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Stanislaw Burzynski: Four Decades of an Unproven Cancer Cure

Article

David H. Gorski

Volume 38.2, March/April 2014

The Houston doctor Stanislaw Burzynski has been using an unproven cancer cure, “antineoplastons,” for decades, but despite its lack of proven anticancer activity, he has still not been shut down. Here is a primer for skeptics on his career and claims.

About a year ago, I received an unexpected email from a film producer named Eric Merola asking me if I would appear in his upcoming movie about Stanislaw Burzynski, MD, PhD. Burzynski is controversial, to put it mildly. Since the founding of the Burzynski Clinic in 1977, he has claimed near-miraculous results treating patients with advanced malignancies, particularly deadly brain cancers like glioblastomas. Merola, who had previously released a film in 2010 praising Burzynski as a scientist with a cure for many cancers who is persecuted by the authorities, explained that he wanted a critic of Burzynski in his new movie. Given Merola’s history of deceptive filmmaking, I politely declined.

Burzynski: Cancer is Serious Business Part II movie poster

When his second movie, Burzynski: Cancer Is A Serious Business, Part 2 was released in June 2013, my decision was validated, because the movie turned out to be every bit the propaganda piece for Burzynski that I had feared, a true sequel (Gorski 2013a). Merola’s movie also continued a pattern that had begun in 2011 of allies of the Burzynski Clinic attacking critics, in this case portraying skeptics as heartless Big Pharma shills harassing patients with terminal cancer.

Unfortunately, as propaganda, Merola’s movie was sufficiently compelling that the leader of a large skeptical group in southern California who attended a screening in March stood up at the Q&A afterward to say that he was persuaded that Burzynski was on to something (Gorski 2013b). Although he quickly reversed himself and admitted that he had made an enormous mistake (Gleason 2013), the damage had been done, and this skeptic’s endorsement can still be found on YouTube (Merola 2013). Given the harm Burzynski has done for four decades and how little most skeptics know about him, Bob Blaskiewicz, who wrote a companion piece to this article about his skeptical activism regarding the Burzynski Clinic, and I decided that a primer for skeptics about Stanislaw Burzynski was long overdue.

Stanislaw Burzynski: The Early Years

Although little is known about Stanislaw Burzynski’s childhood and youth aside from what he himself has told sympathetic sources like his longtime lawyer Richard A. Jaffe (Jaffe 2008) and columnist Thomas Elias (2009), in many ways he represents a classic immigrant rags-to-riches story. Born in Nazi-occupied Poland in the city of Lublin on January 23, 1943, as the Holocaust in Poland was entering its deadliest phase, Stanislaw Burzynski was mostly sheltered from the grim reality of Nazi-occupied Poland during his earliest years because of his mother’s wealth. After the war, when Burzynski was five, Stanislaw’s older brother Zygmunt was killed fighting the newly installed Communist regime. In his book, Elias quoted him invoking his brother thusly, “The idea of fighting people in authority became natural to me. I learned that you must never let them defeat you in your own core.” This sort of determination could have been an admirable trait—if only Burzynski had found a worthy cause to serve.

Unfortunately, the cause he found was antineoplastons.

Burzynski and Antineoplastons(Jerzy Dabrowski/ZUMAPRESS.com)

Antineoplastons

The first use of the word antineoplastons (ANPs, derived from “neoplasm,” or cancer) in a PubMed-indexed article occurred in 1976 (Burzynski 1976), but Burzynski claims that he had thought of the concept a decade earlier as a medical student at the Medical Academy at Lublin. There, the young Burzynski had become intensely fascinated by amino acids and peptides in wild mushrooms and studied uses for them in agriculture. His work was productive—impressive, even—for a medical student, with six scientific papers indexed in PubMed. In medical school, Burzynski studied differences in peptides and amino acids found in the blood and urine of renal failure patients, claiming that cancer patients had a lower level of some of these substances. In 1968, his work in this area resulted in a thesis titled Investigations on Amino Acids and Peptides in Blood Serum of Healthy People and Patients with Chronic Renal Insufficiency (Elias 2009; Green 2001; Smith 1992). By 1970, as a promising young research physician Burzynski was being recruited to join the Communist Party but obstinately refused and soon learned that as a result he would be drafted into the Polish Army. Not wanting to end his research, he fled Poland and arrived at JFK Airport, as he delights in recounting, “with only $20” in his pocket.

