Here’s a little thought experiment for proponents of “alternative medicine.” Imagine, if you will, a small pharmaceutical company. Founded in the 1970s, it has starts out with only one product, a drug that its founder thought to be a very promising anticancer agent. So enamored of this particular drug was the founder of the company that he left a job with an academic medical center, founded his own clinic, and then his own research institute and company to manufacture the new drug. After first having painstakingly isolated the substances that make up his drug, he later started to synthesize them. Over the years, the company grew and became more famous.
Things looked really good for this little company. Patients were very interested in what it was selling, and its reputation grew and grew in a certain segment of the cancer community. There was just one problem, however. This drug didn’t have FDA approval. What that meant was that it couldn’t be used except under the auspices of properly constituted clinical trials because it’s an unapproved drug. So the company set them up, dozens of phase II trials over the course of a couple of decades. In the meantime, the company published paper after paper in low tier bottom-feeding journals in what is often referred to as the MPU (minimal publishable unit). These papers tended to be quickie cell cultures studies, case reports, and small case series. Somehow, despite racking up over 60 clinical trials over 20 years, the company never seemed to publish the results of any of them, certainly not in a form that would allow other scientists to judge of the drug had efficacy against the tumors claimed. After a while, the company stopped trying, or so it seems, given that the number of publications in peer-reviewed journals indexed on PubMed ceased years ago.
Pretty horrible, isn’t it? You’d be outraged at such behavior, wouldn’t you? And rightly so!
To fund the company, patients who agreed to take part in these clinical trials were charged huge sums of money, tens, even hundreds, of thousands of dollars. Oddly enough, through all these clinical trials, almost every patient received the new drug. There was never much of an effort to do proper randomized clinical trials to determine if the drug really was efficacious against the tumor types being treated. Over time, a group of sycophants, toadies, and lackeys sprang to promote the clinic, claiming the drug could cure even the most horrifically advanced cancers. Stories of “miracle cures,’ all without sufficient clinical evidence presented to allow a reasonable determination of whether or not the tumor shrinkage and good outcomes observed were really due to the new drug. The drug remained unapproved by the FDA or any other governmental body, but that didn’t stop the company from administering it to thousands of patients.
The company also promoted the drug prodigiously, with its founder frequently doing interviews with fans wanting to promote this new drug as a “miracle cure.” A filmmaker whose company specializes in informational videos for corporations made a movie about the company and the man who founded it. In the process, this filmmaker brought more publicity and notoriety to the company than ever before. Around the same time, a cottage industry of websites, blogs, and YouTube channels arose, all portraying the company as a miracle and its founder as a scientific genius. To what degree the company aided and abetted this online promotion is unknown, but it clearly cooperated with the filmmaker and at the very least did nothing to dispel the hagiographies or the claims made for its products in these videos. Meanwhile, the founder of the company gave interviews to an ex-TV star turned hawker of exercise devices and dubious health products. This ex-TV star devoted an entire chapter to the founder and his treatments in a book she ultimately wrote about “doctors who are curing cancer,” basically promoting the founder’s message that his therapy was natural, nontoxic, “not chemotherapy” (it is chemotherapy), and “targeted” to the genetic abnormalities of the patient’s tumor. It ignored all the evidence that the drug company’s treatment had been associated with significant toxicities and even possibly deaths because of its high sodium load. Such complaints, the company dismisses as being inconsequential and the toxicities “minor and easily managed.”
it all sounds very unethical, doesn’t it? You’d be outraged at such behavior, wouldn’t you? And rightly so!
The company also became very insular. Because its drug was not FDA-approved and the evidence that it did what was claimed for it was weak, there arose…critics, skeptics who did not believe the hype, skeptics whose scientific examination of the company’s claims did not come to the same conclusion as the company did, so much so that they viewed the company’s claims as the equivalent of false advertising. The company did not like this. So it hired a man to threaten and bully them, including a teenaged boy who was sent pictures of his house as a clear message that the company’s man knew where the boy and his family lived. This particular PR scheme backfired; so the company was forced to fire the man because he had gone too far. He was now a liability causing more harm to the company’s reputation than good. He was, to put it mildly, a major embarrassment.
Not that the company learned a lesson. Oh, no. It helped filmmaker make a second infomercial for it. This informercial was even more focused on attacking the company’s critics than the first movie. The company then hired a new PR person. Through it all, there remained no convincing evidence from randomized clinical trials to demonstrate the efficacy and safety of its primary drug product, despite 30 years of existence, which led the company to try to diversify into a different product areas. the first area was “personalized” gene-targeted anticancer therapy. After all, genomics has become a hot area, and analyzing tumors to guide therapy is increasingly becoming viewed as the future of oncology. The problem was that the company had no expertise in genomics, interpreting network biology, or using genomic data to guide therapy. so it basically outsourced the job. More promising and lucrative was the company’s foray into anti-aging medicine. In essence, it found a new way to market its main product.
So what would you think of a pharmaceutical company like this? Let’s recap. The company:
- Markets a drug that is not FDA-approved
- Charges patients on clinical trials exorbitant sums of money, leading them to desperate fundraising efforts.
- Runs dozens of clinical trials but never publishes the results.
- Hired an online thug to bully and intimidate critics
You’d be outraged at such behavior, wouldn’t you? And rightly so! Such behavior would be unacceptable, as it is exploitative and harmful to patients. You’d want the FDA or other government enforcement agency to swoop in and do something. You’d want the state government to do something to stop this pharmaceutical company. You’d want the state medical board to shut down the doctors using this stuff without its having been FDA-approved. You’d want it to be prosecuted to the fullest extent of the law, wouldn’t you?
Sure you would! And rightly so!
Yes, if this pharmaceutical company’s name were Plexxikon, or if it were larger, for instance Merck, Sanofi-Aventis, Bayer, or Bristol-Myers Squibb, and it did these sorts of things, you’d view it as confirmation of your view of rapacious pharmaceutical companies concerned only with profits and not with patient well-being or science. You would certainly not believe its spokesperson if he said that the company is only doing it to help as many patients as possible because its founders “know” that its drug works so much better than current therapy. You wouldn’t believe the filmmaker who is producing the infomercials about the company and its “miracle” drug when he says that the company doesn’t charge patients for its drug or to be on its clinical trials but rather charges a “case management fee.” You’d consider that so transparently a dodge that your intelligence would be insulted that anyone even tried that line on you, even more so after you learn of a lawsuit by a woman dying of cancer whose complaint is that the company requires patients to buy all other drugs used from a pharmacy it owns at inflated prices. You wouldn’t believe the company’s claims that the government was out to get it. In fact, you’d wonder why the government has been so easy on it. You’d wonder why the company can keep registering phase II trials for an unapproved drug, charging patients, while seemingly never publishing its results. You’d wonder how it could apparently keep using the drug even in patients who are apparently not on clinical trials, even though the drug is not approved.
You’d wonder all this, unless, of course, the pharmaceutical company were a “research institute” run by a man named Stanislaw Burzynski. Then it’s all OK, the government really is out to get the company, and he is to be defended tooth and nail at all costs.