Harnessing the generosity of kind-hearted strangers to pay for woo

With the recent passage of President Obama’s health care reform bill, I can only hope that we as a nation have finally begun to address what has been a serious problem in our health care system, namely the unemployed. Although I remain somewhat skeptical of the plan as passed and remain largely agnostic about it, as a cancer physician I can’t help but hope that it actually does some good. Although I can say that I have yet to see a patient with breast cancer who wasn’t able to get treatment because of lack of insurance, I have seen a lot of difficulties thrown into their paths. For instance, in my particular state Medicaid won’t kick in until a diagnosis of cancer is made. The problem is that, just in my practice alone, I’ve seen several patients over the last year who couldn’t afford to get the biopsy necessary to diagnose the cancer. Fortunately, the Komen Foundation has a fund that helps women like this, but, given the economic downturn, it’s under enormous strain. Consequently, even at an institution whose mission includes taking care of the poor and less well-off, we have trouble meeting the need as the numbers of uninsured continue to climb.

As a consequence of this, more and more I seem to be seeing something known as the “medical fundraiser.” I first noticed it last fall in a coffee house, when I saw not one but two ads, one for a woman with leukemia and one for a two year old girl with neuroblastoma, both of whom were apparently uninsured and couldn’t afford their medical bills. Both flyers advertised fundraising concerts, one at a local bar and another at a local park. It was a profoundly depressing thing to see.

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Unfortunately, one thing I also learned was that these fundraisers are not always about science-based medicine. In fact, a lot of them are about paying for woo and sometimes even quackery, in which case it doesn’t matter whether or not a person has insurance because insurance companies quite correctly don’t pay for quackery. For example, I saw this flyer at a local business:

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Note what’s there. While no one would have any problem with paying for speech therapy, occupational therapy, swim lessons, and applied behavioral analysis, the latter of which can be quite expensive, what’s the deal with homeopathy?

Homeopathy?

Yes, homeopathy. And how is it that homeopathy is the second most expensive treatment on Colton’s list of treatments for his autism? At $800 to $1,000 per month, that’s some seriously expensive water! Wow! I’m definitely in the wrong business. I mean, here’s a homeopath making $1,000 a month treating just one autistic patient with magic water! I guess the best that can be said is that it’s not harming the child; after all, it is nothing more than water supposedly imbued magically with the “memory” of the substance formerly in it that the homeopath has diluted to nothing. However, homeopathy is wasting money–a lot of money for this child–and anyone who has contributed to this child’s treatment should know that about 25% of the money is going to pure quackery that has no hope of helping the child.

What reminded me of these flyers whose photos I snapped with my trusty iPhone last fall was a story I saw the other day that I wanted to write about for yesterday, at least until grants got in the way. Entitled Friends, strangers help pay unwieldy medical bills through fundraisers article’s a few days old, but I only just saw it on Sunday. it shows exactly what I’m talking about, albeit through a very credulous reporter who doesn’t really distinguish between woo and science-based medicine in what she no doubt saw as a human interest story about how lack of insurance and hard economic times are pushing people to have fundraisers like these to try to pay for their medical bills:

Facing the most advanced stage of thyroid cancer and given at most five years to live, Rene Louis awoke one night last summer with a dream about how she could help others like her raise money to pay their medical bills.

Louis, 40, of Allen Park, Mich., needs to pay $14,000 up front to be treated by an alternative medicine doctor in Houston. So she started the I Promise Campaign to help her pay for it. She hopes to get nonprofit status and raise enough money to cover not only her treatment, but the medical bills of others as well.

It’s not good “if you have hope and you can’t get it,” said Louis, referring to the treatments she wants but can’t afford. She has two children, ages 10 and 8, whom she hopes to leave with “good life lessons about making good out of bad.”

