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Six-year-old cured of lymphoma leukemia.
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olePigeon
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Dec 11, 2012, 08:47 AM
 
I posted about the phase 2 clinical trial a few years ago where two of the four people were eventually cured of stage IV lymphatic leukemia, and the other two were in remission. All of whom were terminal. Because it was a phase 2 clinical trial, it was limited to a single dose treatment. Hopefully this new treatment will be approved soon, and the two other patients in remission can get a second dose and cure it completely. I've been following it pretty closely.

Phase 3 clinical trials had started, and one of its first recipients was a six-year-old girl named Emma. There have since been other patients treated with the same method, all of them now cancer free.

Basically, cancer's DNA is a mutation of your own DNA. On very, very rare occasions, a person's immune system may actually attack and destroy cancer cells. So the trick for 99.99% of the rest cancer cases is to somehow modified the T-cells that make up our immune system, and then have them attack the cancer directly. Here's the catch: how do you easily modify the T-cell so it knows to attack cancer?

As it turns out, we already know of a particular retrovirus that is notoriously good at manipulating T-cells, HIV. So researchers at the University of Pennsylvania modified a virus by changing its "payload" and disabling its ability to replicate itself. The new payload is the RNA extracted from the sequenced DNA of the cancer. After introducing the modified HIV into the blood of a host, it will chemically change the RNA into DNA, then modify the T-cells like it usually does. However, instead of replicating itself to the point of destroying the immune system, the infected T-cells now have a brand new set of instructions: destroy the cancer cells.

So now you have T-cells programmed to attack cancer! You administer those T-cells to the patient (almost like a flu shot), and now the body knows how to target and attack cancer cells.

This is an extremely laymen's description, but I hope you get the gist of it.

In all cases (the original four patients and now this little girl and the others), the treatment was risky because it causes a cytokinetic swarm. The immune system suddenly knows how to identify and attack cancer cells that have metastasized and spread throughout the entire body, so the immune system goes into overdrive as it starts attacking the cancer. This wreaks havoc on the body as it does everything it can to neutralize the threat. Basically, take the absolute worst cold/flu you've ever had in your life, then make 100 times worse. Yes, 100 times. I'm not exaggerating.

However, the risk is practically negligible considering the alternative is certain death. The treatment is 6 and 0, but I'd call those two in remission a tie. That's over six people in stage IV leukemia cured 100%, and two in remission. I call that awesome!

Incidentally, my mom just finished surgery on her other breast yesterday, she is now tumor free. It's promising to know that in the near future should she get cancer again, she may be able to just take a pill for it. Reminds me of Star Trek IV when McCoy gives that woman a pill and cures her diabetes.

"…I contend that we are both atheists. I just believe in one fewer god than
you do. When you understand why you dismiss all the other possible gods,
you will understand why I dismiss yours." - Stephen F. Roberts
     
The Final Dakar
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Dec 11, 2012, 08:48 AM
 
Shout out to CHOP
     
BLAZE_MkIV
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Dec 11, 2012, 09:56 AM
 
The part of this that is really brutal is that they don't need to inject you with a billions of T-cells. Just enough to jump start the recruitment of other T-cells.
     
Uncle Skeleton
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Dec 11, 2012, 10:16 AM
 
In case anyone was wondering what I was:
The new genes program the T-cells to attack B-cells, a normal part of the immune system that turn malignant in leukemia.
...
It is not clear whether a patient’s body needs the altered T-cells forever. The cells do have a drawback: they destroy healthy B-cells as well as cancerous ones, leaving patients vulnerable to certain types of infections, so Emma and the other patients need regular treatments with immune globulins to prevent illness.
     
Snow-i
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Dec 11, 2012, 12:39 PM
 
This is incredible.
     
P
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Dec 11, 2012, 12:56 PM
 
I read about this case elsewhere. Sounds a bit like a holy grail - if you can reprogram T-cells to attack whatever you like, that would be a cure for a lot of conditions.
The new Mac Pro has up to 30 MB of cache inside the processor itself. That's more than the HD in my first Mac. Somehow I'm still running out of space.
     
