seekingalpha.com: Genzyme and Isis Announce FDA Approval of KYNAMRO™ (mipomersen sodium) Injection for the Treatment of Homozygous Familial Hypercholesterolemia

[twa]

I have no idea what any of this means.

me either,and my wife has some genetic cholesterol condition

good news for somebody

[Warpath11]

Ok, I semi understand that what they are doing is introducing a molecule (into what?) which then triggers a specific response from the body's (immune system??) in order to defend against disease.
How is this different than a vaccine? I know that vaccines use a germ (or replica) that tricks the body into producing defenses against the live/real version so when the live/real version is encountered the body already has a defense ready for it.

In a nutshell:

Typically a vaccine is an inactive form of the virus that still triggers an immune response to the virus (thus creating long-term immunity to that virus when it is encountred in the real world). In this case a small interfering RNA designed to bind native ApoB-100 is injected, the RNAi gets into the liver from circulation, triggers a set of responses from the cells in the liver that cause, in this case the native ApoB-100 protein, to be degraded thus the liver can not package LDL particles since ApoB is required for the production and secretion of LDL from the liver.

[AsburySkinsFan]

In a nutshell:

Typically a vaccine is an inactive form of the virus that still triggers an immune response to the virus (thus creating long-term immunity to that virus when it is encountred in the real world). In this case a small interfering RNA designed to bind native ApoB-100 is injected, the RNAi gets into the liver from circulation, triggers a set of responses from the cells in the liver that cause, in this case the native ApoB-100 protein, to be degraded thus the liver can not package LDL particles since ApoB is required for the production and secretion of LDL from the liver.

Ok.....seriously...that didn't help.
I think I'll just accept this all as a good thing and be glad that there are people who do this type of thing and understand it much better than my feeble mind can.

[PeterMP]

Ok, I semi understand that what they are doing is introducing a molecule (into what?) which then triggers a specific response from the body's (immune system??) in order to defend against disease.
How is this different than a vaccine? I know that vaccines use a germ (or replica) that tricks the body into producing defenses against the live/real version so when the live/real version is encountered the body already has a defense ready for it.

You have to get the molecule into the cells that you want to affect.

Not what we traditionally think of the immune system, but a part of the bodies defense against viruses.

There are two differences:

1. The resistance to viruses from vaccines depends on your body making different antibodies. The antibodies for the flu virus are different than the antibodies for the small pox virus. You are inducing the production of things that have little functional over lap.

2. The vaccine for the flu is very different than the vaccine for small pox. The process by which you generate flu vaccine are not the same as the processes by which you generate small pox vaccine.

Here we're talking about using a general process that your body is already carrying out and inducing a response based on molecules that are made by the samethings to treat different things.

This has important results that can be seen based on information in the OP. This is being used to treat a non-viral infection. We can induce the RNAi system to change human cells. However, it also shows great promise (and has been used in non-human models) to treat viral infections. We can use the RNAi system to get human cells to kill viruses.

The key is very related molecules can be given to the cells to cause a response that is very different (i.e. affect different things) and specific.

[Warpath11]

Ok.....seriously...that didn't help.

LOL I guess I should stick to not teaching...

[AsburySkinsFan]

Here we're talking about using a general process that your body is already carrying out and inducing a response based on molecules that are made by the samethings to treat different things.

This has important results that can be seen based on information in the OP. This is being used to treat a non-viral infection. We can induce the RNAi system to change human cells. However, it also shows great promise (and has been used in non-human models) to treat viral infections. We can use the RNAi system to get human cells to kill viruses.

The key is very related molecules can be given to the cells to cause a response that is very different (i.e. affect different things) and specific.

Ok, I think I get this now, it is not about engaging the immune system, but instead changing the very construction of the cells themselves.
Is anyone wondering about the law of unintended consequences?

[Warpath11]

Ok, I think I get this now, it is not about engaging the immune system, but instead changing the very construction of the cells themselves.
Is anyone wondering about the law of unintended consequences?

Not about changing the physical construction of the cell per se, rather you are changing one protein out of many a cell can make. The cell is still fully functional it is just now not secreting the protein that was knocked down by the RNAi.

[PeterMP]

Ok, I think I get this now, it is not about engaging the immune system, but instead changing the very construction of the cells themselves.
Is anyone wondering about the law of unintended consequences?

That's always an issue with medicines and one reason why I said in my previous post I'd be a little concerned about adding it to a cell permanently like WP was in one of his other messages (i.e. for now at least I'd want a mechanism to turn it off or take it out).

In this case, the change in the cell is due to the injections. If you stop the injections, the cell should go back to normal (should based on what we know).

Also, in this initial case, we're talking about a small subset of people that don't have a lot of other good options (so that it makes it more okay to use them as guinea pigs (hopefully)).

We've also been doing this for a long time in other animals to study specific things so it isn't that likely that we're missing something REALLY MAJOR (though the devil is in the details).

But you are right, you are altering the cells and not causing a change in the immune response. This is why this is a big deal, You could change the cells so that deal with infectious agents more effeciently, but you can also change the cells so that deal with internal genetic issues that you can't with vaccines (at least not very well).

The biggest thing though is the commonality. Every drug currently essentially has its own route to make it. Treating very different illnesses/diseases will be possible with molecules that are very similar and therefore synthesized very similarly. No more patents for how to make the molecules for every disease (once its clear how to make the molecule to get to a specific cell type (e.g. the liver)).

Making these molecules is chemically simplistic. It is possible to imagine that some day you could to a machine and blow your nose into a machine, and they'd analzye your snot an deliver a molecule effective to the particular virus affecting you.

All of the molecules to affect a large number of viruses at some level would be similar and their synthesis all closely related.

The differences in the way vaccines have to be made would not seem to make that possible.

[AsburySkinsFan]

Yeah, I'm gonna go with...this is really good....but there is still the risk of us turning an entire segment of our population.

Jokes aside, what about the risk of the immune system seeing these new cells as threats, like what happens in organ transplant rejection?

[PeterMP]

Yeah, I'm gonna go with...this is really good....but there is still the risk of us turning an entire segment of our population.

Jokes aside, what about the risk of the immune system seeing these new cells as threats, like what happens in organ transplant rejection?

Not much in most cases.

When we give medicines, we're already changing cells. This is just changing through a different process.

Your targetting specific proteins. Not altering the cell in a massive manner. If you went after the wrong protein, I guess it is possible, but the majority of the proteins in your cells the immune system doesn't see (only proteins on the surface of the cell can trigger an immune response).