Louis, it turns out, is insured, but wants to pursue dubious therapies:

Given deductibles many insured people must pay before their insurance coverage kicks in, and with co-pays that mount for patients with serious, catastrophic illnesses, many Americans may need help for years to come. Others like Louis, who is insured, need help because they have insurance policies that won’t pay for alternative treatments not proven as safe and effective as conventional therapies.

Obama plan or no Obama plan, it’s a disgrace that, in the U.S. in 2010, many people with cancer, mostly uninsured but even those who are insured, debts that they cannot pay leading to potential financial disaster and thus feel they have no choice but to hold fundraisers of various sorts to pay for their care. Unfortunately, among this wave of people is a subset who have decided that, insured or not, they want woo rather than science-based medicine, and, for whatever reason, they’re riding the same wave as the unfortunate uninsured people with life-threatening illnesses who can’t pay for life-saving medical care. They’re appeals that are very hard to resist, too, as this video of Rene Louis describing her plight on her website shows:

Whose heart wouldn’t be melted–or at least softened–by the plea of a woman diagnosed with what is very likely terminal illness? Who among us, if placed in Louis’ situation, might not feel the siren call of an unproven and highly dubious therapy whose practitioners claims complete responses in advanced cancer and gives her hope of logn term survival when conventional medicine cannot? I can’t even guarantee that I would, although I like to think I would. Apparently Louis has metastatic medullary thyroid cancer. Like so many solid tumors, once it metastasizes beyond the regional lymph nodes medullary thyroid is no longer curable. In the case of this specific cancer, the five year survival rate is excellent with surgery if there are no metastases beyond the lymph nodes in the neck; once the genie’s out of the bottle and the tumor has gone to lung, liver, or wherever, the five year survival is around 20%. That’s why, when Louis asks the viewer what he or she would do in her situation, who among you doesn’t feel your eyes welling up? Just a little bit?

Adding to the potency of the appeal is the fact that conventional medicine can’t really offer Louis much in terms of therapies that will save her life (although it most certainly can offer a lot that will palliate her). Consequently, when she proclaims that the “alternative” she has found gives her hope, it produces an incredibly powerful emotional appeal. Unfortunately, the “hope” that Louis has found is not real hope at all, but false hope:

Rene found a clinic in Houston Texas that has some success with cancer treatments. Insurance companies were skeptical to the Dr’s claims, putting the burden on the patient. That’s when the idea of this Campaign was born. Not having the money needed for any possible treatments, Rene decided to ask for help. She went public with her plea for any assistance that could be given. Her promise was to “pay it forward”, meaning any money raised over and above what was needed would be shared with others in similar situations.

She’s talking about Stanislaw R. Burzynski, MD, PhD.

Dr. Burzynski is a rather difficult woo-meister to tackle for a variety of reasons. First, he really is a legitimate MD/PhD, proving beyond a doubt that having an MD/PhD double threat degree does not necessarily inoculate one from falling prey to pseudoscience. He’s also an example of a brave maverick doctor (specifically the “iconoclast” type) who’s discovered The One True Treatment for cancer, HIV, and a wide variety of other conditions. In Dr. Burzynski’s case, his treatment of choice is something he once dubbed antineoplastons, and, like all brave maverick doctors, be they Mark and David Geier, Andrew Wakefield, or Robert O. Young, he believes himself to be a crusading researcher rather than a quack. He also gives good science-y speak in that he has many of the trappings of a reputable scientist, including publications in reputable journals. Come to think of it, that’s just like the Geiers or Wakefield too, all of whom have managed to insinuate themselves into the medical literature. Like Wakefield (but unlike Mark Geier), at one time Dr. Burzynski was actually viewed as a promising young researcher. Unfortunately that time was well over 30 years ago, when he was on the faculty at the Baylor College of Medicine. It was there that he discovered what he would later call “antineoplastons.”