After staying briefly with an uncle, Burzynski soon obtained a research position in the Department of Anesthesia at the Baylor College of Medicine in a laboratory headed by Georges Ungar, a Hungarian refugee with whom he immediately hit it off. Ungar was famous at the time for proposing that memory resided in peptides in the brain and for his experiments to “transfer” memory by transferring the putative “memory” peptides from one mouse brain to another. It was a hypothesis that seemed to be supported by his experiments but soon faded from favor (Setlow 1997). At Baylor, Burzynski split his time between working on Ungar’s projects and studying his antineoplastons (Elias 2009, Smith 1992). He appeared to be well on his way to becoming a successful cancer researcher, securing an NIH grant in 1974 (Smith 1992, Burzynski 2012) and publishing several peer-reviewed papers.

So where did everything go wrong? How did this promising young Polish researcher evolve into the dangerous “brave maverick doctor” we know today? To answer that question requires a discussion of ANPs.

Do ANPs Work?

After nearly forty years, it is still not entirely clear exactly what Burzynski originally isolated, but it is clear that antineoplastons almost certainly do not have significant anticancer activity. Excellent detailed summaries of the state of the evidence have been provided by Saul Green (2001; 1992) and, more recently, on the American Cancer Society (2012) and National Cancer Institute (2013a; 2013b) websites. In brief, based on his hypothesis that a naturally occurring biochemical system in the body, distinct from the immune system, could “correct” cancer cells by means of “special chemicals that reprogram misdirected cells,” Burzynski used gel filtration to separate blood and urine fractions and test them in cell culture for anticancer activity. Of his original thirty-nine fractions, today Burzynski treats patients mainly with AS-2.1 (also known as Astugenal or Fengenal) and A-10 (also known as Atengenal or Cengenal). As Saul Green (2001; 1992) and others (Antineoplaston Anomaly 1998) have reported, AS-2.1 is the sodium salt of phenylacetic acid (PA), a potentially toxic chemical produced by normal metabolism and detoxified in the liver to phenylacetylglutamine (PAG). To boil ANP chemistry down to its essence, AS-2.1 is primarily a mixture of PA and PAG, and AS-10 is primarily PA. Of note, PA had been studied as a potential anticancer agent years before Burzynski discovered it (Sandler and Close 1959) and, although it has been studied intermittently for fifty years, it has shown little promise against brain tumors (Chang et al. 1999).

Consistent with what is known, the National Cancer Institute (NCI) characterized the concentrations of ANPs required to show antitumor effects in cell culture or animal studies as “excessively high” and reflecting a “lack of activity” (NCI 2013a), concluding very generously that the evidence that ANPs have significant anticancer activity is “inconclusive.” In 1999 the Mayo Clinic published a phase 2 clinical trial of ANPs versus recurrent glioma (Buckner et al. 1999). Other investigators have had difficulty replicating Burzynski’s results, including the NCI, Sigma-Tau Pharmaceuticals, and the Japanese National Cancer Institute (Green 2001; 1992). The one exception is Hideaki Tsuda, a Japanese anesthesiologist at Kurume University, who claims to have observed remarkable results in a randomized clinical trial adding ANPs to chemotherapy infused directly into the hepatic artery to treat liver metastases from colorectal cancer. Indeed, Dr. Tsuda appeared in the most recent Burzynski documentary touting impressive results from this clinical trial. Unfortunately, at this writing, these results remain unpublished, and Tsuda’s previously published ANP work is not impressive. Amusingly, Eric Merola sent out a complaint on social media lamenting that the Lancet Oncology rejected Dr. Tsuda’s manuscript, ascribing the rejection to a Big Pharma conspiracy to suppress ANPs (Gorski 2013c).

