New breakthrough claimed to stop MS completely in mice and possibly other autoimmune diseases.
November 19, 2012 1:14 PM   Subscribe

Nanoparticles covered in proteins trick immune system. A breakthrough new experimental treatment that uses nanoparticles covered with proteins to trick the immune system, managed to stop it attacking myelin and halt disease progression in mice with relapsing remitting multiple sclerosis (MS). The researchers say the approach may also be applicable to other auto-immune diseases such as asthma and type 1 diabetes.
posted by aleph (23 comments total) 23 users marked this as a favorite
Oh please oh please oh please
posted by Tomorrowful at 1:20 PM on November 19, 2012 [18 favorites]

(I mean, yes, I know about all the ten thousand caveats and it's just mice and there's a long path from "Something might be useful" To "here is a drug." It's just, well. You know.)
posted by Tomorrowful at 1:21 PM on November 19, 2012

I wish we lived in the future. My sister-in-law has ALS. If this were applicable -- if is saves other future sister-in-laws -- that would be a very great thing indeed.
posted by offalark at 1:22 PM on November 19, 2012 [2 favorites]

This sounds like magic. Is this something that could be used to treat allergies?
posted by wemayfreeze at 1:25 PM on November 19, 2012

It's early days yet but the technique seems to be a general way to trick the immune system into accepting stuff that it's objecting to.
posted by aleph at 1:29 PM on November 19, 2012

So just to be clear, this is not a cure for MS but a treatment of its worst symptoms? Thanks tiny particles!
posted by Potomac Avenue at 1:34 PM on November 19, 2012

I haven't read the link yet. I want to hold on to what those few sentences might mean to me.

I'm in my early twenties and a more comfortable life would be hard to imagine. I've been lucky, blessed but I popped into this world 3 months early and I've had a little metal cylinder, an inhaler, in my pocket just about every day since.

I never, as my family might tel you, hated sports, I just knew they were nothing I would excel at. They require a more ideal management of oxygen. I love cats. I love dogs. My lungs tend tighten fairly drastically the longer I am in their presence. Sometimes, it's just the end of the day and those two bags in my chest are just tighter and....less than they were at the beginning.

There are folks with worse asthma than me, certainly. There are far worse conditions out there that, if it were a universe that forced us to make such choices, I would wish cured before asthma.

But, like I said, I'm only in my early twenties. If this research means the possibility, decades and more studies later, that I might run a down a wooded path, breathing cold night air, with a dog of my own by my side and to take that for some kind of granted..... I'm happy to just be able to cross my fingers at these kinds of things.
posted by sendai sleep master at 1:41 PM on November 19, 2012 [9 favorites]

Quick note on the diabetes angle: Don't hold your breath for a cure. There are a number of techniques being looked at to prevent type 1 diabetes. The issue is that once the immune system eliminates β cells, they don't grow back out of nowhere, so you have to get there before this happens. They would have to use nanoparticles to protect these cells in the early developmental stages of diabetes. See Takiishi et al. for more T1D prevention.
posted by battlebison at 1:45 PM on November 19, 2012

Oh Christ--let this be the exception to the "if they use the word 'breakthrough' in the headline or first sentence it is bullshit" rule. Getting a handle on autoimmune responses is the key to such an enormous load of human misery.
posted by yoink at 1:49 PM on November 19, 2012 [2 favorites]

The actual article is here. Nature seems to be having some trouble right now though, so I can't actually read it.

Bear in mind though, that animal models =/= humans, and there are many cases where what works in one system has no effect in the other.

And, most science reporting is bunk.
posted by Orange Pamplemousse at 1:52 PM on November 19, 2012 [5 favorites]

Here is a link to the actual fucking paper seems to be down for the moment though.
posted by Blasdelb at 1:54 PM on November 19, 2012 [1 favorite]

"So just to be clear, this is not a cure for MS but a treatment of its worst symptoms?"

