GELFAND’S WORLD-In a previous article, I wrote of the case of Liza Cozad, who is dying of a rare brain tumor. She wants the option of being treated with a drug that the vast majority of scientists and doctors feel is worthless.
We are faced with a governmental decision to deny treatment to a dying woman, however bogus that treatment may be. Cozad's friends and followers including rock star Sammy Hagar have done benefit concerts for her and signed onto an internet petition. I would argue that the balance here actually lies on the side of the government, even though my argument will be different from the ones set forth by the defenders of medical orthodoxy. Still, it's not a completely convincing case on either side.
Meanwhile, we have a very different story, that of Josh Hardy, who was first denied, but then granted, access to an as yet unapproved experimental drug. We need to make a distinction between these two cases, and the difference will lie in hard rationality and history.
First, a bit about Josh Hardy. It's a very difficult and stressful story. In his mere 7 years, this boy has had an early cancer. Then he had to have a bone marrow transplant. This was to treat a side effect of his previous treatment. Then, probably due to his weakened immune status, he got a viral infection that most of us would shake off. He didn't. He was in very poor condition at St Jude's, unlikely to survive for very long. Absent some form of antiviral therapy, he probably would succumb.
The big problem in his case is that the kind of virus he got is not the kind that is readily treatable by the standard anti-viral drugs. In his case, if he had had chicken pox or AIDS, it would be treatable with the standard cocktail, but for a double stranded DNA virus, no such luck.
However, the doctors were aware of a drug that is currently in development and which has shown some promising results against several such viruses. The problem for Josh and his doctors was that the drug is not currently for sale, as the clinical trials have not been concluded.
This means in practice that it is not possible to know everything about the safety and effectiveness of the drug until all the results have been tabulated. To be a bit blunt about things, the question has to do with whether the test subjects survive their infections (or not) and whether or not they develop some terrible side effect from the drug itself. After appropriate results, the drug will either be licensed for use on patients, or it won't.
The problem for Josh is that he was effectively terminal, and there was no time left for him to wait for a promising new therapy. The argument in support of supplying him the drug was that there is some credible basis to believe that this drug can suppress infections by double stranded DNA viruses, based on a decade of lab work and preliminary tests on humans.
Still, Josh was not the kind of test case that the drug company was prepared to experiment on at the moment. It just wasn't part of their immediate plan to test the drug on small children, considering that the first priority would be to show that it doesn't kill adults and that it actually helps them.
Due to the modern era's social media explosion, word got out about Josh's plight, and thousands of people petitioned the company and the government to act. It's curious how quickly things can get done when unanimity suddenly appears. The company invented a new clinical trial almost within hours, made some educated guesses at a pediatric dosage (that's really all they could do), and got the drug into the hands of the doctors. We don't yet know whether the boy will recover, but the legal and ethical model for compassionate usage was evident in this case.
That legal and ethical model has not yet been widely adopted, considering that we are only a few days into what may turn out to be a new era in patient access to experimental treatments.
By the way, it is not entirely obvious that throwing away the old rules is a completely good thing. Being careful about introducing new drugs into the American market is something we have generally taken pride in, considering that the drug Thalidomide was kept out of American practice at a time when it was creating birth defects in Europe. The American standard has generally been more conservative than in other civilized places.
But when there isn't much choice, perhaps the standards can be relaxed a bit. Terminal illness is one such situation. If Josh Hardy survives and recovers from an otherwise fatal condition due to this as yet unlicensed drug, there will be reason to reconsider the current practice. Notice that the government was not actually forbidding the dispensing of this drug to Josh, and the creation of an individual trial on the fly (which the drug company expanded to 20 patients) was all done according to standard legal protocols.
And then there is the case of Liza Cozad, a woman in her mid-forties with an inoperable brain tumor that happens to be in a very bad place. Her survival from the time of diagnosis is unlikely to be more than a couple of years, and several months have already run. Cozad and her husband, rock drummer Dave Lauser, have been asking, petitioning, and arguing that she be allowed the option of getting treatment from Stanislaw Burzynski in his Houston clinic.
Burzynski is famous for his introduction of compounds into human cancer treatment originally isolated from human urine. As I wrote previously, he has been treating patients with the compounds he calls antineoplastons for the past 37 years. He flogs his discovery evangelically, and he has built up a small but vocal group of supporters. He has also fought off the law, who have tried repeatedly to shut down his use of these compounds, and have now finally succeeded (perhaps) in forbidding Burzynski the right to add new patients to his trials.