Over the last decade, Burzynski appears to have given up trying to appear scientific and has not published in anything resembling a reputable journal for a long time. A PubMed search reveals no primary scientific reports since 2006, and the only clinical trials he has published were preliminary results of two phase 2 trials ten years ago (Burzynski et al. 2003; 2004) or retrospective. In short, Burzynski’s science has failed to progress since the late 1970s. If Burzynski’s science is stagnant now, how did it reach this stage? The answer to this question began nearly thirty-eight years ago.

1976: The Descent from Science Begins

In 1976, Burzynski deemed antineoplastons ready to be tested against cancer in a clinical trial. Characteristically, despite having had no formal training in oncology and no experience in clinical trial design, Burzynski judged himself to be uniquely qualified to be principal investigator of such a trial. Unfortunately for him, internal politics at Baylor had led to Ungar’s ouster from the Department of Anesthesia, and the new chair, not unreasonably, did not view Burzynski’s research as appropriate for a department of anesthesia. If Burzynski is to be believed, the director of Baylor’s new cancer research center wanted to hire him, as did Ungar at his new job. However, Baylor wanted Burzynski to sign away rights to his discoveries (a standard condition in academia), and Burzynski did not want to follow Ungar to Knoxville because he was afraid that the University of Tennessee would impose the same condition. There was another condition that rankled him as well. Shortly after he had obtained his Texas medical license in 1973, Burzynski started working part time at a private practice. There, he had apparently administered antineoplastons to cancer patients, the result being a twenty-one patient case series published in 1977 (Burzynski et al. 1977), implying that he had likely been treating patients with ANPs as early as 1975. Baylor’s additional requirement was that Burzynski give up his private practice—a deal breaker, because, as Elias describes, “As long as he [Burzynski] had a private practice, he believed he could use whatever medications he thought most effective, subject only to the consent of his patients.” This speaks volumes about Burzynski’s attitude toward scientific medicine and ethics, an attitude that appears not to have changed appreciably since then.

In late 1976, Burzynski applied to the Baylor Institutional Review Board (IRB), the ethics committee that approves and reviews human subjects research, to begin a clinical trial of ANPs. He was turned down. Both Elias and Jaffe claim that the reason was because Burzynski didn’t have an “investigational new drug” application (IND), which the FDA requires before it will approve a clinical trial of an experimental drug, an explanation that rings false because in general it is not necessary to have IRB approval before applying for an IND (US FDA 2013a). In fact, it is not surprising that the Baylor IRB balked. In 1977 there almost certainly were not sufficient preclinical data to justify a clinical trial. It wasn’t even clear yet exactly what ANPs were, as Burzynski hadn’t yet identified all their constituents, and institutional review boards are very reluctant to approve a clinical trial involving compounds that are incompletely characterized. Undeterred, Burzynski shopped his protocol around to other hospitals. Ultimately, the IRB at Twelve Oaks Hospital approved his application. Jaffe’s account of this time period (Jaffe 2008) illustrates the incipient ethical slide into oblivion. For example, before leaving Baylor, Burzynski had lawyers investigate the legality of treating patients with ANPs. Their advice to him was that, because Texas didn’t have a “mini-FDA” act, in which only FDA-approved drugs could be administered to patients, treating patients with ANPs was legal under Texas law at the time, as long as the ANPs were not shipped across state lines (Merola 2010).

In the late 1970s, Burzynski went to great lengths to obtain the raw materials necessary for his work, given that before he figured out how to synthesize antineoplastons chemically in 1980, isolating ANPs required thirty liters of urine per day per patient. The difficulty in obtaining such huge quantities of “raw materials” can only be imagined, but ANPs could also be isolated from blood. Amusingly, before he left Baylor, Burzynski was notorious for appearing at social functions with blood collection supplies and begging, wheedling, and cajoling friends and acquaintances to donate blood from which he could isolate ANPs. Jaffe drolly noted that after a while Burzynski “noticed he was getting fewer and fewer invitations to parties, and, when his friends would see him on campus or the street, they would turn and walk away quickly, pretending they didn’t see him.” After Burzynski opened his clinic in 1977, huge quantities of urine were required as raw material to isolate ANPs. To get it, Burzynski arranged to install urine collectors in public parks and even the state penitentiary system. He even collected urine from Gilley’s Bar, where Urban Cowboy was filmed. Perhaps John Travolta himself contributed to some of those early ANP batches.

