What: Glybera is the brand name (like Tylenol) for alipogene tiparvovec (like acetaminophen), and it is a therapy with the goal of replacing the lipoprotein lipase enzyme in large muscles of the body in patients with LPLD, to make the symptoms and the disease go away. In order to make the body make LPLD, the drug has to get a gene sequence for the LPL gene into the DNA of the patient. Gene therapy! It is undergoing testing in Europe. From what I've heard, it was denied the chance to do testing in human subjects in the US at this point.
Who: Glybera is manufactured by the company uniQure. I am always wary of pharmaceutical companies because they stand to make a lot of money off of patients.
How: After receiving an epidural-type numbing medication (or being sedated), patients receive up to 60 injections into the large muscles of their legs to introduce the DNA that will join up with their own DNA and start to manufacture the lipoprotein lipase enzyme.
Where: Lipoprotein lipase is naturally located in just about every tissue of your body. The injections are only into a big muscle because the muscle is easy to get to, and your legs use a lot of energy, so they could definitely benefit from the extra energy contained in those pesky triglycerides, taking them out of the blood!
When: All the individuals involved in the studies have been adults. No one is even thinking of using this in kids anytime soon.
|My LPLD girls!|
Pros of Glybera:
- First gene therapy to be approved (in Europe), ever! How cool is that! We are entering the age of gene therapy, a topic of sci fi novels for ages!
- Well designed testing and treatment - I have a little bit of a background in research and with the adeno-associated virus that they use to 'infect' the patient's cells with the new DNA, and I find it all very clever and exciting!
- Rare diseases like LPLD hardly get any attention from drug companies, it's wonderful that someone is looking into a cure!
- Price tag of $1 million. Yikes! Admittedly, it might be a fair price given all the research that has gone into it, and the small percentage of the population that actually has LPLD and therefore might benefit from the treatment, but for a disease that can be largely controlled by diet, that's a tough call to make. For my family, without any episodes of pancreatitis, it's not worth it at this time.
- First gene therapy to be approved (in Europe), ever! How scary is that! We are entering the age of gene therapy, a topical of sci fi novels for ages because there is SO MUCH that we can imagine going wrong when you start to mess with DNA.
- Effectiveness - from what I've seen, this treatment will not let an individual with LPLD eat like someone without LPLD. At best, an individual experiences fewer episodes of pancreatitis, while maintaining a very low fat diet, than they did before. But maybe that's because the only patients trying this out are ones who have many episodes of pancreatitis in their history. Maybe someone with LPLD who has never had pancreatitis would get to eat macaroni and cheese every day, and still not get pancreatitis. An interesting thought... but it's a lot of money and pain to go through just for mac and cheese!
- Longevity - from what I've seen, the effects only last maybe 10 years. Maybe less. After that, maybe you spend another $1 million to get retreated? Or you go back to eating the very low fat diet you were on before? Hmm.
- Immune suppression - patients with LPLD may have to take additional medications so that their immune system doesn't fight off the DNA that Glybera is introducing. This means the individuals may be more susceptible to other infections, since their immune system is kept from working as well as usual.
- Not an option until my girls are 18, at least, anyway!
Interested in a more detailed description of the trials Glybera has already gone through? Try here