In brief, the FDA forbade the use of antineoplastons on new pediatric patients not long ago (due to the death of a young boy from an overdose), and more recently extended the ban to new adult patients. That's where Liza Cozad comes in, because the ban on new patients occurred at a time that now makes it impossible for her to enroll in a Burzynski antineoplaston clinical trial.
The Cozad case burst into the internet blog universe a few days ago on the pages of Respectful Insolence, the subsection of Science Blogs authored by a cancer surgeon who goes by the pseudonym Orac.
The Respectful Insolence blog had previously taken up the Cozad case (and supplied much of the impetus for previous articles by me) because Orac and many of his colleagues have been extremely critical of Burzynski's treatments, logic, and medical conduct. Earlier takedowns of Burzynski's work were carried out by Quackwatch.com, the website I once referred to as the gold standard in surveillance of medical quackery.
Briefly summarized, the criticism of Burzynski's method is that he has had 37 years to demonstrate whether his miracle cure works or doesn't work, and in all that time, and after seeing probably thousands of patients, there is little or no published evidence that his compounds have ever worked to make cancer patients better. There are a few anecdotal cases of people who had the Burzynski treatment and survived, but that is the way of cancer itself -- some patients who would be expected to be dead in short order have some kind of spontaneous remission and go on living as if they had never had the cancer to begin with. It's not common, and your odds are usually a thousand times better by getting treated for whatever is treatable, but once in a while, people survive under very unusual conditions.
In addition, as Quackwatch explained, there were independent tests of the Burzynski formulation decades ago, and they came to nothing.
There are critical scientific tests for whether or not some new cancer treatment improves upon old treatments. It usually takes a lot of patients and careful statistical analysis to show that some new drug is actually better than the older, cheaper treatment. Once in a while over the past few decades, there have been breakthrough therapies that pass muster. We can think of drugs like Tamoxifen and Herceptin in breast cancer, the androgen inhibitors for prostate cancer, and some remarkable advancements in blood and immune system cancer treatment.
In other fields, we have seen remarkably effective new drugs for high blood pressure, cholesterol, treatments for stomach ulcers that were previously chronic, and a myriad of other effective pharmaceuticals. And of course, the big event for my generation was the triumph over polio in the United States.
What provoked the latest round in the blog Respectful Insolence is that Cozad's husband Dave Lauser wrote into the comments section. His argument, briefly paraphrased, is that his wife is an adult facing certain death unless she can find some effective treatment, and she should have the right to choose how she is medicated.
Lauser rejects most of the standard comments made by Orac and by many of his commenters. Lauser makes clear that whether or not he has to pay for Cozad's treatment really isn't the business of anyone else. It's a strong statement, considering that many of the criticisms of Burzynski's behavior go to the high prices his clinic traditionally has charged. Lauser claims that Burzynski is offering to provide the treatment for free if the government will allow him to provide it.
The remainder of the argument is, one might say, the socially libertarian view that Cozad should be free to subject her own body to the treatment, no matter what Orac or the government want to think about it.
I think you have to agree that this is a fairly powerful argument on Lauser and Cozad's part. It is not an argument in favor of suicide, since Cozad would prefer to live rather than die, it does not deal with parents making unwise choices for their childrens' treatment, and it is not a choice being asserted by a minor. The age of 46 years ought to be enough for anyone to be considered a legal adult.
So for me to argue against this point of view is not easy, and I mostly reject a series of arguments made by commenters on the Respectful Insolence site. These counterarguments can be summarized for the most part as follows: Burzynski is not telling the truth about some severe side effects that accompany his treatment; Burzynski has not come clean about the actual effectiveness, or lack of effectiveness of his drug; Burzynski has never really done a legitimate scientific study of his drug that would really put it to the test and answer the question of whether it works or not.
I think these are all legitimate points. In fact, I believe that I explored them fairly deeply in my earlier piece. But once these points are made clear to Liza Cozad, she is free to consider them in making her decision. She is also free to reject them out of hand, not because it would be a wise or learned thing to do, but just because she has that right.
But there is an additional right that ought to be considered. It is the right of society as a whole, acting in collective fashion, to ensure the safety and effectiveness of its medical practice and its pharmacology. When considered in this way, Burzynski's methods, his ethics, and his drugs all come up short.
