Choking a Tumor MRI scans show that blood flow [red] decreases in liver tumors after ALN-VSP therapy, which stops cancer cells from making proteins that form blood vessels. Courtesy ALNYLAM

Since last April, 19 cancer patients whose liver tumors hadn’t responded to chemotherapy have taken an experimental drug. Within weeks of the first dose, it appeared to work, by preventing tumors from making proteins they need to survive. The results are preliminary yet encouraging. With a slight redesign, the drug might work for hundreds of diseases, fulfilling the promise that wonder cures like stem cells and gene therapy have failed to deliver.

The biotech company Alnylam announced in June that its drug ALN-VSP cut off blood flow to 62 percent of liver-cancer tumors in those 19 patients, by triggering a rarely used defense mechanism in the body to silence cancerous genes. Whereas conventional drugs stop disease-causing proteins, ALN-VSP uses RNA interference (RNAi) therapy to stop cells from making proteins in the first place, a tactic that could work for just about any disease. “Imagine that your kitchen floods,” says biochemist and Alnylam CEO John Maraganore. “Today’s medicines mop it up. RNAi technology turns off the faucet.”

Here’s another analogy: If DNA is the blueprint for proteins, RNA is the contractor. It makes single-stranded copies of DNA’s genes, called mRNA, which tell the cell to produce proteins. In 1998, scientists identified RNAi, a mechanism that primitive organisms use to detect and destroy virus’s double-stranded RNA and any viral mRNA. Mammals’ immune systems made RNAi’s antiviral function irrelevant (although all vertebrates, including humans, still use RNAi to regulate mRNA activity), but researchers found that introducing small segments of double-stranded RNA to cells could trigger the ancient mechanism and selectively halt the production of specific proteins.

That ability makes RNAi a potential fix for many diseases, including cancer, that arise when abnormal cells produce excessive amounts of everyday proteins. In theory, manipulating RNAi to kill proteins is simple. ALN-VSP, for example, consists of synthetic double-stranded RNA designed to match tumor mRNA that codes for two proteins: VEGF, which cancers overproduce to help grow new blood vessels, and KSP, which sets off rapid cell division. The researchers send the synthetic RNA into liver cells, and the body’s RNAi system kills both the synthetic RNA and any matching tumor-grown mRNA. Knock out the mRNAs coding for those proteins—which in the liver are produced only by cancer cells—and the tumor stops growing.

“We can turn off any one of 20,000 genes with RNAi,” says Bruce Sullenger, a molecular biologist researching RNAi at Duke University. “The challenge has been to get a drug into only the desired cells and not harm others.” Researchers have worried that a drug might disrupt normal protein production in a healthy cell, or that the immune system will destroy the drug before it reaches its target.

Alnylam overcame both concerns by packaging the drug in a fatty envelope that is absorbed primarily by the liver. This allowed doctors to administer the drug through the blood, rather than by an injection to one spot, which improves results by ensuring that the entire liver receives an even dose.

The technique’s ability to attack single genes could lead to drugs for the 75 percent of cancer genes that lack any specific treatment, as well as for other illnesses. Alnylam is already testing RNAi therapy for Huntington’s disease and high cholesterol in cell cultures; other researchers are tackling macular degeneration, muscular dystrophy and HIV. The potential has driven nearly every major pharmaceutical company to start an RNAi program.

Because the approach is fundamentally simple, RNAi therapy could be ready within two years, say experts including John Rossi, a molecular geneticist at City of Hope National Medical Center in California. Alnylam plans to enroll an additional 36 patients in the ALN-VSP trial and increase the dosage, but the early results are good enough to suggest that it could be among the first RNAi therapies to hit the market. “I think RNAi could work for anything,” Rossi says. “But even if it only works for liver cancer, it would be pretty good.” For liver-cancer patients who have been failed by chemotherapy and radiation and felt their harsh side effects, that would be wonder drug enough.

35 Comments

I for one will be watching the development of this treatment closely. We are moving in the right direction on eradicating cancer.

Simple, yet brilliant. Most research into cancer has been based on attempts to kill it. But why attack it when you can starve it.

too bad we probably won't be hearing anything from them anytime in the future again... :(
Most stories like this on this site and similar sites go that way it seems :(.

1. Holy crap this is awesome news!
2. "fulfilling the promise that wonder cures like stem cells and gene therapy have failed to deliver."

This really shouldnt be the case. We should use theses as well as the new RNAi treatments.

3. “The challenge has been to get a drug into only the desired cells and not harm others.” Researchers have worried that a drug might disrupt normal protein production in a healthy cell"

This part could turn out badly if we dont do it right. Instead of our cells eating themselves they just starve. Gots to be careful.

I do agree with lloWoll. I started coming on this site maybe 5 months ago, and already this is probably the third or fourth article relating to a cancer cure. However, I have never heard of it outside of Popsci.
These articles are particularly interesting to me too, as my mother was diagnosed with melanoma last month.

@IIoWoII and @TheFourth, Popsci does tend to catch these stories before they get through clinical (or even human) trials. So the fact that they disappear is not surpriseing. On the other hand, the trials do take a long time...

