16-Year-Old Discovers Potential Treatment For Cystic Fibrosis 236
Bob the Super Hamste writes "According to a story at LiveScience, a 16-year-old Canadian 11th grade student has discovered a possible treatment for cystic fibrosis. The treatment is a combination of two drugs which, in a computer simulation on the Canadian SCINET supercomputing network, did not interfere with each other while interacting with the defective protein responsible for the disorder. He has also tested the drug combination on living cells with results that 'exceeded his expectations.'"
My money is on him winning that science fair. n/t (Score:2)
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Re:My money is on him winning that science fair. n (Score:4, Insightful)
Nah. Some kid who tested 6 different denture adhesives in Coke will win because the judges actually understand WTF he did. At least that's how it worked at science fairs when I was in school.
You probably should have learned how to explain your volcano better.
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How are you holding up? Because I'm a potato.
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From the article:
"Now Zhang and a trio of Montreal students who took second place for their technique for making sorbet without gelatin move on to compete against U.S. and Australian teams at the International BioGENEius Challenge in Washington, D.C., June 27."
Man, the second place team is looking pretty pathetic now...
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This *was* in fact part of a science fair of sorts.
I happened to see him presenting to the judges at the NRC here in Ottawa.
His presentation was one of several very impressive science projects.
It was the "Sanofi/Aventis Biotech Challenge", an annual event that calls up science projects from across the country and culminates in a trip to Ottawa and presentations to a panel of judges.
Go Canadian science-interested youth! woot!
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My money is on this being fake.
16 year olds don't have access to super computers nor a clue about how to find one.
Nor do they know what drugs do which things, (street drugs excepted of course).
The relationship with the "mentor" company, Sanofi-Aventis apparently suggests there is
some angle being played here by the mentor company, and I'm willing to bet it has
something to do with patents.
The story doesn't pass the sniff test.
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Yes, if its one thing they are know for is not providing new drugs.
Idiot.
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They are known for providing profitable drugs, and suppressing unprofitable drugs, or more accurately, drugs that interfere with the profitability of other drugs. For example, a drug that treats the symptoms of a disease, and needs to be taken for the duration of the patient's lifetime, would likely be a profitable drug. A drug that cures the disease with a single dose, while perhaps somewhat profitable on its own, would be devastating to the profitability of the first drug, and would therefore be a candida
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You're an idiot. Let's break down how:
1) Life expectancy for people with CF is ~40-45 years today. WITH modern medical care.
2) The reportedly successful drug cocktail of two already-existing drugs:
a) Would extend the lives of those people with CF by years - meaning more sales over time;
b) Would require $0 new res
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is that 44-53% absolute or 44-53% vs the best currently used drug or best currently used drug out of patent?
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http://en.wikipedia.org/wiki/Escitalopram [wikipedia.org] would be a perfect example. Citalopram's patent expired, so they found a new way to encapsulate the drug so that they could continue to sell the patented version. The new drug works exactly the same way that the old drug worked, has exactly the same mechanism, and a nearly identical success rate in clinical trials, but because it's encapsulated, it's technically a different drug, so they were able to renew the patent. I would lay odds that they have already developp
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Its very easy to see this happening all the time. Its been happening over the last 10 years or so with all those new-fangled anti-depressants. You can get the brand name one in capsule form or the Generic out of patent one in pill form.
I forget the two it was, but I had a shill doctor prescribe the first one and then a real doctor prescribe the cheaper, same effectiveness, generic.
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You can still get the generic of Celexa.i.e. Citalopram'
For someone who spouts off, you could at least know that Citalopram is the generic.
Medical professionals? that' term is so wide, it's non sense. Did nurses? Drs? or was it lobbied by the sales side of a pharma company?
Look, the pharmaceutical hires the best scientists in the world. They also hire the best sales people int he world.
They don't make a lot of profit, and the produce and sell some thing near cost becasue of the public good.;
They are very co
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Idiot.
The poster implied that he would sell the rights to a pharma company and indeed licensing compounds from smaller companies/research labs is indeed standard practice. If you meant that the pharma companies don't have enough new drugs of their own, this is in fact wrong.
The second part of the post implied the kid would never be heard from again. If he made enough money it's possible. I'm guessing you misinterpreted this as a st
Re:My money is on him winning that science fair. n (Score:5, Insightful)
No, just drugs that they either didn't adequately test ... or that they selectively dropped the results indicating that they gave you a higher likelihood of killing you.
