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16-Year-Old Discovers Potential Treatment For Cystic Fibrosis

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.'"

16 of 236 comments (clear)

  1. He will shortly find himself in court... by mckorr · · Score: 3, Insightful

    as Big Pharm sues him for using their drugs in a manner not properly prescribed. This will effectively lock him down while they rebrand the drugs, package them, and patent the cure for their profit.

    1. Re:He will shortly find himself in court... by devleopard · · 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.)

      --
      The best thing about a boolean is even if you are wrong, you are only off by a bit.
    2. Re:He will shortly find himself in court... by devleopard · · Score: 3, 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/

      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)

      --
      The best thing about a boolean is even if you are wrong, you are only off by a bit.
  2. Re:Great Expectations by Beardydog · · Score: 4, Funny

    Notes:
    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.

  3. I have Cystic Fibrosis, and this rocks by devleopard · · Score: 4, 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)

    --
    The best thing about a boolean is even if you are wrong, you are only off by a bit.
    1. Re:I have Cystic Fibrosis, and this rocks by pz · · Score: 3, 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.

      --

      Put my fist through my alarm clock with its ding-dong death inside my ear. - The Blackjacks.
  4. Re:My High School sucked by Ecuador · · 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.

    --
    Violence is the last refuge of the incompetent. Polar Scope Align for iOS
  5. Re:My money is on him winning that science fair. n by Anonymous Coward · · Score: 3, Insightful

    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 candidate for suppression.

    In this particular case, however, the treatment involves the use of two existing drugs, so there's really no profitability to discuss.

  6. Re:My money is on him winning that science fair. n by KUHurdler · · Score: 3, 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.

    --
    Fix Your Own TV - RiddledTV.com Avoid the Landfill
  7. Re:My money is on him winning that science fair. n by MrBippers · · Score: 3, Interesting
    You clearly misunderstood the post you're responding to, or are yourself, an

    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 statement the company would buy his compound and it would never see the light of day, thus garnering your idiot comment. While it's not what he meant, it is in fact also common practice in pharma for companies to license the rights to compounds similar to those they are developing just to eliminate potential competition. It's why often when licensing a compound stipulations are added that the purchasing company must intend to develop it.

    All of this is likely moot as the kid does not own the rights to the compounds. TFA doesn't specify whether they are novel but my guess would be he worked with a library of existing compounds that showed some activity against cystic fibrosis in preliminary screenings.

  8. Re:My money is on him winning that science fair. n by gstoddart · · Score: 4, Insightful

    Yes, if its one thing they are know for is not providing new drugs.

    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.

    --
    Lost at C:>. Found at C.
  9. Totally Overated Pseudo Research by Wdi · · 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.

    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.

    1. Re:Totally Overated Pseudo Research by Wdi · · Score: 3, 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!

    2. Re:Totally Overated Pseudo Research by gordo3000 · · 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.

    3. Re:Totally Overated Pseudo Research by Wdi · · Score: 3, 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.

  10. Re:Automated testing ? by lbgator · · Score: 3, Informative

    In that vein, FoldIt 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.