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 I have the urge to get a first post?
The best way to ruin your hobby is to try to make a living at it. Waiting on the paperless office since 1997
...I fear this is more about big pharma milking scared people than about saving people.
Smile, don't click...
i sure hope autism isn't something that is more-or-less cemented at birth, making drugs like these not very useful.
Beware the Jubjub bird, and shun the frumious Bandersnatch.
What if it's the people who don't have Autism who are sick? What if it's 'normal' that's wrong?
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)
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?
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"
[Fuck Beta]
o0t!
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!
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
intellectual property law is philosophically incoherent. it is your moral duty to ignore it or sabotage it
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.
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.
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.
If Slashdot were chemistry it would look like this:Cadaverine
have one of these startups that test drugs... honestly...
... 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.
(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.
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.
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.
I notice a number of other industries are copying Apple's iPhone App commercials now. This would work perfectly for the drug industry: "Have this disease? theres a drug for it!".
This paradigm is so deeply ingrained in the American psyche now that I automatically wonder about some new medicine for all the new disease syndromes we hear about. There are substances for mental health and physical fitness. Even herbal remedies are squished into pill shapes to make them seem more "medical".
Autism vaccine is linked to flu in children. News at 11.
people with autism have common genes, both originally and epigenetic expressions
http://hmg.oxfordjournals.org/cgi/content/abstract/6/13/2233
this one is still inconclusive consisting of repeated studies that disagree
this one seems pretty clear
http://www.ncbi.nlm.nih.gov/pubmed/16103890
doesnt make it a disease. hey i reckon many social 'norms' are diseases; cause harm and are certainly pathalogical. I think the best medicine for autism is cannabis, it helps them enjoy, learn and benifit/constructively utilize, being "stuck in their brain" and "thinking too much" ...
The dangerous case with your note is that it's result dependent.
"Spends all his time with math" ... and writes semi-pro papers, tutors a kid, and writes little freeware puzzles to amuse the net hordes. Win.
"Spends all his time with math" ... and tries to figure out the numerology of life, but crucially, gets the worst of new age with none of the science of emergence. Then he's total bait for medical pigeonholing. Too smart for "ordinary" types, but too flawed to play the "strange genius" card.
My first Journal Entry ever, in 8 years! http://slashdot.org/journal/365947/aphelion-scifi-fantasy-horror-poetry-webzine
Autism is purely based off of a person ingesting Mercury, then storing that nuro-toxin, becoming infertile, losing mental capacity, and then passing it on through his sperm/her egg, and THEN shooting up a newborn with 6-16 NEW shots, ALL with Mercury, Thermisol* and Squaline*.
Wake up...if we did not ingest it in the first place, then autism rates would never have gone up!!
I'm a test subject in a trail.
From what I understand (from my doctor) autism is thought to be genetic but it does have certain environmental triggers that affect whether or not you develop symptoms and to what extent. One of these triggers is exposure to the measles virus (the vaccine is an attenuated form of said virus). Apparently a person's body develops inappropriate antibodies that attack certain parts of the brain (Amygdala) after exposure to one of these triggers.
The neurotransmitter Oxytocin (which is what I'm trying BTW) is also thought to be malformed in people with autism along with the receptors in a that person's brain.
The Oxytocin supplementation has helped greatly but it doesn't fix everything.
Biology (hard science) has a constructive approach going from the ground up - chemicals and physical operations.
Psychology (soft science) is about identifying trends and describing them clearly to other experts; something like anthropology or sociology does. Attempts to deconstruct these trends using other more "atomic" trends as well as finding interdisciplinary connections is extremely difficult and builds prestige; however, these often amount to being temporary fads that are later found to be weak or false or specific to a group/culture/time. Not to undermine the work, humans will never have easy explanations.
Here we have Autism and Asperger's which are placed on the same spectrum merely because they have some outward common traits in their generalized diagnostic definitions. Neither is well understood and classification at the mild end of the spectrum is difficult for professionals who are trained to have an eye for such things. Even when understood fully-- its psychology and one shouldn't expect the biology to ever align with it - Asperger's is often described as developmental in which case, it may have much more to do with nurture than nature. Autism might actually be 5 different things that produce somewhat similar results creating this so called "spectrum" which again, has been defined by only observing the resulting behaviors of all of them.
There are many similar examples. Nature being on the constructionist side and Nurture on the developmental side.
One that bugs me is how we linked depression to biology when most of the time it is not biological by psychological in origin - they only connect in a niche case; otherwise, the two fields are not aligned. This misunderstanding has led to many people only drugging the affect of their mental condition to change physical feelings and not the psychological ones (not to mention the increase in messed up people who take these drugs which may not cause direct complications but cause some people to mentally go nuts by failing to treat/experience the actual problem. Reminds me of some form of severe repression...)
Asperger's might be a form of evolution and not related with autism at all. Who knows. Just thought I'd throw out that idea ... if humans evolve - how would we react? (obviously, we'd label them with some syndrome... note I said "evolve" not wanting to create a thread about our devolution.)
Democracy Now! - uncensored, anti-establishment news
Elizabeth Moon saw this coming. Speed of Dark is an excellent novel about an autist who is coerced into taking treatment against adult autism, though the book is set a bit further in the future from now (2030's, I'd guess, the story doesn't specify exactly).
(I read it recently on recommendation of another /. comment)
And if you think ADD is nothing like Asperger's... why not look at the official DSM-IV Criteria
How about we also go looking at differential diagnosis, then?
From http://apt.rcpsych.org/cgi/content/full/7/4/310
The conditions differ in that ADHD lacks the classic impairment in reciprocal social interaction, narrow interests, repetitive routines and non-verbal problems of Asperger syndrome.
Often talks excessively.
vs
lack of social or emotional reciprocity and stereotyped and repetitive use of language or idiosyncratic language
A kid with ADHD will talk about trains all the time. A kid with Asperger's will talk in weird ways about trains all the time. Definitely, def-definitely all the time.
Is often easily distracted.
vs
encompassing preoccupation with one or more stereotyped and restricted patterns of interest that is abnormal either in intensity or focus
Erm... what? One says "doesn't focus for more than 20 seconds", the other says "focuses for 20 days in a row."
Diagnosed aspie here. Mod parent up!
Also, to provide some evidence to backup the claim about cures scaring the shit out of us, have a read through some of the posts on this forum, particularly under "media representation".
Our culture doesn't get smarter, it just finds new ways of being retarded.