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AstraZeneca Boards RNA Bandwagon

AstraZeneca today became the latest drug maker to bet on a young technology known as RNA interference. The company just made a deal that could ultimately be worth about $400 million with Silence Therapeutics, an RNA specialist.

RNA interference (RNAi) is based on basic research less than a decade old that showed that RNA sometimes suppresses the activity of certain genes, such as those that wind up in the wrong part of the genome, or those carried by viruses. Two scientists won last year’s Nobel Prize for that work. Industry hopes to use the insight to develop drugs that shut off genes that cause disease.

Merck bought Sirna Therapeutics, an RNAi specialist, for $1.1 billion last year. Alnylam Pharmaceuticals, another player in the field, has a $700 million deal with Novartis. And Pfizer in-licensed macular degeneration drug from Silence and another company.

In the deal announced today, Silence will work with AstraZeneca to develop RNA molecules designed to silence as many as five specific targets identified by AstraZeneca. The research will focus on respiratory illness. AstraZeneca’s upfront payments included a payment of about $5 million and an equity stake worth about $10 million. The rest of the deal is tied to development and commercial milestone payments.

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    • It's interesting about all this. It may be very important to zero in on different genes and proteins. However, when actually taking the "targeted" drugs that come from this research, do the drugs even enter the cancer cell? Once entered, does it immediately get metabolized or pumped out, or does it accumulate? In other words, will it work for every patient? No!
      What needs to be done is to sort out what's the best profile in terms of which patients benefit from this drug or that drug. Can they be combined? What's the proper way to work with all the new drugs? If a drug works extremely well for a certain percentage of cancer patients, identify which ones. If one drug or another is working for some people (not average populations) then obviously there are others out there who would also benefit.
      What's good for the group (population) may not be good for the individual, affirms that in the tactic of using "fresh" biopsied cells to predict which cancer treatments will work best for the individual patient, these "smart" drugs have to get inside the cells in order to "target" anything.
      So, all the validation of this gene or that protein providing us with a "variety of sophisticated techniques to provide new insights into the tumorigenic process" (boy!, there is another new buzzword!), if the "targeted" drug either won't "get in" in the first place or if it gets pumped out/extruded or if it gets immediately metabolized inside the cell, it just isn't going to work.
      Each of these new "targeted" drugs are not for everybody. Even when the disease is the same type, different patients' tumors respond differently to the same agent. Upgrading clinical therapy by using drug sensitivity assays measuring "cell death" of three dimensional microclusters of live "fresh" tumor cells, can improve the situation by allowing more drugs to be considered. The more drug types there are in the selective arsenal, the more likely the system is to prove beneficial.
      Hello! cancer medicine, is anyone out there???

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