

Re:What if (1)

Teppy (105859) | more than 4 years ago | (#30723208)

Not necessarily true. It's plausible that Asperger's syndrome was an evolutionary disadvantage millennia ago (also the food allergies that tend to go with it) , but an advantage today. (And it's no longer hard to avoid troublesome foods.) Furthermore, smart people tend to have kids with Asperger's, and people with Asperger's tend to mate with others with the same condition. This may not be run-of-the-mill natural selection at work - we may be witnessing speciation. How cool is that?!?

Re:What if (2, Funny)

scapermoya (769847) | more than 4 years ago | (#30721254)

what if what you think is red is actually blue to me lol!

Re:What if (5, Insightful)

Anonymous Coward | more than 4 years ago | (#30721288)

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.

Re:What if (0)

Anonymous Coward | more than 4 years ago | (#30721406)

What if it's the people who don't have Autism who are sick? What if it's 'normal' that's wrong?

It could be. And considering that folks who aren't artistic can be helped with art therapy tells me that artism is a natural part of being human.

Re:What if (2, Interesting)

Anonymous Coward | more than 4 years ago | (#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 (1)

mwvdlee (775178) | more than 4 years ago | (#30722078)

Could you describe how your "tools" work compared to the "tools" of non-autistic people?

Re:What if (1)

maxume (22995) | more than 4 years ago | (#30723748)

Probably not, having an extensive understanding of 'normal tools' would likely allow him to utilize them.

Re:What if (1)

Beretta Vexe (535187) | more than 4 years ago | (#30722196)

It's not the good screwdriver ! It's a torx screw and you are using a crosshead screwdriver. Good lord are use using imperial unit too ?

Re:What if (1)

clickclickdrone (964164) | more than 4 years ago | (#30721428)

>What if it's 'normal' that's wrong?
Indeed. It's all part of the medicalisation of perfectly normal conditions and behaviours. There have been no real breakthroughs in terms of treating real disease since the 60/70's when all the big stuff happened. Since then, outside of cancer, not much has really happened. As a result, big pharma have started to work on finding cures for problems we never knew we had - controlling behaviours outside the norms, rafts of mumbo jumbo like anti-oxidents, miracle foods etc. Read 'Bad Science' by Ben Goldacre (and check his web site of the same name) for some enlightening stuff.
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.

Re:What if (2, Insightful)

rve (4436) | more than 4 years ago | (#30721544)

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.

Re:What if (1)

clickclickdrone (964164) | more than 4 years ago | (#30721608)

http://boingboing.net/2009/07/16/autism-as-an-academi.html [boingboing.net]
Gives some examples. Not sure it outweighs the negatives but it's still food for thought.

Re:What if (1)

rve (4436) | more than 4 years ago | (#30723380)

I get the feeling that you're using the tag autism where you really mean 'nerd'.

Re:What if (1)

uglyduckling (103926) | more than 4 years ago | (#30723100)

Really? Have you ever heard of antiretrovirals? There have also been many antihypertensives, antiepileptics and analgesics developed since the 1970s. Intensive care medicine (adult, paediatric and neonatal) has moved on in leaps and bounds since the 70's. I won't go on except to say you're profoundly wrong.

Re:What if (-1, Flamebait)

Anonymous Coward | more than 4 years ago | (#30721464)

You are fucking stupid.

Re:What if (0)

HaZardman27 (1521119) | more than 4 years ago | (#30722768)

Is it common for autistic individuals to reproduce? If not, then non-autistic individuals would definitely be "normal."

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

PDoc (841773) | more than 4 years ago | (#30721202)

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.

Re:$60m is pocket-money (1)

BiggerIsBetter (682164) | more than 4 years ago | (#30721520)

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?

Re:$60m is pocket-money (1)

Gerafix (1028986) | more than 4 years ago | (#30721542)

Just buy the Chinese version when it comes out, that's probably where the drugs will be made in the first place anyway.