Abuse of the Clinical Trial Process

One of the most common claims made by Burzynski and his supporters is that he must be on to something because the FDA keeps letting him register phase 2 clinical trials and even let him register a phase 3 clinical trial in 2010. Phase 2 trials are small preliminary clinical trials, sometimes not randomized, designed to identify indications of efficacy. They lay the groundwork for phase 3 trials, which are the large randomized clinical trials that ultimately result in drug approval by the FDA. To date, although Burzynski has published occasional case studies and partial results of two phase 2 trials, he has not published the complete results of any of his phase 2 trials. Of the sixty-one clinical trials registered on ClinicalTrials.gov with Burzynski as the principal investigator, only one has been completed, but it has not been published. Of the remaining sixty trials, the statuses of fifty are unknown; seven were withdrawn; two have been terminated; and one has not yet been opened to accrual (ClinicalTrials.gov 2013), and the phase 3 trial has never accrued a single patient. Merola promised in his movie that Burzynski’s phase 2 results will be published “soon,” and others claim that Burzynski is preparing at least a dozen manuscripts for publication. However, Burzynski has been promising to publish for years and has not produced anything substantive. This failure to publish is not surprising given the origin of these trials, as we will soon see.

From the late 1970s to 1998, Burzynski was under nearly constant investigation by medical authorities, beginning with the Harris County Medical Society in 1979 (Jaffe 2008; Elias 2009; Null 1979) and continuing with the Texas Medical Board and the FDA. Indeed, the Texas Medical Board has tried to strip Burzynski of his license to practice at least twice, failing to do so in 1993 (Jaffe 2008; Elias 2009) and most recently in 2012 (Gorski 2012). However, it was the prosecution brought against Burzynski by the FDA during the 1990s that spawned the oft-touted “six dozen” clinical trials. Here’s how it happened. In the fall of 1995, a grand jury indicted Burzynski for seventy-five counts of insurance fraud and violations of the Food, Drug, and Cosmetics Act. As part of this process, Judge Simeon Lake of the U.S. District Court for the Southern District of Texas, ruled that Burzynski’s “continued pretrial release” was contingent upon his administering his drugs exclusively through FDA-approved clinical trials (Antineoplaston Anomaly 1998).

By this time, however, Burzynski had cultivated powerful allies, in particular Representative Joe Barton (R-Texas), who held a series of hearings featuring cancer patients who were, quite understandably, terrified that Burzynski would be convicted. (Remember, these patients were completely convinced that Burzynski was the only person who could save them.) Between the cynical political theater, featuring weeping parents of children with brain tumors, national press stories of demonstrations featuring patients chanting “FDA go away! Let me live another day!” and the intense political pressure brought to bear by Barton, who dragged then-FDA Director David Kessler in front of his committee four times over two years to explain why the FDA was “harassing” Burzynski, the FDA ultimately relented and entered negotiations to let Burzynski set up clinical trials. Taking advantage of the ruling and the political pressure on the FDA, Burzynski and Jaffe decided, in essence, “If the judge wants clinical trials, we’ll give him clinical trials.” So that’s just what they did.

Prosecutors pleaded with the FDA not to give in because it would undermine their case, but the FDA overruled them. First, patients already being treated were enrolled in a wastebasket trial known as “CAN-1” (Jaffe 2008; Antineoplaston Anomaly 1998), a retrospective trial looking at all patients then being treated at the Burzynski Clinic. Of this trial, Jaffe (2008) wrote:

. . . As far as clinical trials go, it [CAN-1] was a joke. Clinical trials are supposed to be designed to test the safety or efficacy of a drug for a disease. It is almost always the case that clinical trials treat one disease.

The CAN-1 protocol had almost two hundred patients in it and there were at least a dozen different types of cancers being treated. And since all the patients were already on treatment, there could not be any possibility of meaningful data coming out of the so-called clinical trial. It was all an artifice, a vehicle we and the FDA created to legally give the patients Burzynski’s treatment. The FDA wanted all of Burzynski’s patients to be on an IND, so that’s what we did.