Well in mice anyway. We can't seem to access the actual paper to see the details of the actual study, but I'd have really fucking serious safety concerns not ameliorated by the fact that the substance they're building these microparticles on is indeed pretty safe. Presenting things to the immune system is a really tricky thing to do, and I'd want to see this tried in a pretty significant number of mice for whom none of them had their immune systems suddenly go batshit on myelin after the treatment before anything like this was even tried in Phase 1 safety trials in humans.
posted by Blasdelb at 2:08 PM on November 19, 2012 [2 favorites]

I've long felt that nanoparticles were going to be the key to solving a lot of diseases, including cancer. That is, if they aren't used for advertisements instead.
posted by mcstayinskool at 2:13 PM on November 19, 2012

The paper's up. On a quick look through the results are nice, but not earth shattering. Mice whose treatment starts before or after triggering of EAE have few to no relapses, which is what you want.

This has been done previously with antigen-coupled spleen white blood cells (cite, free), which are incredibly costly to produce (and still went on to human testing). The current paper describes nanoparticles which have the same effect, and are obviously a lot easier to make.

Basically, we now have a better understanding on what is required to stamp down the autoiummune response, and we have a better lead candidate for making a drug that does so therapeutically. What we don't have is a magic pill that will cure all your ills.
posted by Orange Pamplemousse at 2:23 PM on November 19, 2012

Three things:
  • Most globular proteins are technically biodegradable nano-particles.
  • We actually have a special word, antagonist, to describe moleculs you put into a patient to drive the kinetics away from the reaction causing a deleterious effect (see also allosteric regulation).
  • I worked on an agonist that received FDA approval 9 years ago so, as a concept, this is not shiny and new.
Having a core particle that already has FDA approval will help genericize things from a discovery standpoint, but picking your coat proteins, developing and scaling your purification scheme and doing clinical testing (the expensive part where drug candidates die) isn't going to get much shorter and the odds of clinical success base on a rodent model, while better than the lottery, aren't exactly a sure thing.
posted by Kid Charlemagne at 2:26 PM on November 19, 2012 [4 favorites]

Here's an NBC article I found easier to follow. The core of the technique seems to be this:
When normal cells die naturally through a self-destruction process called apoptosis, immune cells called macrophages come in and eat up the mess.

The macrophages are carried to the spleen where they show these ground-up bits of cells to other immune cells called T-cells. It’s a kind of introduction that familiarizes the T-cells with the body’s normal cells. Then T-cells know not to attack healthy cells.

Miller’s team had been trying to find ways to use this process to re-educate the T-cells. They have been attaching bits of the myelin that T-cells mistakenly attack to healthy cells from MS patients that were self-destructing, then infusing the concoction back into MS patients.
So the goal is to take whatever the patient's immune system is improperly attacking, put it in the spleen, and teach T cells that it's harmless. The process of attaching myelin to healthy self-destructing cells from the patient has shown some early promise in human trials, but is really expensive. So now they've shown that (at least in mice) it seems to work if you attach the stuff to a cheap nanomaterial that's already used in sutures instead.

In terms of cure vs. treatment of symptoms -- if it works, this will prevent ongoing harm being done by the immune system, but won't do anything about existing damage. It might also have to be repeated occasionally. So a cure, but not a complete cure.
posted by jhc at 2:28 PM on November 19, 2012 [2 favorites]

Here's hoping, but personally I find that my already-low confidence in science reporting takes at least a 70% hit when a word like "tricks" is used.
posted by phearlez at 2:45 PM on November 19, 2012

It's an encouraging paper, but as pointed out in the case of RA there is still a lack of knowledge of the underlying mechanism leaving the technique useless as they don't know which proteins to target.