Re:$60m is pocket-money (1)

PDoc (841773) | more than 4 years ago | (#30722018)

A good, and fairly impartial summary is here [america.gov] . A more detailed guide (PDF) is here [innovation.org] . However, perhaps the most salient stat is: "Only 5 in 5,000 compounds that enter the preclinical testing phase actually make it to human testing. One of these five drugs tested in people is approved." As for trials, a good break-down of costs is found here [lifesciencesworld.com] . I work in pharma as a scientific researcher, and I find the costs terrifying - especially as my salary appears to be the only thing smaller than six-figures...

Re:$60m is pocket-money (1)

MrMr (219533) | more than 4 years ago | (#30722058)

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)

Re:$60m is pocket-money (1)

Abcd1234 (188840) | more than 4 years ago | (#30723130)

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.

Re:$60m is pocket-money (1)

paiute (550198) | more than 4 years ago | (#30723678)

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.

Re:$60m is pocket-money (1)

Yewbert (708667) | more than 4 years ago | (#30723722)

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 producers of those things aren't required to prove:
  that they work;
  that they have a consistent strength/dosage across production lots;
  that they aren't adulterated with uncontrolled substances not related to the API;
  that they are safe.

FDA-governed pharmas are required to show all those things, and to a degree far past the diminishing returns of effort that you'd find if we were required to meet *only* a 99% consistent result, and that's only at the point where real production is underway.

Final-phase clinical trials are expensive enough, requiring as they do statistically significant cohort sizes, medical professionals to run them, teams of doctors and statisticians to understand and interpret the results and a huge infrastructure to supply the API in the relatively tiny CT quantities, built despite the significant risk that it could all amount to nothing even having gotten through all the earlier stages of development.

Earlier phase testing isn't qualitatively much different, though there are some interesting expenses that most lay-trolls don't know about, like animals used for various types of studies. Since you have to study under laboratory conditions, you have to buy animals bred explicitly for the purpose. A single monkey can cost $50,000 just to purchase, and again, you need a statistically significant number of them to run a study.

But it's so much easier to dismiss the complexity and difficulty of the effort and to presume that no one involved in the process is doing any sort of earnest job and just say it's all bullshit and greed.

Side effects? (4, Interesting)

Lord Lode (1290856) | more than 4 years ago | (#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:Side effects? (0)

Anonymous Coward | more than 4 years ago | (#30721504)

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. He might be able to button his own shirt or wipe his own ass, though.

You're exaggerating (2, Insightful)

jonaskoelker (922170) | more than 4 years ago | (#30721658)

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 [businessweek.com]

Or win the Nobel Prize in economics; see http://en.wikipedia.org/wiki/Vernon_L._Smith [wikipedia.org] and http://www.econtalk.org/archives/2008/03/vernon_smith_on_1.html [econtalk.org]

(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.

You're trivializing (2, Insightful)

Abcd1234 (188840) | more than 4 years ago | (#30723086)

(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.

Re:Side effects? (2, Insightful)

JasterBobaMereel (1102861) | more than 4 years ago | (#30721550)

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 ....

Re:Side effects? (1)

Lord Lode (1290856) | more than 4 years ago | (#30721696)

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 could imagine a cure, but I think in some cases it isn't a cure and a person with totally different abilities than regular people, even if these abilities prevent them from living a normal live, should be allowed to keep living with this special ability instead.

Re:Side effects? (2, Interesting)

JasterBobaMereel (1102861) | more than 4 years ago | (#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:Side effects? (0)

Anonymous Coward | more than 4 years ago | (#30721616)

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?

Being extremely good at something is genius, not autism.

Re:Side effects? (1)

TaoPhoenix (980487) | more than 4 years ago | (#30722568)

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.

Re:Side effects? (1)

ShakaUVM (157947) | more than 4 years ago | (#30722620)

>>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.

