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Biotech Science

Gene Therapy Corrects Hemophilia in Mice and Dogs 42

FiReaNGeL writes "Researchers successfully corrected hemophilia, the oldest known hereditary bleeding disorder, in dogs and mice. They introduced the gene coding for clotting factor VIII, which is defective in type A hemophilia, in the liver of newborn animals. All animals achieved expression of the clotting factor, averaging 139 and 115 percent of normal factor VIII activity and are stable after more than a year. The treatment targeted newborns exclusively for two reasons; their liver is still developing and their immune system is immature, facilitating the gene transfer. Researchers plan to perform tests in higher primates in the next few years. A summary of the research and the reference to the original paper are available."
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Gene Therapy Corrects Hemophilia in Mice and Dogs

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  • WHats sad is.... (Score:5, Insightful)

    by Creepy Crawler ( 680178 ) on Monday April 18, 2005 @08:06PM (#12276313)
    Most drug companies will petition AGAINST these types of treatments for the sake of "medical scrutiny not up to standard".

    Go take a look at [] and take a look how Type 1 IDDM can be cured by using YOUR OWN BODY PARTS.

    Now, why would drug companies NOT want to do away with insulin dependancies? Well, soo much for selling:

    Testing Kits
    Monitoring Equipment
    and much more.....

    Thats one hell of a profit margin that would be lost if even 1 person was cured of insulin dependancy. Now think of if all were...

    • I doubt CORPORATE AMERICA(tm) would directly oppose such a development, if only because the bad PR which would arise from said opposition. The continuation of scientific development is the lifeblood of the American Market; especially the upgrades required to stay competative.
      • by Creepy Crawler ( 680178 ) on Monday April 18, 2005 @08:24PM (#12276493)
        Then you fail to grasp corporate language.

        When a group of well-paid 'scientists' appears in front of a FDA panel for phase 3 testing saying:

        "We feel, after going through our and their data, that this proposed treatment has unforseen detrimental effects and must be delayed for further study"

        And of course, unlimited delays mean no treatment. And more profit for the insulin profit-derived makers.

  • Haemophilia (Score:3, Funny)

    by rookworm ( 822550 ) <> on Monday April 18, 2005 @08:10PM (#12276358)
    the oldest known hereditary bleeding disorder

    Aww nuts, I thought it was that other hereditary bleeding disorder.

  • by Xenkar ( 580240 ) on Monday April 18, 2005 @08:27PM (#12276519)
    I'll probably bleed to death before they release a real cure.

    BeneFix Recombinant Factor products cost about $1000 per 10CC, with a typical dose being 40CC. Typically a patient receives three treatments a day for two weeks after surgery or an accident. That's $168,000. There is just too much profit in these drugs for hemophilia to ever be cured.

    Of course, that cash amount doesn't look nice to potential employers. I don't participate in anything now since I can't afford this stuff.

    I've considered living out in the woods, but I'd probably injure myself and die a slow and horrible death.

    In conclusion, I'd like to thank Benefix for saving my life and curse them for making my ass unemployable with thier overpriced medicine.
    • I thought such treatments required huge quantities of donor blood, hence such a large price. Is this correct?
      • by Xenkar ( 580240 ) on Monday April 18, 2005 @09:05PM (#12276887)
        Actually the Benefix Recombinant Factor products are made from genetically altered hamsters. The process of harvesting the factor kills the rodents so my existence is the bane of any animal rights advocates.

        This also means that I won't get HIV or any other nasty blood disease. Most hemophiliacs don't feel safe using donor blood due to the HIV outbreaks of the Early 80's. About 44% of hemophiliacs at the time were infected.

        The blood supply is supposedly perfectly safe now, but I wouldn't touch that stuff even if it'd save my life.

        My uneasiness comes from the thought that someone could get infected with HIV and go donate blood before any detectable amount of HIV anti-bodies form in the blood. They actually test for the presence of the anti-bodies instead of the actual virus.
        • They actually test for the presence of the anti-bodies instead of the actual virus.

          That's because the antibody test is simpler, quicker and cheaper and usually very accurate. Not saying it's Right, just an observation.
        • A cousin of mine who was a hemophiliac contracted HIV from donor blood. They discovered he had it after tests run by the company making the treatments. They had discovered a bad batch had been released for use. He was around 18-20 at the time and lived to about 25-26 before his immune system succumbed to the HIV. That was about 15 years ago.
    • Just out of curiosity, do you know what it costs the manufacturer to actually produce this stuff? I am imagining maybe $100 per cc at most. As you say, the pricing is obscene. The manufacturer probably would cover their research costs after 5 years at the most.
    • I lost a very wonderful friend, (that had hemophilia) due to the AIDS complications from infected clotting factor. He had to introveniously inject these clotting factor all of his life. He was living in America and was transfered to Scotland on a job assignment. It turns out his brother had hemophilia also and had been diagnosed as having an unusual brain tumor that was linked to AIDS. His brother was remembered to have told the doctor when he was told of having AIDS that he had never had intercourse with
  • One problem... (Score:1, Insightful)

    by Game Genie ( 656324 )
    Where did they find enough hemophilic mice and dogs? I wouldn't think there is much screening for such genetic disorders in animals. Perhaps dog breeders might do this, but mice? Or do they just breed them on purpose from hemophiliac parents?
    • Re:One problem... (Score:1, Informative)

      by Anonymous Coward
      Perhaps dog breeders might do this, but mice?

