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Experimental Drug being tested on Liver Cancer patients
"There is a new experimental drug being given to 19 patients with liver cancer, who haven’t responded positively to chemotherapy, and the drug has been shown to be effective by preventing the malignant tumors from making proteins they need to survive. The new drug is raising hopes that an effective treatment for cancer may at last be found. What’s more, the drug could possibly be redesigned to treat hundreds of other diseases.
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."
Originally Posted: http://thetechjournal.com/science/a-new-protein-killer-could-be-the-much-sought-after-cure-for-cancer.xhtml#ixzz1CdJewp7w
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."
Originally Posted: http://thetechjournal.com/science/a-new-protein-killer-could-be-the-much-sought-after-cure-for-cancer.xhtml#ixzz1CdJewp7w
Yes this is clear progress if it can treat liver cancer at an advanced stage and is a more efficacious and targeted form of treatment. At this point from what I can read it is still in the early stages of testing and controlled studies on a larger scale to determine further results will be the next step but if they show the same or similar results to the early phases of testing then this would clearly be an advance in treatment and would be able to help treat people who now are beyond treatment or wont respond to it well. This kind of progress as regards treatment is crucial and hopefully other companies will continue this kind of research as well.
http://www.news-medical.net/news/20110606/Alnylams-ALN-VSP-Phase-I-trial-data-on-liver-cancer-presented-at-ASCO-2011.aspx
FDA approval seems to always be a slow process, but hopefully this drug will prove to be as good as it seems and maybe they will fast track it - doubt it but time will tell..
Yes this seems quite promising and if the manner in which this medication works could be duplicated with other disabilities or illnesses it would open lines for whole new areas of research. One of the reasons it has been so hard to find fully effective treatments for cancer is although there were increasing effective means at killing the cancer cells that were part of the abnormal growth that causes cancer there was no fully effective means at turning off the cause of this abnormal growth rate among cells to begin with so that it would reemerge in other cells and potentially metastasize. Turning off the means of production of the cells would of course be a breakthrough and hopefully this treatment can be realized and FDA approved.
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