A Protein Killer Could Treat All Cancers, and Possibly All Illnesses

[n2doc]

By Corey Binns


Choking a Tumor MRI scans show that blood flow 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.

more

http://www.popsci.com/science/article/2010-07/rx-every-disease

[trotsky]

1. This is wonderful news. K&R. n/t

[yellerpup]

2. So exciting.

Hopeful developments! :kick:

[Bitwit1234]

3. The tears are flowing down my face

my sister passed away with liver cancer from metastasized breast cancer. She had the inflammatory kind. I am recovering from breast cancer tumor. I have been so worried that it metastasized because they found some cancer cells in the lymph nodes. THIS GIVES ME HOPE. THAN GOODNESS. AND THANK THE SCIENTIST WHO I THINK DESERVE THE MILLIONS AND MILLIONS THAT CEO'S AND SPORT FIGURES GET.

[katandmoon]

4. Best wishes to you and I share your sentiments.

[mike_c]

7. .

:hug:

[supernova]

8. All healing to you

:hug:

[Duppers]

9. ...

hugs from me too.
:hug:

I hope you contact Duke University or Johns Hopkins or UCLA Medical Center, etc. to get into their Alnylam program.

Best wishes to you!


p.s. And you're right: Scientists do deserve more appreciation and compensation! These things are more important than our damn entertainment.

[Deep13]

5. Sounds very promising. nt

[supernova]

6. This is wonderful news

I know that for many years researchers have been working on ways to cut off the blood supply as a way to stop tumor growth.

Ready in two years for the market? eek! don't know about that.

I'll be watching. But congrats to the researchers involved. Cancer research is just so damn depressing most of the time; it's good to see a positive development.