While Big Pharma does crank out drugs, they're not exactly showing a stellar track record of actually making sure they're safe. They mostly assume they're safe if it doesn't kill you in the first few weeks.
And, then of course there's the constant commercials for a drug you should "ask your doctor about" -- sometimes they don't say what it treats, but they give a litany of side effects which sound like you'd need to be desperate to try. So, when a patient goes into a doctor insisting they should get some astra-awesome-a or something, the doc just writes a scrip of gives out the free samples the sales rep dropped off.
You'll excuse us for not attributing any concern for our welfare to these companies. They're like the tobacco industry in a lot of ways ... it's in their interests to tell you their product is perfectly safe and didn't kill more than half of the 100 rats they tested on. At least, not right away.
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And, then of course there's the constant commercials for a drug you should "ask your doctor about" -- sometimes they don't say what it treats
That would be the commercials intended for the Canadian or international markets.... in Canada, it's not legal to advertise for drugs, and the way around that is to avoid mentionning both the drug name and what it treats in the same advert. On the other hand, that legal loophole has led to some of the more amusing Viagra and Cialis commercials out there...
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He will be disqualified from the science fair for using living cells, chemicals and drugs. He will then be expelled and arrested in accordance with the schools zero tolerance policy.
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Maybe the science fair has been replaced by an intelligent design fair already?
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I don't know, I heard some kids at the same fair got a pig and an elephant to make love.
That was the senior prom.
(I'm sorry, I'll shut up now)
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I don't know, I heard some kids at the same fair got a pig and an elephant to make love.
Ridiculous! Haven't you ever heard that song by Loverboy?
Great Expectations (Score:2)
He has also tested the drug combination on living cells with results that 'exceeded his expectations.'
This may or may not be impressive depending on what his expectations were. Hopefully they were higher than "causes massive trauma to healthy tissue," where "causes significant trauma to healthy tissue" would exceed expectations.
Re:Great Expectations (Score:5, Funny)
11:45 am - Upon administration, injection site immediately burst into flames. Combustion of patients blood followed, with progressive explosive rupturing of all blood vessels in a pattern emanating from injection site. End-stage release of parasitic alien spores ( from eyeballs ) noted in earlier formulations has been reduced to a degree exceeding expectations. Recommend further human trials to determine ( presence of? ) risk factors for blood combustion.
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I couldn't help but notice that he used a super computer as a part of his project. Call me old fashioned, but I really don't like this use of technology to avoid actually getting your hands dirty. It just strikes me as throwing money at a problem whereas in the past the actual displays were a lot more interesting, as they'd actually be able to show more than just print outs and diagrams.
That being said, I wouldn't be surprised if there was parental involvement, that's always been a buzz kill, especially if
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He's got tenacity, though.
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Yeah. People with their computers these days. You should have to create your own molds, to create your own types, to use with your hand-built printing press.
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He will shortly find himself in court... (Score:4, Insightful)
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does that work in Canada too?
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We buy the same drugs, from the same companies, and usually once the USFDA approves it ... Canada just rubber stamps it for use.
So, yeah. Pretty much.
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Except those same drugs from the same companies cost far less and still produce huge profits for the pharma companies. Fancy that.
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That is because those drug purchases are subsidized by the government via taxes collected. The drug companies don't charge less because the purchasing agent is Canadian.
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yes they do. it's well proven that drug prices in the US are several time more expensive than other countries. I know for one particular example (as my friend worked at the implant company) the Japanese govt remitted 10% what they received for a sale in the US from our insurance companies (and Medicare). I've seen margins between 50-75% less on various drugs.
Collective Bargaining (Score:2)
That is because those drug purchases are subsidized by the government via taxes collected.
Wrong - it is because the federal government, in combination of the provincial governments, do a collective bargin with the drug companies so that all of Canada gets one price for a drug. This works out very well because the federal government controls the laws on patents and damages and can threaten greedy companies with changing the law to allow canadian companies to made copies of their drugs. Of course this would come at a large political price but at least they have a large enough stick to threaten th
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GASP! Communism!
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Oh, so you shift the burden of payment to countries that don't do this. I love paying for Canadian health care here in the US!
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Except those same drugs from the same companies cost far less and still produce huge profits for the pharma companies. Fancy that.
The produce "huge profits" because those who are in non-subsidized countries (ie, the US), actually pay for it
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Pharmaceutical companies don't make a lot of money.
I'm not sure why people think that. I suspect they confuse revenues with profit.