I like the final comments in the article though. It's fun to daydream about miracle cures for all diseases, but if this does make it through trials and is only a successful treatment for liver cancer, it's still an amazing medical advancement.

I think this one will work, just cause its easy to understand and it sounds safe.

I think the only thing that could kill it right now is that pharmaco we lose profit on it and decide to destroy it in my opinion.

This is a very simple science, ever get that why didn't they/I think of that sooner feeling, gee wiz it was there all along nobody saw it in the "tangle of the wire's of life", I'll keep it simple KIS ever splice/match 200 or more color coded cables find the 2 in 4 hundred first then it gets a little easier as you go, this is just a small very small example of looking for that magic potent/potion needle in a hay pile big hay pile at that, then you have to test it for safety every which way then some then more, the trick of RNAi is find a protein remove it and you can stop the formation of a particular gene which is needed to make a cancer cell, cholesterol, tumors, fat cells, all kinds of cells and things that the body makes any body plant, fish, human, has any kind of light bulb gone off yet in your mind yet, if not then search the net for anti-sense, RNAi, Dr's Fire & Mello, ISIS pharma., etc. etc. it's not the gene's you have it's the RNA that make the gene's.

The only way we'll ever see this applied to medical practice en mass is if it is insanely profitable for pharmaceuticals to invest in it, of that I doubt. I sincerely hope this research is put into practice. That would be amazing!

What's going to really up the anti here is combining this technique for killing cancer cells with methods of extremely accurate application via say... bacterial propellers controlled by targeted magnetics to guide the RNAi into place. (no worry to the rest of the body then) Sounds weird but I know the technology already exists but it's being designed for a different application :( Sometimes kickass technologies don't get to combine do to patent laws and si-uch :P or they just don't know the other exists. It's getting ridiculous at how much technology is being developed in every single field of the sciences.
I digress.
RNA1 is already in human trials and gene therapy is already being successfully applied for multiple ailments already.
So don't go sayin you never hear of this stuff. You've got to listen to hear it :P
I'm puttin that in a song...

You've gotta listen to hear it
You've got to find it to hold it
Don't let anyone tell you you have to let it go!
Streak across the sky find a way to hide.
Then trouble sinks into your broken mind!
Take the truth out of the lies.
Taste the essence of bad alibis.
Choose the life of endless dreams.

I'll put that in my second single lol
It's sounds better sung... trust me.

the treatment to cancer and all biological problems is holistic medicine...we should not focus on how to kill the cancer but how to prevent it

Now the last time I heard about a drug that cured all cancer, it also ended up curing everything else including J-walking and mutated a select few humans into super zombies that murdered the rest of the human race.

Who said stem cells were able to cure cancer? They’re more likely to form a tumor than do that. They're more for injuries and degenerative diseases, at the moment anyway.

The most interesting part of this is the delivery system, and we heard very little about that.

Disrupting translation of proteins is a powerful but dangerous process. As the article points out, if this "drug" (in quotes only because I can't see calling RNA a drug) manages to make its way into other cells than it's meant for, it could cause serious--possibly lethal--side effects.

So we know how to target liver cells: using a fatty membrane. But with, for example, the Huntington's RNAi treatment mentioned here, how do you target only neurons and not any other type of cell? Not to mention that with Huntington's, the mutated protein still needs to exist to be healthy, so you'd not only need to suppress the mutant proteins but add the normal ones. I'm assuming this would still require lifelong transfusions, then?

So, as exciting as this is, there's a lot to consider.

-IMP ;) :)

All the nay sayers. BooHoo!! What if Edison had listened to the guy that said," Nice, but if you stare at the bulb for hours you go blind".

We are going to need much more than an article for this to pan out well...

Now we just need to find a 'political' killer to eradicate all the bad politicians from office to restore America's health.

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Maler/Femaler, relative Therapychemo you.

… ever is uneven any drugging but never was not even one drugst unever, bey quadrate. Mean, all drugs are always without theory because with practum is also not the real drug …

Amazing. Hopefully it develops into something that works in my lifetime. The next 20 years might see the cure for many diseases and the big problem will then become overpopulation.

The current health care system and the pharma industry is profit-based. Until this changes - and unfortunately I don't think it ever will - the money is in treating the symptoms, not curing the actual disease.
Just like the article said, they are mopping the floor instead of turning off the faucet, and from their standpoint, it's understandable. What makes more economic sense for a company: spending a fortune on developing a medicine that cures, which the patient uses for a short time and that's it, or treating the symptoms for many many years for a continued flow of revenue?
I'm not a doctor, so I can't really judge whether the method described here is really viable or not, but as an economist and from seeing how big companies operate, the more this would work, the more it will be buried.
Shame on us actually (I mean all people on this planet), we're letting them do this to us.

check the tech journal,face punch,times of india and others for similar coverage of topic

@IceMetalPunk...thank you for posting the most intelligent comment on this article

@CeeZee...I'm fairly certain with RNAi that you have to keep taking it since it isn't very long lived in the cell and eventually the cell will start to translate the harmful proteins again. If anyone knows differently please correct me

I understand the drug targets the mRNAs coding for VEGF and KSP which in the liver are produced only by cancer cells. That's fine so far and lauded. The question remains whether this drug could target those proteins in organs where they are needed OR can the drug can be modified to target specific mRNAs of cancer cells in other organs without damaging the proteins that those organs need for functioning?