The FDA is down on Burzynski because he has failed to do what he promised (carry out clinical trials according to standard rules and practices) while endangering patients. He has destroyed or lost critical data that would be necessary to evaluate his treatments, and he has done all of this in a way that speaks more to his casual contempt for rules than to any devotion to science.
The rules for doing clinical trials exist for a couple of reasons. One reason is mostly historical: At one time, and in some places, medical research was done on people against their will, or absent their informed consent. That last term, informed consent, is central to all modern American medical practice.
It is supposed to really mean what it sounds like -- that you the patient understand the risks you will undergo when any medical procedure or medication is prescribed for you. The propensity of antineoplaston treatment to induce dangerously high levels of sodium in the blood appears to have been understated by Burzynski and his clinicians, and they have failed to report many serious cases of sodium overloads in their patients, even though the law required them to make such disclosures.
There's a whole lot more in the federal findings on the behavior of the Burzynski clinic. Taken as a whole, it argues pretty strongly that Burzynski should not be in charge of a research lab that experiments on human patients.
These findings are damning enough on their own. What clinches the argument is the strong implication based on all of the evidence that Burzynski's antineoplastons don't fight cancer the way he would like us to believe they do.
In the face of substantial research misconduct and a reasonable implication that antineoplastons are not, in fact, antineoplastic (ie: don't cure cancer), there is plenty of reason to bar the Burzynski clinic from carrying on its charade of treating cancer using a therapy that didn't work 37 years ago and has become more outmoded every decade.
The right of the American people through their elected government to protect the American people as a whole is an accepted principle. We take it seriously in regard to the national defense and we take it seriously in regard to public health.
Admittedly, the right to inject selected individuals with a toxic compound simply to make money is a little different than allowing typhoid carriers to work in restaurants, but there is certainly an overlap in terms of protecting the public health. One process may involve active surveillance of infectious disease carriers whereas the other protects the public from choosing fraudulent forms of treatment, but the goal of keeping us healthy and safe is the same.
In this sense, the balance of logic and ethics implies the permanent closure of the Burzynski clinic as a research organization and the prohibition of antineoplastons as human pharmaceuticals.
But then there is Liza Cozad and the issue of how we protect her rights. The socially libertarian concept of letting her choose her own poison (whether literally or figuratively) obviously contrasts with the argument that Burzynski should be shut down. Curiously, there is a solution to this problem, and it was suggested by one of the commenters at Respectful Insolence who goes by the equally amusing pseudonym of Herr Doktor Bimler. (At least I hope it's a pseudonym, otherwise there is a seriously weird set of parents somewhere.) The good doc Bimler points out that the antineoplastons are well out of patent and can be obtained from a chemical supply house inexpensively.
If Cozad is that determined to get antineoplaston treatment, she could find a doctor (perhaps in another country) and get it done. In fact, there is no reason other than knowledge and medical ethics that doctors in other parts of the world couldn't be using Burzynski's compounds right now. There is nothing to stop them except good sense.
But Burzynski himself should not be able to prescribe antineoplastons because of his documented violations of patient safety.
All this having been said, I wonder if Cozad and Lauser are working through their difficulties and fears by making their search for this treatment into some sort of medieval quest. Lauser at least seems to understand that it is unlikely to be a cure, but it gives him something to do and it allows for that shred of hope that they would like to hold onto as long as possible.
The Respectful Insolence author and commenters obviously feel that they are doing a service to Cozad and Lauser by informing them of the hard truth while sparing them the ordeal of being deceived by a quack. I'm not so sure that Cozad and Lauser really want this level of recognition at this time, and would like at least a little more time where they can pretend about a happy future rather than a dismal end.
That's why this is a hard case. It would be much better if there were some credible treatment that is based on a rational, scientific chance of improvement, that Cozad could receive.
High functioning cancer centers like St Jude's mix hopeful sympathy with experimental treatments. If you read their press releases about treating pontine gliomas in children (the same kind of tumor that Cozad has), you can find examples of cheerful doctors offering hope, only to see the children die in reasonably short order. The only difference between St Jude's and Burzynski is that St Jude's is doing actual leading edge treatment based on modern science.
It would be nice if Liza Cozad could find some similar level of clinical trial somewhere in this country.
(Bob Gelfand writes on culture and politics for CityWatch. He can be reached at email@example.com).
Vol 12 Issue 23
Pub: Mar 18, 2014