That's actually not true. The pharmaceutical industry makes a lot of profit. For instance, a little googling shows that according to Fortune they were the third most profitable industry in both 2008 [cnn.com]
and 2009 [cnn.com]. If you keep going back you'll find that they've been quite profitable for a long time.
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Not true. Most drugs that're approved in Canada are also approved in the US, but there's several drugs that are approved in the US that are not available in Canada (including a few that've been pulled from the shelves for being dangerous in the US, which were never approved in Canada specifically because of those dangers), and similarly some drugs are available years earlier in Canada than the US. One of the drugs I'm taking right now is on that list... it's been available in Canada for almost a decade but
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Yes, hence my ingenious usage of the word usually ... which allows me to express a subset of all, while still being most -- but nowhere near just a few, or even almost none. Also, not to be confused with the very vague "some of the time".
Because I'm sneaky like that.
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Re:He will shortly find himself in court... (Score:5, Interesting)
Maybe, but not necessarily. CF isn't a huge profit center like heart disease medications or even HIV. Even though CF is the most common chronic genetic condition in the US, the numbers just aren't there. Most of the major CF meds (Pulmozyme, Creon, Tobi, Cayston, etc) is given away by the pharmas when the patient can't afford. While it may not be true for other conditions, when it comes to CF the pharmas ensure that those who need their meds get them. The emphasis for profit in CF just isn't there.
I should know - I have Cystic Fibrosis, and despite periods of no insurance, I've never done without. (Yes, I'm in the United States.)
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Wouldn't it be 300 thousand people? The article cited 1 in 3000.
Re:He will shortly find himself in court... (Score:4, Insightful)
There are about 30,000 CF patients in the US. I've seen numbers that say 1 in 3500 are born with CF, but a number possibly die shortly after birth. Note that numbers vary by ethnicity, so statistics may vary by how categorized.
http://www.cff.org/AboutCF/ [cff.org]
30,000 is a very small number to build a market on when compared to diseases/syndromes like MS (400,000), breast cancer (200,000+ new cases in 2010), HIV (over 1,000,000). (US only)
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And yet ... you can get sued for an upgrade [slashdot.org] button.
The way patents are used now ... they do anything but promote innovation. They're mostly used to stifle competition, and seek to extort licensing fees for something obvious.
They can also patent genes that people are born with. I'm afraid I no longer believe that patents serve the purpose they were intended to.
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But doctors can already approve drugs for another use (off-label use) if they're already approved for one use.
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If they can patent genes which exist in humans naturally ... why not sue for running a computer simulation that uses their patented compound?
I don't know that one is any more absurd than the other.
The way research is going, it will be illegal to research anything since the lawyers will have laid claim to everything, or patented a "system of examining the natural world using hypothesis and experimentation to arrive at predictive statements".
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Bastard! ;-)
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No problem, just pay up in monopoly money. Simulated use = simulated payment.
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or patented a "system of examining the natural world using hypothesis and experimentation to arrive at predictive statements".
Sounds like a plan...I have to contact a patent attorney...thanks for the idea!
My High School sucked (Score:3)
Re:My High School sucked (Score:5, Funny)
While we did not have drug trials at school, just outside our school there was a little park where you could sometimes find syringes from whatever drug research activities were going on overnight.
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What? You didn't do your own drug research in school?
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Yea I was thinking the same thing when I heard a kid going to the Intel science fair saying, "I have been doing research in applied Physics for the last five years." Freak at 12 I had to get the principle's permission to launch a model rocket at school! Man things seem to be getting better but it makes me feel old.
A bit of perspective (Score:2)
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I have Cystic Fibrosis, and this rocks (Score:5, Interesting)
As a 34 year old dealing with the health issues and the ridiculous costs that let me breathe, digest my food, and not be knocked on my butt by blood sugar spikes, I'm excited by this. Goes to show that sometimes we just need some fresh thought at a new problem - the traditional, mega-millions research methods may not be the answer. (similar to Space-X :: NASA)
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Well, the drugs interact on their intended targets without interacting with each other. Given that both are safe on their own, the odds of them causing problems together in a more complicated system are lower than it otherwise might be.
Re:I have Cystic Fibrosis, and this rocks (Score:4, Insightful)
As a 34 year old dealing with the health issues and the ridiculous costs that let me breathe, digest my food, and not be knocked on my butt by blood sugar spikes, I'm excited by this. Goes to show that sometimes we just need some fresh thought at a new problem - the traditional, mega-millions research methods may not be the answer. (similar to Space-X :: NASA)
Uhm, this kid -- talented though he may be -- was doing a summer project in a regular old lab (run by Dr. Christine Bear, at the Hospital for Sick Children's Research Institute, according to the article) that was, according to their web site, funded by Canadian and US governments and private foundations. More importantly, it would appear to be the very essence of incremental research to think that one might try combining two effective drugs together to see if the combination works better. That's not what I would call a ground-breaking, radical new idea; combination therapy is so common that we have a nifty term for it (that is, "combination therapy"). This is traditional, and effective, research.
