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Medicine Science

Crispr Wins Key Approval to Fight Cancer in Human Trials (bloomberg.com) 71

Tom Randall, reporting for Bloomberg Technology:An experimental cancer treatment that alters the DNA of patients has won a key approval to proceed with its first human tests using the controversial gene-altering tool known as Crispr. Scientists from the University of Pennsylvania want to edit the immune systems of 18 patients to target cancer cells more effectively. The experiment, backed by internet billionaire Sean Parker, won approval from the Recombinant DNA Advisory Committee (RAC), a federal ethics panel set up at the National Institutes of Health 40 years ago to review controversial experiments that change the human genome. The trial still needs final approval from the U.S. Food and Drug Administration. The experiment targets difficult-to-treat cases of multiple myeloma, sarcoma, and melanoma. The scientists will remove blood samples from patients and alter their T-cells -- central to human immune response -- to more effectively target and pursue cancer. The T cells will then be infused back into patients and studied for the safety and effectiveness of the technique.STAT News has an article in which it discusses the probable consequences of altering the DNA of a cancer patient.
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Crispr Wins Key Approval to Fight Cancer in Human Trials

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  • by jedidiah ( 1196 ) on Thursday June 23, 2016 @03:42PM (#52376443) Homepage

    They already have a procedure similar to this where they harvest and grow unmodified T-Cells. They extract them from vicinity of the tumor and then replicate them.

    It's a little bit like cloning Osmosis Jones.

    It looks like either approach is highly custom and not any sort of mass market thing.

    • by XXongo ( 3986865 )

      They already have a procedure similar to this where they harvest and grow unmodified T-Cells. They extract them from vicinity of the tumor and then replicate them.

      That's not really the same thing. What's new here is using CRISPR to edit the genes of the T cells. Gizmodo: http://gizmodo.com/everything-... [gizmodo.com]

    • by The Real Dr John ( 716876 ) on Thursday June 23, 2016 @04:43PM (#52376829) Homepage

      And why is the test of worthiness for a medical procedure whether it can be "mass marketed"? What neoliberal thinking. How about if it is cheap, and effective, maybe then it doesn't matter if it is not "mass marketable"? I find it fascinating that people don't care about solutions to problems if they think they won't make lots of money.

      Crispr/Cas is a very interesting gene editing technology that looks like it is going to replace current methods. But as with all existing methods, getting it to edit exactly what you want, the way you want, is a bit tricky. But it works well enough that it is being used on a wide scale basis to make gene knockout animals and cell lines.

      • by jedidiah ( 1196 )

        Can you think of any other area where "custom" equals cheap"?

        My thinking is more "conservative". How error prone will this "custom" work bet? How expensive will this procedure be? How much individual effort will it require from highly expensive specialists that are in limited supply?

        A generic solution allows for manufacturing and quality control. It also allows for cheap.

        The current approach that doesn't require gene manipulation isn't terribly scalable.

        • CRISPR/Cas9 is generally not very error-prone, and they're searching for more specific variants quite often. Penn recently put a lot of effort and money into setting up a robotic assembly line-type facility, so if they can get that working, each patient shouldn't require much effort from specialists. It'll probably still be expensive, of course, but so far the CAR T cell therapies have also been very effective.
      • why is the test of worthiness for a medical procedure whether it can be "mass marketed"

        Because someone has to pay for the research and development - which, please remember, involves large-scale clinical trials to get regulatory approval - and they're not going to front the money for a treatment that has no chance of recouping their investment, unless they have some other personal interest. You can wring your hands all you want about society's priorities, but new medical procedures aren't magically exempt f

      • I had an internship at a place that did research bringing things like this to market. The problem isn't just marketability. The problem is being able to reproduce something. There is a big gap between what one very smart person can do in their lab, and what a factory can produce. A technique that can't be mass produced can't help the masses. Profit is obviously a motive, and that's an entirely different discussion. That being said, a lot of really interesting research is lost because it can't be made into
        • That is the problem with capitalism and making everything contingent on being marketable. I wonder if corporations have so thoroughly brainwashed people that now people cannot conceive of doing anything that isn't mass marketable, you know, like sending people to the moon. Of course now, rich people will now be our saviors, and they will build the rockets and send rich people who can afford 20 million dollar tickets to the moon. Sad that everyone thinks in terms of marketability and profit, rather than coop

          • The success of any new product or service has always been contingent on it being marketable. The difference between the golden age of yesteryear and today is that we now try to figure out the viability of success beforehand so that we don't waste as many resources on untenable things, whereas in the past the new thing would come about and then disappear when the business went under. In the long run, there isn't much of a difference for the failed vs never tried.

