Glybera and the massive over-reaction it’s inspiring!

This week saw the approval of a new drug called Glybera. This is the first in a long awaited class of gene therapy compounds. Officially classed as an adeno-associated viral vector expressing lipoprotein lipase, a non-biologist like me can only assume that the virus will be responsible for locating the appropriate site within the body and then “inserting” the DNA into the appropriate cell’s DNA. Then when the newly adjusted DNA comes to be transcribed the appropriate enzyme will be the result (in this case lipoprotein lipase). Now I’m not going to get into the specifics of this…well, because I’m not an expert and frankly I have a lot of reading to do on this before I’d feel comfortable enough to really go at it. But I will say that in my opinion this has been a long time coming! I thought that with the advent of the human genome project, these kinds of drugs would be all over the place! Sadly this appears not to be the case.

an analogous enzyme

There isn’t a crystal structure for LPL but this is a phospholipase and I think it’s quite pretty. They’re from the same gene family (lipases) so It’s like putting in a picture of a good looking cousin and claiming it’s you. It’ll do for now but it’s not optimal!

The one big thing I will talk about though concerns the recent interaction I’ve had with other bloggers on here. Since deciding to make more of an effort with my blog, I decided to have a look at other writers out there in the big bad blogosphere. The first, second and third pieces that I came across all appeared to be engaging in some serious scare-mongering (Although curiously enough each one said the same thing, almost verbatim!.. that in itself is something I’m sure I could talk about). Does the community at large (and I ask this especially of non-scientists) think that we’re all going to turn into genetically modified super-humans? Or, more to the point, mutants? Do people really think that when a drug hits the market that scientists (and their marketers alike) have some magical power to make them take this drug? What is there to be afraid of? If you don’t trust something, don’t take it!

Let’s remember that, this drug has just been approved and if anything untoward does happen, you can bet your ass that it’ll be whipped off the market at break-neck speed. This leads me to my final point. This drug has only been approved for people whose genes don’t produce lipoprotein lipase naturally. So what this drug is doing is allowing their body to produce a naturally occurring enzyme that will prevent them from developing a potentially fatal disease. This is not the beginning of the real X-men!

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  1. Great to discover your site Craig. I like your writing style too. Tweeted your post

    • Genuinely glad you enjoyed it. Stay tuned and feel free to continue commenting! I’m always open to suggestions on how to improve as a science communicator. Thanks for the tweet.



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