@IceMetalPunk

Yeah actually Huntingtons would be easier because there are very simple mechanics for effecting neurons only. Neurotransmitters, like serotonin, dopamine, and others are all released from the end of the cell and effect the next. After their effect has been had they will return to thier home cell. Drugs that alter this "re-uptake" process by either increasing or inhibiting it are commonplace if not Over-perscribed. Using one of the re-uptake sites, you could easily insert the chemicals into a neuron.

You'll get into only one foreseeable (by a mechanical engineering guy) problem in that often with other substances that alter these processes, they can be stored in fat elsewhere in the body. I think leaving this in highly controlled Hospital conditions would limit risks here greatly, but it is a definite concern.

So yeah: liver, stomach, large intestine, nerves, and skin tumors/conditions would benefit fast than other body parts because these areas have easily accessed cells or local processes that can be exploited (like the fat envelope being dissolved by Bile in the liver).

But anyway, to the writer: try and call these guys back and get them going on brain tissue. I know a guy who needs a hand on that front.

Interesting! This research is very important to me since my father has cancer.

11/22/09 at 6:46 pm
As long as their is a strong Medical Industrial Complex, we will never have a Disease free world! They don't want to cure anything, for if they do the money train shuts off!

All they want to do is Doctor, Doctor, Doctor. This is getting out of hand, with the Center Disease Control and the World Health organization, and all this Flu Vaccine, which is a money machine for wall Street and their stock holders. Want the Flu and Cold killer, Nasal B-12 Cobalt, up the nose, will kill any respiratory disease on contact.

It is very simple to have a Disease Free World by Fasting, and Semi-Fasting, by Protein Management. All Viruses have a double shell of protein around them, and will die if they don't have protein by 5 to 7 days. Bactria's heart is protein, and if it doesn't have protein between 3 to 5 days, it dies. Cancer cells can die within 7 to 15 days, with out protein.

Why do you think when a person who goes to a Fasting retreat, as a Basket case, and in two weeks comes out CURED, and is a new person. Because the body didn't have a new protein, and the viruses and Bacteria starve to Death, as the Medical Industrial should! It doesn't take a Einstein to figure that out! Most all medical Research is lost in the Forest, for all they can see is just the trees.

The medical Industrial Complex is killing Americans, with their drugs, Vaccines,Insurance Costs, and it is about time Americans realize it, and start taking Political action and control against this bureaucracy. Are they going to start giving Americans a Different Vaccine Every month? This just getting out of hand!

yet another hoax of chemistry as usual. Keep the kitchen example of leak and mob because that example is good for everything in isolated science of empty nuts!
Keep mobbing and never ever get to the bottom ground of fixing the leak forever because that would be bad for business and the revolution in science we do need so badly remains wishful thinking.
energy based background to all organic life forms did crack all cancers and deseases already time ago but as long as you applaud to the ¨we have a new pill for everything¨ (patent, production, sales, dependence)of secondary or complementary science of biology and chemistry no good to humanity can happen.

potsona1, which type of cancer does have your dad? If you need assistance write me, you wont loose nothing but maybe gain a lot. I am Dr. H G Ludwig of an international NGO, ong.asfam@gmail.com

mix me up a batch... I volunteer

if you have to ask why, then you probably wouldn't understand the answer anyway...

That is absolutely amazing information, thank's for sharing.

Smoke Remedy

O.k. Cure all deseases. What are we going to do with all those healthy humans that refuse to die?

Great news. This means I can go out and get drunk everyday, and get my liver all screwed up, right? But in all seriousness, I'm still a bit skeptical. I always hear news of the next groundbreaking cure for cancer almost every month, and none of these promises came true. I am optimistic though. I actually own a startup that donates 50% of its profits to curing cancer non-profits: www.dailysnap.com

This is VERY interesting to me since I have liver cancer at the moment. I will mention this to my oncologist.

This is regretfully going to end up with all the other possible cures. People are no longer easily fooled. Yes, it is great that this could be a multipurpose cure. However, the reality is sad. It was mentioned in one form or another. Our health system is based on revenue and profit. Being non-profit does not mean no revenue. Our health care system is already on the verge of collapsing. People must get sick, get treatment and pay. That is the only reason that U.S. Universal health care is sought. Too many people just stopped paying for health insurance because they couldn't afford it. Then they would go to the hospital and get care and not pay the bill. Others just lived with the illness and suffered and/or died. It this protein really can do what it says, it will be sold to the pharmaceutical companies and they will bury it. If someone tries to sell it, it will be discredited. It is the same story with battery technology. The US can not have the average life span to jump to 100. It is great to hear, but the reality is that it is highly unlikely we see a cure for any disease or illness. Only a treatment. It's just business. Nothing personal.



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