I've mentored a few of projects like these in my lab, and, when I was younger, participated in a couple myself. Sometimes the students are really smart and do a lot of independent work, and sometimes they are spoon fed the entire way. But, again in my experience, all of the major ideas are entirely provided to them. High school students really don't have enough experience to understand what the big questions are; that's the role of a lab head. Kudos to this kid, for sure, but saying that it's a new fresh perspective, or some non-traditional methodology, is probably off the mark.
Stiff competition (Score:2)
Totally Overated Pseudo Research (Score:3, Insightful)
Sorry, but this is *not* any innovative science. Rather, it is a computational reproduction of facts already well known. Nothing more than a typical molecular modeling class assignment during a graduate chemistry education.
He did not invent any new drugs - the really breakthrough was by the researchers of Vertex Pharmaceuticals, see for example VX-770 [cff.org].
He did not discover the mechanism of action of the drugs. Rather, he took published protein structures and published compounds and re-ran some docking studies (of the same type Vertex and other pharmaceutical companies probably spend hundreds of thousands of processor hours on, with the difficulty that they had to check tens of thousands of compounds, not just two already known to work).
He was not the first to notice that different promising compounds in clinical trials have different points of interaction with the defective proteins of CF. Thinking that a drug combination may be useful is not exactly a new and brilliant insight, and this was for example even discussed a couple of months ago in CE&N (the general chemistry member journal of the American Chemical Society). I am very confident that is has been evaluated before, and probably there are patents already filed.
The only interesting point here is that the guy is 16,not 20 or 22 like the normal chemistry student. But then pressing the right buttons in a molecular modeling software is really not that difficult, especially when you already know the outcome you want to reproduce.
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To be fair we are talking a Canadian education system not American... :)
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He did not discover the mechanism of action of the drugs. Rather, he took published protein structures and published compounds and re-ran some docking studies (of the same type Vertex and other pharmaceutical companies probably spend hundreds of thousands of processor hours on, with the difficulty that they had to check tens of thousands of compounds, not just two already known to work).
He was not the first to notice that different promising compounds in clinical trials have different points of interaction with the defective proteins of CF. Thinking that a drug combination may be useful is not exactly a new and brilliant insight, and this was for example even discussed a couple of months ago in CE&N (the general chemistry member journal of the American Chemical Society). I am very confident that is has been evaluated before, and probably there are patents already filed.
(Emphasis mine)
Thank you for so clearly demostrating what's wrong with the Pharmaceutical industry. First they brute-force through computer simulations looking for combinations that might work, then they file patents on those results as if they had done any actual research, and then just to add salt to the wound they don't even bring them to market and into the hands of patients or this kid wouldn't have even tried to do this experiment in the first place.
But then again, this is the kind of industry that bl
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Thank you for so clearly demostrating what's wrong with the Pharmaceutical industry. First they brute-force through computer simulations looking for combinations that might work, then they file patents on those results as if they had done any actual research, and then just to add salt to the wound they don't even bring them to market and into the hands of patients or this kid wouldn't have even tried to do this experiment in the first place.
But then again, this is the kind of industry that blackmails governments for a living and even patents freakin' DNA so really, it shouldn't be surprising.
You are mistaken. The Vertex compounds are now in phase 3 clinical trials and will be marketed if nothing bad shows up (and if it did, it would be a financial disaster, Vertex and various foundations spent well over 100 mil USD on this project). Drugs without FDA approval (or the Canadian equivalent) cannot yet be bought in a pharmacy, that is the law. But be assured, Vertex certainly wants them on the market - otherwise there will be no recovery of expenses, and later proft.
Finally, drug combinations can o
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I have MOD points and right now I want a moderation selection that says, "You'd Shit on your own mother!".
Jesus fucking Christ dude, a 16 year old, yes that is a KID, a junior in HIGH SCHOOL and you go out of your way to belittle his accomplishment!
Yes that is how we motivate the young and the obviously gifted to excel in science by crapping all over their accomplishment.
Look up the definition of asshole in the dictionary and you will find your name, address and photo.