The FDA also permitted Burzynski to set up nearly identical phase 2 trials for every cancer that he wanted to treat. Burzynski claimed these were based on a protocol used in a trial done by the National Cancer Institute in the early 1990s when the NCI had tried to work with Burzynski. (This effort failed because of strife between the NCI and Burzynski, who viewed the NCI as trying to sabotage the trial (Smith 1992). These trials had but one purpose, to allow Burzynski to continue treating patients with ANPs (Antineoplaston Anomaly 1998), as Jaffe himself boasted (2008):

CAN-1 allowed Burzynski to treat all his existing patients. That solved the patients’ problems, but not the clinic’s. A cancer clinic cannot survive on existing patients. It needs a constant flow of new patients. So in addition to getting the CAN-1 trial approved, we had to make sure Burzynski could treat new patients. Mindful that he would likely only get one chance to get them approved, Burzynski personally put together seventy-two protocols to treat every type of cancer the clinic had treated and everything Burzynski wanted to treat in the future.

The prosecution thus undermined, the first trial ended in a hung jury in 1997, and a second trial on a subset of the original charges resulted in Burzynski’s acquittal. Since then, Burzynski has practiced (mostly) untroubled by the law, other than intermittent FDA inspections and warning letters. Investigations by the FDA in the 2000s resulted in reports citing Burzynski for failure to report adverse events and to follow proper informed consent procedures and a warning letter (US FDA 2009) citing the Burzynski Research Institute (BRI) IRB for deficiencies such as failing to conduct continuing reviews, approving research without determining whether the risks were reasonable compared to potential benefits, and conflict of interest of IRB members. For example, its chair is an old Burzynski crony from Baylor and the current chair of the board of directors of the BRI, Carlton F. Hazlewood.

Most recently, in response to what is rumored on patient blogs to have been the death of a patient treated with ANPs in 2012, the FDA issued a partial clinical hold on antineoplastons for children, meaning that no new children could be enrolled in Burzynski’s clinical trials; the FDA then extended the hold to adults. The identity of this child was established in a recent investigative article in USA Today to be Josia Cotto (Szabo 2013a). This same article also reported that from January to March 2013, the FDA investigated the Burzynski Clinic. Based on its report (FDA Form 483 2013), the FDA issued a warning letter to the Burzynski Clinic, citing its IRB for, among other violations, enrolling patients in clinical trials without determining that risks to subjects were minimized and were reasonable in relation to anticipated benefits, inappropriately using the expedited review process to treat subjects on single patient protocols, misinterpreting MRI scans to overestimate response to therapy, and destroying original patient records (Szabo 2013a, US FDA 2013b). Until this most recent clinical hold, none of the FDA investigations had stopped Burzynski from “case management fees” of hundreds of thousands of dollars, even though it is generally considered unethical, except in very narrowly defined cases, to charge patients to participate in a clinical trial. It is, however, not illegal.

Dr. Stanislaw Burzynski and a patient with a signDr. Stanislaw Burzynski stands with one of his patients outside a courthouse during a demonstration in his support. (Jerzy Dabrowski/ZUMAPRESS.com)

Enthusiastic support for Burzynski among the alternative cancer cure subculture is all the more puzzling given that what he is really doing is administering unproven chemotherapy at very high doses. This ANP chemotherapy is not without side effects, contrary to claims otherwise. In addition to causing rashes, fevers, and other side effects, ANPs contain so much sodium that they can cause life-threatening elevations of sodium in the blood. Indeed, Josia Cotto died of hypernatremia, with USA Today reporting that he was recorded as having a serum sodium of 205 mEq/L, well into the lethal range (normal is between 135 and 145 mEq/L). Consistent with this, in a June 2013 report on the BBC series Panorama, the chief of the pediatric intensive care unit at nearby Texas Children’s Hospital related her experiences taking care of patients from the Burzynski Clinic suffering life-threatening toxicity from ANP treatment. In the same report, a parent who took her daughter to Texas Children’s Hospital after she had suffered such toxicity reported that the Burzynski Clinic had a very bad reputation there because of the frequency with which they had to care for critically ill and dying Burzynski patients. Despite all the criticism and recent revelations, Burzynski remains combative, referring to his critics as “hooligans” and “hired assassins,” while describing the patients who complain about him thusly: “We see patients from various walks of life. We see great people. We see crooks. We have prostitutes. We have thieves. We have mafia bosses. We have Secret Service agents. Many people are coming to us, OK? Not all of them are the greatest people in the world. And many of them would like to get money from us. They pretend they got sick and they would like to extort money from us” (Szabo 2013a).