Like many brave maverick iconoclasts frustrated with the “arrogance,” “blindness,” and “inflexibility” of his academic colleagues, Dr. Burzynski apparently ultimately decided that academia was not for him (or maybe the decision was made for him); so he founded his own clinic, which offers a “variety of alternative cancer treatments for patients diagnosed with over 50 different types of malignancies, including colon, pulmonary, breast, prostate, head and neck, ovarian, pancreatic, esophageal, hepatic, renal, bladder, brain, malignant melanoma, lymphoma, and many others,” and research institute, which is currently described as “a biopharmaceutical company committed to developing treatment for cancer based on genomic and epigenomic principles.” Apparently it’s not just about the antineoplastons. Since then, it’s been a life of woo for Dr. Burzynski, and of late he appears not to be publishing in anything resembling reputable journals anymore. A PubMed search reveals no publications since 2006, and the closest I find to a recent publication in a reputable journal is this report in 2004 of a single arm uncontrolled trial in children.

In any case, Dr. Burzynski first gained fame for his antineoplastons back in 1988, when Sally Jesse Raphael featured four “miracle” patients of Burzynski, who, according to her, had had incurable cancer and failed conventional therapies but were then cancer-free, thanks to Dr. Burzynski. Unfortunately, four years later in 1992, Inside Edition followed up these four patients:

In 1992, “Inside Edition” reported that two of the four patients had died and a third was having a recurrence of her cancer. (The fourth patient had bladder cancer, which has a good prognosis.) The widow of one of Raphael’s guests stated that her husband and five others from the same city had sought treatment after learning about Burzynski from a television broadcast — and that all had died of their disease. In 1995, a federal grand jury indicted Burzynski for mail fraud and marketing an unapproved drug. The indictment charged that he had billed insurance companies using procedure codes for chemotherapy, even though his treatment was not chemotherapy. He was tried in 1997 but not convicted.

Not surprisingly, in 1998, the State of Texas secured a consent agreement with Burzynski stipulating that he: (a) cannot distribute unapproved drugs in Texas; (b) is allowed to distribute “antineoplastons” only to patients enrolled in FDA approved clinical trials, unless the FDA approves his drugs for sale; (c) cannot advertise “antineoplastons” for the treatment of cancer; and (d) on his website and in promotional material his ads must have a disclaimer that the safety and effectiveness of “antineoplastons” have not been demonstrated. Obviously, big pharma got to Texas.

It also turns out that the substances that Burzynski claimed to have isolated from urine (his “antineoplastons”) have never been shown to do anything in cancer by anyone other than Burzynski. As the article at Quackwatch points out, the National Cancer Institute could not replicate Burzynski’s results. Neither could drug company Sigma-Tau Pharmaceuticals. Neither could the Japanese National Cancer Institute. So what we have here is a therapy that has marginal (if that) credibility based on prior probability. AFter all, it’s not entirely implausible to think that the body might make substances that arrest the growth of cancer. One might even postulate that it is a deficiency in these substances that allows cancer to develop. Unfortunately, there is no evidence for this, other than from Burzynski. If there’s one thing that should raise a red flag for pseudoscience, it’s when only one scientist can produce the reported results and no one else can. Replication is utterly key to the acceptance of science, and if other groups could have replicated Burzynski’s work I might scratch my head and say, “You know, Dr. B may be on to something there.” But no one else can, and I’m left scratching my head and wondering how so many people can believe in Burzynski’s results in the absence of any good science.

Faced with a consent decree mandating that he can only use antineoplastons in the context of a clinical trial, Dr. Burzynski used his institute to set up a staggering number of clinical trials. As noted by The Cancer Letter, these clinical trials failed to meet minimal standards for scientifically sound studies. How these things got approved by any institutional review board, I have no idea, unless Burzynski made like the Geiers and created his own IRB. However he did it, what he is doing is sloppy at best, grossly incompetent at worst. Here are some excerpts from The Cancer Letter:

Howard Ozer:

Dr. Burzynski is studying a heterogeneous, ill-defined patient population.