Uncle Skeleton
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Dec 11, 2012, 01:12 PM
 
Originally Posted by P View Post
I read about this case elsewhere. Sounds a bit like a holy grail - if you can reprogram T-cells to attack whatever you like, that would be a cure for a lot of conditions.
Well there are some serious conceptual limitations to the strategy. It targets a certain cell type, but it can't separate bad ones from good ones and just kills them all, so it has to be a cell type you can live without. It also creates an immune response (illness) proportional to the amount of target cells, so you have to be lucky enough to have a target cell type that's not very numerous.
     
Waragainstsleep
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Dec 11, 2012, 01:52 PM
 
On the plus side its cheaper than the bone marrow transplant which would otherwise be the last-gasp treatment.

There was another interesting one I heard about today too. They designed a tool which is essentially a needle which they can chill with liquid nitrogen. The poke it into breast tumours and freeze them repeatedly in they basically just get so badly damaged they die. A novel idea and a great alternative to surgery.
I have plenty of more important things to do, if only I could bring myself to do them....
     
BLAZE_MkIV
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Dec 11, 2012, 03:03 PM
 
They'll figure out how to get it to trigger based on some property of the cancerous versions of the cell. I bet that's what's next. This is just better than the alternative because the patients don't have time to wait for a more refined version.
     
andi*pandi
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Dec 11, 2012, 05:23 PM
 
Originally Posted by Waragainstsleep View Post
On the plus side its cheaper than the bone marrow transplant which would otherwise be the last-gasp treatment.
There was another interesting one I heard about today too. They designed a tool which is essentially a needle which they can chill with liquid nitrogen. The poke it into breast tumours and freeze them repeatedly in they basically just get so badly damaged they die. A novel idea and a great alternative to surgery.
Saw something similar being done to liver tumors, but not sure it has cleared FDA in the US yet. Needle has radiofrequency ablation (heat) to the tumor, but has cool liquid circulating so it doesn't burn healthy tissue.
     
Waragainstsleep
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Dec 12, 2012, 02:00 AM
 
Originally Posted by BLAZE_MkIV View Post
They'll figure out how to get it to trigger based on some property of the cancerous versions of the cell. I bet that's what's next. This is just better than the alternative because the patients don't have time to wait for a more refined version.
I think thats tricky because each cancer is different but I guess since the treatment is already individualised to each patient it wouldn't be a massive extra step as long as they can find a unique trait to target and an exclusive way to bind for it. Which sounds pretty time consuming now I spell it out. Still better than chemo which just nukes everything.
I have plenty of more important things to do, if only I could bring myself to do them....
     
P
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Dec 12, 2012, 03:02 AM
 
Yes, if you're making the T-cells custom anyway, you might as well make the custom to recognize cancer cells as well - assuming they can figure out how.

An idea: In women, each cell has one of the X chromosomes randomly deactivated (because without that, they'd produce twice as much of each protein on it as males do). In a cancer, all cells have the same X deactivated (because they're all just copies of the same single cell gone bad - in fact, this is one of the main evidences of that theory). If one could target the T-cells to only target the cells with the same active X as the cancer cells, the patient would have half the immune response and still have a good percentage of healthy cells around to tide her over.
The new Mac Pro has up to 30 MB of cache inside the processor itself. That's more than the HD in my first Mac. Somehow I'm still running out of space.
     
BLAZE_MkIV
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Dec 12, 2012, 05:59 AM
 
This would only work if the protein specific the to cancerous mutation was coded for on the X chromosome and is different on each chromosome. I don't think the odds of wither of those is good.
     
Uncle Skeleton
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Dec 12, 2012, 07:03 AM
 
Originally Posted by BLAZE_MkIV View Post
This would only work if the protein specific the to cancerous mutation was coded for on the X chromosome and is different on each chromosome. I don't think the odds of wither of those is good.
I think the idea is more like racial profiling: you won't get the right answer but you'll get closer than before.
     
P
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Dec 12, 2012, 11:22 AM
 
Originally Posted by BLAZE_MkIV View Post
This would only work if the protein specific the to cancerous mutation was coded for on the X chromosome and is different on each chromosome. I don't think the odds of wither of those is good.
No, it would work if there were a single surface protein on the relevant type of cell that is encoded on the X and different on the two copies. Does not have to be specific to the mutation - we're not trying to kill only cancer cells at this stage, we're trying to reduce the number of healthy cells we kill.
The new Mac Pro has up to 30 MB of cache inside the processor itself. That's more than the HD in my first Mac. Somehow I'm still running out of space.
     
   
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