Less than a month after the USA Today report, the FDA issued two more warning letters, citing serious violations, including losing patient records, misclassifying tumor responses, failing to report serious adverse reactions, and advertising antineoplastons as safe and effective even though they were unapproved. The FDA even noted that the medical records of Josia Cotto provided to the FDA Division of Medical Products did not match the medical records that the FDA directors on site had examined (Szabo 2013b).

At the close of 2013, Burzynski’s allies were replaying their 1990s strategy by recruiting patients with brain tumors to lobby their legislators and persuade others to do the same, patients such as Liza Covad, the wife of Sammy Hagar’s drummer; McKenzie Lowe, a girl with a brainstem glioma; and Elisha Cohen, a Houston area boy with a brainstem glioma whose plight has rallied the Jewish community, both here and in Israel, to donate to his cause and write to their elected officials to pressure the FDA to allow them to receive ANPs under a compassionate use protocol.

As 2014 dawned, Burzynski had enlisted the Alliance for Natural Health USA, which duly published “action alerts” smearing USA Today and Liz Szabo as in the thrall of pharmaceutical company advertising lucre, insinuating wrongdoing, and trying to rally support to Burzynski patients trying to obtain compassionate use exemptions. Meanwhile, it looks as though the Texas Medical Board will be taking yet another crack at Burzynski in 2014, having filed a complaint in December 2013 charging Burzynski with advertising drugs that are not FDA-approved.

But What about the ‘Miracles’?

Patients are drawn to the Burzynski Clinic by reports of “miracle cures,” and over the years Burzynski has specialized in treating unresectable brain tumors. Indeed, the Burzynski Patient Group, created in the 1990s, features a website chock full of testimonials of patients with “incurable” cancer who are alive today. Burzynski and his ANPs are, of course, touted as the reason. There can be several reasons why these testimonials are not convincing evidence that antineoplastons cured these patients. For example, many of these patients have had conventional surgery, radiation, and chemotherapy, and it was the conventional therapy that eliminated the tumor. Also, contrary to popular belief, there are occasional survivors of brain cancer. In some cases, it is not clear whether the patient actually had cancer in the first place. In still others, patients have died, but their deaths are not as well known as their testimonials. One famous example occurred in 1988, when television talk show host Sally Jesse Raphael featured four Burzynski “miracle” patients, who had incurable cancer and failed conventional therapies but claimed that Burzynski had rendered them cancer-free. Four years later, Inside Edition followed up on these four patients and found that two of the four had died and a third had recurred, while the fourth had originally had a good prognosis. A more recent Burzynski failure is Christina Lanzoni, who was the sister of model and actor Fabio Lanzoni. At her brother’s urging, she sought care at the Burzynski Clinic for advanced ovarian cancer and died in September 2013. Fabio himself has appeared in YouTube videos extolling Burzynski as a “medical genius.”

Finally, one potential explanation for some of these seemingly miraculous responses to ANP therapy in brain cancers could come from a phenomenon known as pseudoprogression in which late effects of radiation therapy can produce enhancing lesions that mimic tumor recurrence on brain MRI (Stuplich 2012) and which can occur as much as 28 percent of the time after radiation therapy (Brandes et al. 2008). Such pseudoprogression “tumors” regress over the course of weeks to months, much as “recurrences” treated by Burzynski almost inevitably regress, and pseudoprogression can even persist as long as a year after radiation therapy (Stuplich 2012). While it must be conceded that it is possible that in some patients ANPs might exhibit antitumor effects, the more plausible and parsimonious explanation is that pseudoprogression likely explains many of Burzynski “miracle cures.”