      Yes, mice. If you ever want to retire early and comfortable, breed a stable, genetically-deficient blood-line of mice. You'll make a killing.
    • Re:One problem... (Score:4, Informative)

      by valkoinen ( 81260 ) on Tuesday April 19, 2005 @05:34AM (#12279636)
      Getting mice with any kind of genetic disorder would not be too difficult, since mice are grown for laboratory purposes all over and if a suitable bacch is not found then the scientists could relatively easily introduce the genetic defect to the mice. It's been done before.

      Dogs are a bit trickier, but as lab animals they are probably not that different from the mice.

      Scientists don't just go to pet shops to buy the lab animals. They are usually bred especially for the purpose of lab experiments.
  • I'm not usually an optimist, but how can you just throw away the hope of a real cure because of lost profits? If this were true, we'd still be using leech courses. Think of how much money those leech farmers lost when penicillin was discovered! This condition has been greatly researched and now we're seeing results. It was even discussed in a biochemistry 100 level class here at my university. My first response to this article was to be thankful.

    As for the drug companies, there will always be anothe

    • We still ARE using leeches in medical practice. 4.html/ []
    • I think you are being a bit naive.

      Food supplements are making inroads into various health issues. However, the drug industry is fighting them tooth and nail with the help of the FDA -- because they hurt profits.

      I'm not a conspiracy theorist wacko, just look at the hatchet job they attempted on the Life Extension Foundation []. I don't have the exact link, you'll have to do some digging to find it.

      • by Anonymous Coward on Tuesday April 19, 2005 @01:23PM (#12283428)
        (Posting AC 'cause I don't want to cancel my good mods above...)

        The drug industry is fighting food "supplements" because drugs are required to prove that they are safe and effective before they can be sold, and food supplements are not. You may be able to get actual help from a food supplement, or you might not. Unlike an FDA-approved drug, neither the supplement nor its mode of administration have been tested for safety and efficacy in double-blind protocols. People may make claims for the wonderful effects of different supplements, but there is literally nothing to distinguish their experiences from the body fixing itself or the placebo effect.

        There have been examples of calcium supplements which contained lead. There are all kinds of supplements which contain widely varying amounts of the supposed active ingredients, including none at all. Quality control is very easy for supplement makers. If you have no standards for your product, everything you produce will meet them. The buyer may be getting nothing, or may be taking an overdose - unless the actual stuff they're taking is assayed, nobody is going to know.

        I personally am taking vitamin and mineral supplements, but they have stated (and presumably assayed) amounts of the various substances therein. I can total the amounts I'm taking and verify that I'm neither deficient nor in the overdose range (both of which are bad). I started taking vitamin D after I found a couple articles in Science News pointing to research showing that many people are D-deficient and that this causes loss of muscle strength as well as bone density - and found that my back pain cured itself after about a week of taking 600 IU per day (which was a totally unexpected result but which made me very happy nevertheless). I'm currently up to 800 IU just to make sure; this is still well under the toxic dose. I don't get a heck of a lot of sun for most of the year and I can't drink milk, so I don't get much otherwise.

        If we wanted to make sure that supplements were actually doing anything (let alone what they are claimed to do), we would require:
        1. Supplements of known and consistent composition
        2. Used in double-blind trials

        Without spending the money ($millions) to do this for every supplement on the market, there is absolutely no proof that they do anything .

        Should the government be funding such trials? HELL, YES! And we should be using the best of modern gene science to find out why everything works, so we can determine who needs what interventions. If you think about all the money that could be saved if we could hold off or prevent problems using vitamins and herbs instead of treating them with drugs, the nation would be far ahead. But without those trials to give us real reliable knowledge, everything might as well be snake-oil sold by hucksters.
        • You are partially correct. However, it is a real stretch to say their is no proof or that there is no research.

          Have you ever done a search on PubMed? There are all kinds of studies being done -- random double blind and so on with strong statistical results to draw from in many cases.

          I will agree that people often fall prey to hype, but is it really the job of the government to act as our nanny? What if you are sick and traditional medicine does not have much to offer? Should the government deny you

    • I'm not usually an optimist, but how can you just throw away the hope of a real cure because of lost profits?

      Ah, but that's exactly why one of the drugs we use to treat Leishmania is running out - they stopped production due to profits.

      When a disease tends to impact poor third-world inhabitants, never underestimate the negative impact on drug treatment production.

  • by khanyisa ( 595216 ) on Tuesday April 19, 2005 @06:27AM (#12279778)
    Spelling it nicely in British English :-) Not all Haemophilia is as severe as mentioned in the article, I have 14% of the normal clotting factor and haven't had a problem for years... of course a head injury etc is still a major risk. But this is really nice research to see

"Well, it don't make the sun shine, but at least it don't deepen the shit." -- Straiter Empy, in _Riddley_Walker_ by Russell Hoban