Jan 30 2007
Yesterday Jeff asked:
“I just read a story about a new drug that apparently cures most types of cancers. The problem is that it is not patented so no drug company will pay for the trials. It just sounds too good to be true. Here is the link.”
There has been a media flurry surrounding this new study by lead author Dr. Evangelos Michelakis, in which he found that dichloroacetate (DCA) can selectively kill a wide range of cancer cells.
Basically, in most cancers the mitochondria are turned off. Mitochondria are the little energy factories inside every cell. They also are responsible for triggering apoptosis – programmed cell death. Cancer cells make their energy outside the mitochrondria, their mitochondria turn off, they lose the ability to trigger apoptosis, and they become immortal. Being immortal is part of what makes them cancer. DCA turns mitochondria back on, which in Dr. Michelakis’s study caused the cancer cells to immediately die, while having no affect on healthy cells.
This is an exciting discovery that is sound in principle and likely to have implications for future cancer research and treatments. And Dr. Michelakis’s study is very hopeful. But (here comes the skepticism) his study was carried out in vitro – on cells in test tubes. Before we get too excited we need to carry out clinical trials in humans with cancer. Experience has shown that it is difficult to predict how a drug will act inside the body based solely on in vitro studies. Even animal data – although very useful – has its limits. We need to prove that the drug will get to cancer tissue in sufficiently high concentrations to kill the cancer cells, and that it won’t just suppress the tumor for a while. We also need to make sure there are no unforeseen negative consequences.
Historically there have been frequent laboratory discoveries that seem to show a promising new treatment for cancer, but when studied in humans the promise is not realized, or the effect is much more modest than was hoped. Cancer research has slowly ground forward, and we have made steady progress, but the “magic bullet” has never been found, despite frequent false alarms heralded in the press. So at this point in time the rational outlook to have is one of cautious optimism. Certainly this is a promising discovery, and it deserves to be studied clinically. We can also hold out reasonable hope that this will turn out to be more than an incremental improvement and will actually be deserving of the moniker “breakthrough.” Let’s do the research and cross our fingers.
There is another very interesting aspect to this story, mentioned in Jeff’s question. DCA has been around for awhile, so no pharmaceutical company can patent it (it is already in the public domain). This means that it is unlikely a pharmaceutical company will pay the millions of dollars needed to fund the research for a drug it cannot own the patent on. I don’t think it’s impossible, just unlikely. As an advantage, the drug is already well studied and so many of the preliminary hurdles have already been overcome. Therefore the cost of research would be much less than if a company had to start from scratch with a new drug.
But let’s assume that the bean counters at all the pharmaceutical companies calculate that the return on investment would not be sufficient to justify the research. And let’s further assume that the PR value of “curing cancer” is not deemed sufficient either. That does not mean that DCA will not be researched.
There are other ways to fund research. Universities fund research programs, programs that also support their infrastructure with money they get from doing pharmaceutical company funded research. So university researchers can use their own resources to do the studies necessary – as Dr. Michelakis and his group have already done. Also, the government funds research through the National Institutes of Health (NIH), which includes the National Cancer Institute (NCI). There are also many charitable organizations that could potentially fund such promising research.
The bottom line is that if the science is solid and the potential very promising, the research will likely get done. Careers can be made from such research, universities could fund cancer centers and raise their prestige and profile, and there could also be a great deal of money to be made from such a treatment (even if it is not as much as from a patentable drug).
There persists a nonsensical and very cynical myth that “the medical establishment” or the pharmaceutical industry already has a cure for cancer, but are suppressing it in order to protect their profits from treating (rather than curing) cancer. This episode shows, in my opinion, how silly that myth is. Researchers are doing university-based research to show the potential of a substance that cannot be patented as a cancer treatment. The research has been published and so is available to the public, and the media are spreading the word far and wide. No conspiracy has prevented this research from happening or knowledge of it from spreading. I predict no conspiracy will prevent the research from going forward. Of course, if the clinical trials do not pan out as hoped then the conspiracy theorists will claim that as proof of their delusions. I sincerely hope that the research does prove DCA or a similar compound to be a radical new treatment for cancer – mostly because I would love for there to be such a treatment. But also because it would be a nice nail in the coffin of “hidden cure” conspiracy theorists, and would validate my prior predictions of how such an event would unfold.
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