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Gene Therapy Corrects Hemophilia in Mice and Dogs
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."
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 Joinleenow.com 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
Needles
Insulin
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...
Aww nuts, I thought it was that other hereditary bleeding disorder.
The toad can't burp - and for some reason can't fart either, so it swells up and eventually explodes. --Anonymous Coward
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.
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?
Why is it called menstruation if only women get it?
Those who sacrifice security to condemn liberty deserve to repeat history or something. - Benjamin Santayana
As for the drug companies, there will always be another disease/condition to develop drugs for and they could invest in gene-therapy developers.
The imminent collapse of space and time is just the Universe's way of hugging you.
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
I think you should ph33r my mad ISO 8859-1 skillz. ;-)
(Hint: the character is æ)
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:
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.
One reason that supplement-makers don't perform such tests is that one cannot patent herbs and the like, and without a way to get a commensurate revenue stream from the product there is no money to perform the kind of double-blind testing that is done with drugs. (If anyone can sell e.g. echinacea, you can't raise your price to recover the cost of testing from your sales.)
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 the opportunity to try non-traditional means?
Health is just an area that people treat as a sacred cow. It is not right that in all other areas we are allowed to make our own decisions, supposedly, but that in health we are forced to let others decide what is better for us.
I would propose we disallow baseless claims and hype, which is really just truth in advertising, instead of disallowing the sale of products to those that want them.