            Economics is the science (and I use that term
            • No, capitalism sucks worse than just about all other options especially when it turns to kleptocracy and oligarchy. Unregulated capitalism under neoliberal stewardship will eventually collapse under the weight of its own corruption and squandering of resources to make a fast buck. The only question is how long will it take before it implodes. Capitalism needs strong regulation and high taxes to prevent it from killing itself with over indulgence. But that ill never happen under neoliberal rule, where market

    • They're also already doing clinical trials using integrating gene therapy vectors (usually a lentiviral vector) for the same purpose as the proposed CRISPR trial, but CRISPR will probably let them do it more precisely. Both of these approaches can be applicable to specific types of cancer, so it's the same procedure for each patient, using the same reagents, but they're probably specific enough that the market can't really be called "mass".
  • Yea, yea, you're going to post something like "alters the DNA of patients, what could possibly go wrong". But it is backed by a billionaire, so he is going to get to do whatever he wants. Stop complaining and move on, nothing to see here.
  • This sounds like where the T-virus starts...

  • Virus (Score:4, Interesting)

    by SumDog ( 466607 ) on Thursday June 23, 2016 @04:04PM (#52376579) Homepage Journal

    One thing I have to wonder about: If the mechanism involves using a virus, couldn't there be massive unintended consequences if the virus transfers to another host? Even if a virus isn't very communicable, and can't survive outside of a host, what if the patient transmits it sexually after treatment?

    • Re:Virus (Score:5, Interesting)

      by ShanghaiBill ( 739463 ) on Thursday June 23, 2016 @04:23PM (#52376697)

      If the mechanism involves using a virus, couldn't there be massive unintended consequences if the virus transfers to another host?

      Viruses already exist, any many of them do things much more nasty than fixing T-cells. CRISPR is programmed to target a specific sequence of DNA, usually around 40 base pairs. Since each pair is two bits, the chance of this sequence just randomly occurring is around 2^80.

      Even if a virus isn't very communicable, and can't survive outside of a host, what if the patient transmits it sexually after treatment?

      If someone has sex with their identical twin, that twin's cancer may also be cured. Otherwise, nothing will likely happen.
      Far more dangerous DNA modifications are happening naturally on a nearby toilet seat.

      • Re: Virus (Score:1, Funny)

        by Anonymous Coward

        So what you are saying is if I have sex with twins I am helping cure cancer? Awesome, Nobel prize here I come!

    • Ideally the virus is not sexually transmittable. They want to use viruses that do not have the ability to reproduce in a cell. They can only reproduce in the lab. So you manufacture the viruses in large quantities, remove T Cells, then infect the T Cells. The T cells have their DNA modified in a way to make them more likely to fight the cancer, but do NOT have their DNA modified to make more viruses - that code is not built into the viruses.

      Then you inject them back into the human, where the T Cells ma

      • by Anonymous Coward

        Nature will find a way.

        I have 4 shitty dinosaur movies that will back me up.

    • You're just using the virus as a big syringe, you don't make it such that it can replicate itself. The patient won't be able to transfer T-cells to anyone with a competent immune system. For those compromised enough to be colonized by foreign T-cells... they're at death's door anyhow.
    • Unless these are particularly rare cancers they are going to treat there should be enough data to establish a baseline for comparison.
      • by jedidiah ( 1196 )

        This tends to be the zone of rare cancers where drugs need the Orphan Drugs Act just to get into the approval pipeline. The non gene-splicing version of this is only used on patients that already have failed to respond to any other therapies.

  • This is incredible technology, The daisy chain gene drive though is about as hard core sci-fi as you can get. But genetics are essentially hyper complex software, and a lot can go wrong when jumping in on someone else's undocumented code.
  • and the main actor was a prince or something?

  • I have altered your DNA.

    Pray I do not alter it further.

  • how Deadpool came to be ?

  • The Nixon administration systematically-destroyed the commissioned-study by the Medical College of Virginia that showed Delta-9-THC killed many cancerous cell-types, both benign and malignant. From that study:

    Delta-9-THC, delta-8-THC, and cannabinol (CBN) all inhibited primary Lewis lung tumor growth, whereas cannabidiol (CBD) enhanced tumor growth. Oral administration of 25, 50, or 100 mg delta-9-THC/kg inhibited primary tumor growth 8, 72, and 75 percent respectively, when measured 12 days post tu

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