Another point is the "hideously expensive" quote in the NBC article. One million dollars per 10 patients, or one hundred grand each, really isn't that much if it's close to a cure or only needs to be repeated every three or four years. Biologics such as the ones I'm on currently cost between $20,000 to $50,000 annually not counting all the adjunctive meds I need to take as well.
posted by michswiss at 2:47 PM on November 19, 2012

The "hideously expensive" technique was the old technique using live cells. The new technique using nanoparticles was created in part to address that.
posted by Mitrovarr at 3:15 PM on November 19, 2012

Has anyone else noticed what seems to be a high preponderance of autoimmune disease in general, and RA in specific, among active MeFites?

My monthly RA infusion is $5,000, plus the costs for the other 14 drugs I take. So $100K seems like a bargain.
posted by SweetTeaAndABiscuit at 3:47 PM on November 19, 2012

I'm an AnkSpond person and on six-weekly infusions at around the same cost. Only on 10 other meds though, so you have me beat there. This is only keeping me mobile and somewhat functional, but I'm still unable to work and on private disability. So the actual socioeconomic cost of my condition and treatment is certainly much, much higher than 100k a year.
posted by michswiss at 5:12 PM on November 19, 2012

I've had a little metal cylinder, an inhaler, in my pocket just about every day since.

*holds mine up, waves it around like a lighter at a Skynyrd concert*

I literally cannot leave the house without mine. I also keep a spare in my EDC bag, two in my bug-out bag, one in the glove compartment of the car, one in the bag of EMT first aid supplies in the trunk, at least one in my office at work... I can't ever get caught without one if I need it, as my asthma combined with scar tissue in my trachea could kill me pretty easy.

I'm thankful for overseas pharmacies that sell Albuterol inhalers for $3-something each (the "full sized" ones with blue bodies, not the little red CFC-safe crappy ones that everyone hates), versus $15 *with* insurance copay from Walgreens/CVS/etc.

Having multiple spares came in handy back in July when I was camping with a group of friends. Someone walked around asking for someone else who they knew had an inhaler; from inside my tent I said "Albuterol? For asthma?" "Yes.." I rolled over, grabbed my car keys, unlocked it with the remote and said "Little white and blue box with a red stripe, glove box, passenger side dash; tell whoever needs it to keep it as I have plenty."

However, if there's something I could do/take that would make my asthma even half as bad as it is now that I've lived with for almost forty years, I would tell you to name your price.
posted by mrbill at 5:21 PM on November 19, 2012 [3 favorites]

So just to be clear, this is not a cure for MS but a treatment of its worst symptoms?

It's actually not really either cure nor symptom treatment. It sounds like it can prevent further myelin damage, so in the sense that the MS disease process would not keep happening, it's sort of like a cure. But areas of myelin that are already damaged will not be fixed by this approach, so any existing deficits (numbness, weakness, balance problems, vision problems, etc.) will not go away if a person has had active MS damaging their myelin for years prior to getting this treatment. Recognizing MS early and being able to treat it with this drug (assuming it ends up working in humans) would prevent a lot of disability, but it won't fix the damage done in someone who's had many relapses over the years.

So, not a cure. But also not just a symptomatic treatment, either: current symptom management drugs for MS are things like Provigil, that helps people stay awake through their extreme fatigue, or Baclofen, which can help alleviate painful muscle spasms. None of these fix the myelin or stop its destruction, they just try to counteract the negative symptoms you get when your nerves don't work right. None of them completely alleviate the symptoms they're intended to treat, and they all have side effects. This new therapy doesn't just try to mask the signs of damaged myelin, it actually prevents further damage. So it's way cooler than plain old symptom management.

OK, a metaphor. Nerves are like electrical wiring. Myelin is like insulation on the wires. Mice are like the immune system cells. If mice come through and chew on the insulation, the wires are going to start shorting out. That's MS. This potential treatment teaches the mice to stop chewing the insulation. There will still be shorted-out wires in the walls, if the mice got to them already, but hopefully you won't get any more damage.
posted by vytae at 7:02 PM on November 19, 2012 [1 favorite]

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