‘Personalized, Gene-Targeted Cancer Therapy’ and Beyond

Burzynski is currently permitted to administer antineoplastons to existing patients, but until the FDA rules based on its most recent investigation he is not permitted to enroll new patients in ANP clinical trials. Perhaps seeing the end in sight for ANPs, over the last several years Burzynski has been “diversifying,” in particular treating patients with a protocol he refers to as “personalized gene-targeted cancer therapy” (Somers 2009; Gorski 2011). He has even gone so far as to declare himself a “pioneer” in personalized cancer therapy (Burzynski 2012; Somers 2009). Eric Merola, picking up on this, portrayed Burzynski as such a pioneer in targeted therapy that the University of Texas M.D. Anderson Cancer Center has emulated him with its Institute of Personalized Cancer Therapy, a claim so overblown as to defy belief.

What Burzynski really does has little to do with research or cutting edge cancer therapy. In 2011, I investigated what Burzynski’s “personalized gene-targeted therapy” entailed. The spokesperson confirmed what I had learned from patient blogs, namely that he has used a commercial test from Caris Life Sciences, which involves various assays of the patient’s tumor and blood plus a gene expression profile of the tumor, and then generates a report of which cancer-associated genes are made by the tumor. For each gene, where applicable, there is a list of drugs that either target that gene or whose antitumor activity correlates with the presence of that gene. More recently, the Burzynski Clinic touts its involvement with a registry study through Foundation One (FMI-001-NGS-500), a company that markets another gene test consisting of a subset of cancer-associated genes, implying that the Burzynski Clinic uses this company’s products now.

The problem for cancer clinicians is what to do with these results. What Burzynski claims to be able to do is to use this information to pick a combination of treatments that he can administer at low dose and much less toxicity than conventional chemotherapy. Frequently, these agents haven’t been tested together, and the potential for synergistic toxicity is unknown. To apply results like this to patients outside the context of a clinical trial is hard to justify except in rare cases, but that’s exactly what Burzynski has done with large numbers of his patients, picking off-label chemotherapeutic agents based on the results of this test and selling it as “personalized gene-targeted therapy” without letting patients know that (1) the relevance of these recommendations is often debatable; (2) the studies used to support them have a lot of uncertainty; (3) few of these recommendations have yet been validated in clinical trials; and (4) it has not yet been shown that using the Caris test or similar tests to direct therapy results in prolonged survival.

After four decades, Stanislaw Bur­zyn­ski remains an example of a practitioner using unproven cancer “cures” continuously without being shut down for a long period. There is little doubt that Burzynski started out trying to be a real scientist, but something happened in the mid-1970s that led him away from the path of responsible science and medicine. Unfortunately, he remains very good at donning the mantle of science to make it appear as though his therapy represents a reasonable alternative to chemotherapy. Even more amazingly, because of his battles with the FDA and Texas Medical Board, he has become a hero in the alternative cancer world, even though ANPs are toxic chemotherapy and his “gene-targeted” therapy is a cocktail of chemotherapies and very expensive targeted agents combined in untested combinations.

Truly, antineoplastons demonstrate the importance of science-based medicine. If Burzynski had “played by the rules” and methodically taken ANPs through the clinical trial process, he (and we) would have known decades ago whether ANPs have significant anticancer activity in humans. In 2014, we still don’t know for sure, although what we do know strongly suggests that ANPs have little or no anticancer activity. Finally, Burzynski’s story is a cautionary tale of just how ineffectual the medical and government agencies that are supposed to protect the public, such as state medical boards and the FDA, can be. These organizations are supposed to protect the public from practitioners like Burzynski, but all too often they fail at their charges, in this case spectacularly.


References

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More from this issue of Skeptical Inquirer: "Skeptic Activists Fighting for Burzynski’s Cancer Patients" by Robert Blaskiewicz.

David H. Gorski

David H. Gorski's photo

David H. Gorski, MD, PhD, is in the Departments of Surgery and Oncology, Wayne State University School of Medicine, and the Barbara Ann Karmanos Cancer Institute, Detroit, Michigan. E-mail: gorskid@med.wayne.edu.