Cost? (1)

TubeSteak (669689) | more than 4 years ago | (#30721388)

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"

fragile x is creepy (1)

circletimessquare (444983) | more than 4 years ago | (#30721592)

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 generations. but the more your number of repeats in the vulnerable region, the greater your chance of having children with fragile x

so fragile x is not something like huntington's, where inheritance is straightforward and pat. in other words, any dilemmas a mother or father who is a carrier for huntington's may feel is the same for that person's grandparent, and its the same for that person's grandchild: its a constant across generations

but instead, with fragile x, you have to consider that your mild number of copies may be amplified through the generations. you have a greater risk than your grandparents, and your grandchildren have an even greater risk still

this creepy pattern of inheritance actually has a name: the sherman paradox

http://en.wikipedia.org/wiki/Sherman_paradox [wikipedia.org]

A bargain (1)

paiute (550198) | more than 4 years ago | (#30721762)

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.

Me to (0)

Anonymous Coward | more than 4 years ago | (#30721842)

have one of these startups that test drugs... honestly...

Fragile X is not an autism spectrum disorder... (1)

jcaplan (56979) | more than 4 years ago | (#30722930)

... and a few other comments.

First is the whole autism connection. First, the Slashdot headline and summary are completely misleading. The treatment is aimed at fragile X syndrome, not autism. Fragile X syndrome is NOT an autism spectrum disorder. Some people with fragile X syndrome are also diagnosed with autism, but given that people with fragile X syndrome suffer mental retardation, the diagnosis of autism in these people becomes complicated. To be fair the article isn't quite as bad as the Slashdot summary on this point, but it is definitely misleading.

Second, the article describes the role of FMRP (Fragile X Mental Retardation Protein) as "to inhibit molecular activity at the connections between nerve cells." This is so simplified as to be completely uninformative. FMRP is a regulator of mRNA. (For the non-biologists, mRNAs are essentially copies of genes that get used as templates by ribosomes to manufacture proteins.) Many mRNAs bind to FMRP. This usually reduces their ability to get transcribed into proteins by ribosomes. The result is that certain proteins, especially ones that are involved in growing synapses get over-expressed. One of the striking characteristics of the brains of people with fragile X syndrome is that they have elongated dendritic spines. (The dendritic spines are the structures that form the "receiving" end of the synapse.)

Finally, I'm skeptical of the researcher's technique of treating the syndrome by simply reducing mGLuR5 expression for two reasons. First FMRP regulates the expression of many proteins, not just mGLuR5. Second, the role of FMRP is activity-dependent. The whole point of controlling the expression levels of certain proteins in the brain is not to have them the same in every synapse, but to allow the activity of the brain to regulate the strength of the synapse. Bear's treatment may reduce seizures and even result in fewer elongated spines, but that may not relieve the mental retardation significantly if the people with fragile X still have difficulty properly regulating the strength of their synapses in response to activation. It is possible, though, that this could work and that just by getting the levels of mGluR5 into a more normal range that other mechanisms could compensate for the other mis-regulated proteins; the brain is a fairly robust system. If this works, it would be wonderful and could make meaningful improvement in the lives of many people.

A cure, not management (1)

assertation (1255714) | more than 4 years ago | (#30723154)

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 mean some reach person would have to pay money and change the way s/he did things.

Interesting drug for Fragile X. But autism? (1)

TheMohel (143568) | more than 4 years ago | (#30723324)

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 a simple inheritance model, and the expression of the gene is quite confusing. She's a superstar.

As far as the broader issue of autism (and even more confusingly, autism spectrum), Fragile X has always seemed to me to be a blind alley. People with Fragile X (I've worked in that community as a physician) have a very specific affect and behavior pattern that doesn't look a lot like the behavior of people with autism (a community I know all too well as a physician and a parent of an autistic young man). Most of the early research in autism was tainted by the inclusion of Fragile X patients, and most of the combined research is just confusing.

I hope that the drug proves useful in Fragile X, although pharmacotherapy for these kinds of disorders has frustrated us over and over again. These are simply very hard diseases to affect very much. At the least, though, it'll be another step toward understanding a serious disease. And I'll continue to wait and watch for anything that will help in autism, but I REALLY don't expect much from this specific drug.

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