I've got good news and bad news. I'll give you the good news first:
A cheap and simple drug that kills almost all cancers by switching off their "immortality". The drug, dichloroacetate (DCA), has already been used for years to treat rare metabolic disorders and so is known to be relatively safe.It also has no patent, meaning it could be manufactured for a fraction of the cost of newly developed drugs.
Evangelos Michelakis of the University of Alberta in Edmonton, Canada, and his colleagues tested DCA on human cells cultured outside the body and found that it killed lung, breast and brain cancer cells, but not healthy cells. Tumours in rats deliberately infected with human cancer also shrank drastically when they were fed DCA-laced water for several weeks.
DCA attacks a unique feature of cancer cells: the fact that they make their energy throughout the main body of the cell, rather than in distinct organelles called mitochondria. This process, called glycolysis, is inefficient and uses up vast amounts of sugar.
Until now it had been assumed that cancer cells used glycolysis because their mitochondria were irreparably damaged. However, Michelakis's experiments prove this is not the case, because DCA reawakened the mitochondria in cancer cells. The cells then withered and died.
And now for the bad news: Michelakis can't get any drug companies to fund a clinical trial, which can cost several hundred million dollars to conduct. Because DCA doesn't have a patent, and costs pennies to produce, there's no potential payoff for the corporations. Unless a non-profit or government steps forward, this research will remain stuck in the lab.
Chances are, of course, that DCA won't turn out be a miracle cure. (This isn't the first anti-cancer drug to look great in the lab, and it won't be the last.) But it still seems ridiculous that a promising treatment is being ignored because it's too cheap. If I were Nancy Pelosi, I'd take the money we will save by negotiating directly with the drug companies, and divide it between the NIH and a new agency, which will undertake clinical trials for promising drugs that Big Pharm isn't interested in.
Update: I think MattXIV, in the comments, really nailed the issue:
This is a major blindspot of the incentive structure of the patent/FDA approval system. If you can't patent the compound, it is often impossible to make a profit on it after the expenses of clinical trials. DCA as a compound may be cheap, but DCA as a drug is expensive, because it isn't considered a legit drug until it goes through clinical trials, which aren't cheap whether the money is recouped by charging monopoly prices for the finished product or collected via taxation.
Hat Tip: Ezra


Comments (7)
I can't believe you bought the whole spin on this story so uncritically.
Some points:
1. It does not cost several hundred million dollars to conduct a clinical trial. That figure is what is usually quoted as the entire cost to bring a new drug to market, from discovery costs, to animal research, to regulatory filings. It's the cost of the whole package, from idea to marketable drug, and that whole package can run up to a billion dollars.
2. To run a small preliminary trial to determine if there is evidence of efficacy could be done for costs that are well within the means of an investigator, if he's willing to apply for grants. True, the funding climate sucks these days, but if this guy is funded by grants from the Canadian Institutes for Health Research (CIHR), Alberta Heritage Foundation for Medical Research (AHFMR), and Canadian Foundation for Innovation. He's perfectly free to apply to the NIH and other organizations for funding. Again, he would not need "several hundred million." All he would require is a few hundred thousand dollars for a small trial, between $1 and $2 million for a larger study, which is par for many research grants.
3. If the smaller human trial shows evidence of efficacy, then and only then is it reasonable to get serious about setting up the much larger clinical trials needed to see if this drug. Before we see that, talking about "hundreds of millions of dollars" is ridiculously premature. Lots of drugs show serious promise in mice and are less than impressive (or even ineffective) in humans. If this drug shows promise in humans, then it might be necessary for the government to step in.
4. Lastly, there's nothing stopping the investigator from patenting the idea of using this drug to treat cancer. I know someone who is doing just that for a use of a drug that's FDA-approved for something totally unrelated to cancer. I sincerely hope that he has, in fact, done that, because now that the results are published it's too late now. If he has done that, he could then license his idea to whatever pharmaceutical company was interested.
Do try to do a little more research before the knee-jerk response. I do hope that Seed Magazine doesn't publish anything like this without some better background and nuance.
Posted by: Orac | January 18, 2007 5:13 PM