Ohh yes and the people who modded you
Re:Totally Overated Pseudo Research (Score:5, Insightful)
I'm sure the kid, who was working in a lab that has been doing CF research for quite a while, is well aware that he probably just recreated experiments done before.
If it was discussed a few months ago in a general journal (I'm not sure, but let's assume the parent is correct), it's more likely this was known several months ago in the specialized academic journals that the lab professor would read. So it's far more likely that he is an ambitious student who found a professor to tell him what to do so he could gain experience, not the inventor or discoverer of a cure.
It's not shitting on him to say he didn't put this together on his own. It's just the truth.
Why does everyone need to be called a genius in order to feel special enough to work hard in an industry? Have you done research before in a lab? If you had, you'd know that while the GP might be crass, it's exactly the most likely scenario.
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No need to foam.
I already praised his efforts in another post. He did well, spent a lot of effort, learned something, and hopefully he will be motivated to study chemistry in university by this experience.
But he did not perform original research, and certainly did not find a cure for CF. He reproduced stuff well known among professionals. He certainly did not discover anything they overlooked.
Its the hype in the article and the Slashdot summary I am protesting against.
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No he's not an asshole. He's being realistic, and he's trying to prevent people suffering from this disease from getting their hopes up too high. The fact that a smart kid did well at a science fair is not front page news (except on slashdot).
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Jesus Christ, man. You put Buzz Killington to shame.
Re:Totally Overated Pseudo Research (Score:4, Informative)
The Vertex people did, and they rightfully received much praise for their results (these CF compounds are without precedent, providing a treatment option for a deadly disease).
But this has all been published, extensively, even in non-specialist journals (CE&N). *EVERY* professional chemist with a minimum interest in pharma research knows about the Vertex compounds, the different interaction points with the proteins, and the possibility of drug combinations.
Reproducing these results is a nice coursework problem, but not research. The novel results produced by what the guy did are ZERO. I am certain this project was a nice experience for him, and it may hopefully motivate him to study chemistry after finishing school. I wish him the very best for his further career.
But HE DEFINITELY DID NOT INVENT A CURE. Stating anything like that is ridiculous hype!
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Yeah, but this is not how Slashdot works.
0 score for you! (not from me, obviously)...
Re:Totally Overated Pseudo Research (Score:4, Insightful)
There are X-ray structures of the protein(s) in question, with and without bound ligands. All published long before this guy started his work. And the drugs were designed by performing docking computations on the protein with the structure, and the most promising candidate structures were synthesized, binding verified, and some of them again solved as crystal structure. By looking at the docking poses, the structures were further refined by re-designing parts to better fit the protein.
Many posters here are assuming he worked on his own, brilliantly and single-mindedly breaking a new path. He did not. He worked in an academic lab and was tutored. The prof was of course well aware of the state of research. He had a bright pupil interested in learning about doing research. So he offered him to learn some of the tools of trade, on a realistic sample problem with known outcome. The guy went to work, learned a lot, made a nice poster (for a highschol student competition, not a scientific conference), and won a price for it. All very laudable and a nice achievement, but no ground-breaking genius moment.
Just to re-iterate it, the guy did not find anything which was not already known and published. He used known protein structures, known drug compounds, known binding sites, known ideas for combining drugs. So there was no original scientific research, and absolutely no novel cure, just ridiculous hype by the press. The guy mastered at age of 16 skills chemistry students are routinely acquiring at 22. Nice, but not world-shaking.
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Um, no. Jerry Lewis = MS, not CF.
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Try MD.
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I really wish there were a drug to cure (or at least stabalize) Sour Grape Syndrome (SGS).
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I listened to him present his results and you sir are a cynic.
No doubt he had significant help and guidance from his mentor. A project like that is a learning experience.
He demonstrated an understanding of the science involved as well as interest and enthusiasm for the research.
He also worked very hard on it and his efforts are worthy of commendation.
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Who appears to do this sorta thing professionally. The 16 year old should be given a BIG pat on the back for contacting a LONG list of scientists to find one that would work with him. Unfortunately, the news industry really likes to play up the whole "Kid Genius" story, since it make all of us feel old, and useless. :)
I doubt they'd have as many people passing around the article if it focused on a normal research group working as intended and including (as is typical) young mentees in the process so that they can learn about the research process.
Re:Automated testing ? (Score:4, Informative)
In that vein, FoldIt [fold.it] is a game where the goal is to make proteins that match target sites. Promising results get tested in labs. Same gist as what you suggest, but you get humans to play tetris instead of a computer trying random proteins.
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