He treats patients who come through the door, and only patients who come through the door. He takes patients with bony disease, liver disease, bone marrow involvement, CNS disease. He organizes data by disease site, whatever the patients’ stage, and whatever treatment they received prior to walking through the door of his clinic.

What we have here are bad trials that could never get past peer review of any clinical trials cooperative group. It’s not in the public interest to conduct trials that are not going to yield clear results. If you are going to test an alternative approach, you need to test it as rigorously as you do mainstream approaches.

Dr. Burzynski’s protocols are written with all the trappings of protocols. They look like protocols. They smell like protocols. But they lack the rigor of protocol design that defines the patient population, defines the endpoints, sets exclusion and inclusion criteria, and allows for statistical analysis.

The protocols are evaluating a single statistical endpoint: response. He doesn’t evaluate disease-free survival, time to progression, quality of life, or overall survival. With these endpoints not prospectively defined, he has no basis for making legitimate claims regarding these parameters. This is a fundamental problem: You have to set your endpoints prospectively. It’s too late to go back and do it after all the patients are treated.

Dr. Burzynski presents no baseline data. He presents no control data. He presents no description of methodology employed to measure active agents in the blood. How are these values affected by other variables, such as how recently these patients have been on other chemotherapy? How many other chemotherapy agents have they had? Is their liver and renal function normal? In the absence of controls, Dr. Burzynski is constructing his controls from memory and experience, which eliminates any possibility of determining a true response rate.

One huge problem that was noted is that Dr. Burzynski’s therapy requires the adminstration of so much antineoplaston as sodium salts that several of his patients developed hypernatremia, in one case as high as 180 mEq/L. (A normal serum sodium level ranges between 135 and 145 mEq/L.) Personally, I’ve never seen a sodium level that high in a living patient. When sodium levels get into the 155 mEq/L and up range, clinicians start to get very worried and usually start aggressive treatment to bring the sodium levels down. Worse, these are patients with brain cancer. One danger is that, in correcting the hypernatremia, sometimes cerebral edema (brain swelling) will result. That’s why we generally try to correct hyponatremia fairly slowly. In a patient with a brain tumor, cerebral edema could be even more dangerous than in a patient without such a tumor. Despite reviewers being alarmed at the hypernatremia some of Burzynski’s patients developed, astoundingly he still claimed that he had no “significant toxicity.”

The bottom line is that Dr. Burzynski is not a miracle worker. He is not a doctor who sees something that mainstream science has not and who therefore has a cure for many cancers that mainstream medicine scoffs at. He is not a bold visionary. Rather, he appears to be a man pursuing pseudoscience. The reason that mainstream scientific medicine has not accepted the existence of antineoplastons or their efficacy against cancer is because there is no credible evidence to support this thrapy and no one other than Dr. Burzynski has been able to replicate his results.

All of this is why I’m even more sad for Rene Louis than I would be otherwise. She has been taken in, wasting her remaining time on earth pursuing $14,000 to pay Dr. Burzynski to treat her, not knowing that his treatment has never been shown in a rigorous clincial study to be efficacious against cancer. Moreover, she probably doesn’t realize that this would likely be just the beginning, a down payment if you will. Yet here she is, “begging for money,” as she put it, and doing it for nothing. As hard as it is to say, she would almost certainly be better served pursuing palliative therapy and spending as much time as she can with her families. I don’t say this out of coldness or lack of sympathy. I say it because it’s true, as hard as it is to swallow.

People can be generous, incredibly generous. They want to help. It’s bad enough that a crappy insurance or lack of insurance lead people into the humiliating and desperate place of having to beg for money to save their lives by paying for science-based medical therapy. It’s even worse when they beg for money for woo. Not only does it put them in the position of relying on the generosity of strangers, but they unknowingly betray that generosity. Donors want to help. They want their donation to make a difference by paying for treatment that will make a difference. Paying for homeopathy or antineoplastons for someone is not doing them any favors; all accomplishes is to enrich practitioners of dubious (at best) medicine.