Chapter Twenty-One

PART FIVE

Physical, Traditional, and Pharmacological Therapies

Chapter Twenty-one

Stanislaw Burzynski–Antineoplastons on the Edge of Medical Credibility

A devoted scientist, Stanislaw Burzynski, M.D., Ph.D, is one of the prominent unconventional pharmacological practitioners of our time, and one who is completely committed to the open scientific evaluation of his therapy. While controversy continues to rage around his work, the promise is substantial that his therapy may be of benefit for some cancer patients.

Burzynski’s work has been described in detail by several authors, most notably Ralph W. Moss in his excellent book The Cancer Industry,1 which provides thorough accounts of many of the prominent figures in alternative cancer therapy. Moss is both a sympathetic investigator of many alternative therapies and a gifted science writer. He also describes in detail the politics surrounding unconventional cancer therapies and has made the most sophisticated statement available for the argument that the cancer establishment systematically suppresses independent scientific research on these therapies.2 In what follows, I draw extensively on Moss’s account, as well as on my visit to Burzynski’s center in Houston in 1982; on a review of original scientific papers; and on my visit to Kurume, Japan, where I met with researchers who believe they have independently confirmed some of Burzynski’s findings.

Burzynski’s Early History

Burzynski was born in Poland in 1943, excelled in chemistry, and graduated first in his class from the Medical Academy of Lublin in 1967. The following year, at 25, he received his Ph.D. in biochemistry, and became one of the youngest recipients of both degrees in memory. He emigrated to the United States in 1970 and became a researcher and assistant professor at Baylor College of Medicine in Houston, Texas.

As a graduate student in Poland he had become intrigued with differential patterns of peptides–small chains of amino acids–in different human illnesses. At Baylor, he continued this work on his own under the supervision of a mentor who was also interested in peptides. He first worked with his own blood, then switched to urine as a peptide source. By 1974, he had isolated 119 peptide fractions in human urine, and published his results with two colleagues in a leading journal of physiological chemistry and physics.3 Moss reports:

Even when he was in Poland, Burzynski had suspected that some of these peptide fractions might have activity against cancer. The blood of one of the prostate patients had proved almost entirely lacking in one of the three faint peptide streaks. …

Writing about Burzynski’s early work, the DuPont scientists remarked: “They found some peptides could produce up to 97 percent inhibition of DNA synthesis and mitosis in the neoplastic cells of their tissue cultures.”

The active peptide fractions consisted of two groups. One was strongly acidic; the other, broad based and slightly acidic or neutral. The strongly acidic group had a very powerful effect on a … number of tumor cell lines, especially osteogenic sarcoma, a kind of bone cancer. But the other kind stopped the growth of many different kinds of cancer cells. With many backward glances, Burzynski decided to focus his attention on this broad-based peptide band. And it was this admittedly ill-defined substance that Burzynski now dubbed “Antineoplaston A.” A new name was needed because these particular urinary peptides had never been described before. The name was derived from the Greek–neoplasm being a medical term for “new growth” or cancer. All subsequent forms of Antineoplastons derived from this substance.4

In a letter to me in November 1990, Burzynski gave a brief description of antineoplastons:

I always considered Antineoplastons as components of the biochemical defense system in the body, which constitutes a repair system for misprogrammed cells. In my early work at Baylor before the NCI [National Cancer Institute] grant was awarded, we were screening peptides isolated from blood and urine for various important biological effects. … [I found it] necessary to concentrate heavily on cancer and the defense system in the body which parallels the immune system [see figure 21.1]. … This is the system which protects us from “the enemy within,” contrary to the immune system which gives us the defense against invaders. Because of that, this is mostly a repair system, not aimed at killing misprogrammed cells. The errors in cell programming may lead to such diverse groups of disorders as cancer, benign tumors, certain skin diseases, AIDS, and Parkinson’s disease [see figure 21.2]. … In each of these disorders, I have a theory how this system should work, as well as preliminary laboratory data. … We also have preliminary clinical experience in which we document objective responses in AIDS, Parkinson’s disease, benign tumors, psoriasis and stimulation of wound healing … These patients came to us initially with cancer diagnoses, but at the same time, they were [also] suffering from these various disorders.5

The Turning Point–Burzynski Refuses to Do Research in the Medical Establishment

Max Gerson’s fall from grace in the mainstream medical community did not occur until after he testified at a congressional hearing at which his cancer recoveries were touted as “miracles” and his colleagues recommended that Congress provide major funding for research into approaches similar to his but that Congress should avoid allowing existing medical organizations (a clear reference to the American Medical Association) to dominate the new inquiry. It was this direct affront to the authority of organized medicine that brought on the legal and professional harassment that destroyed Gerson’s career. In Burzynski’s history there is a parallel, although his challenge to the medical establishment was not as direct and his capacity to survive the subsequent harassment proved greater.

Moss reports that Burzynski’s peptide discoveries were at first enthusiastically welcomed by fellow scientists and the media. He was invited to join the faculty of the department of pharmacology at Baylor, which would mean his leaving his position in the department of anesthesiology, where he had enjoyed complete freedom to conduct his research but where his work was obviously not closely connected to the department’s basic mission. In a critical decision, Burzynski refused the offer to join the department of pharmacology. As Moss reports:

It looked like a promotion. There was one condition, however: he had to give up his budding private practice. Others might have grabbed at the chance and been happily absorbed into the cancer mainstream. There were, after all, ample rewards for doing so. But Burzynski hesitated. … Deep in his marrow, he feared institutionalization.6

This was, like Gerson’s challenge to organized medicine, a critical turning point in Burzynski’s career. Burzynski, a central European immigrant like Gerson, was being welcomed into the mainstream research establishment precisely because of his exciting findings regarding antineoplastons. A man with the talents Burzynski was later to demonstrate–in his awesome personal effort to build a cancer research program entirely on his own–might have been expected to thrive in the intensely political atmosphere of large-scale cancer research, where political acumen is often as important to success as research brilliance and scientific good fortune. And yet, for better or worse, Burzynski refused the offer, despite clear warnings from his superiors of the likely political consequences. He later told Moss:

“Most medical breakthroughs,” [Burzynski said] with a sly touch of irony, “have happened because there was some lack of suppression by the supervisors of people doing some innovative work.”… In addition, his private practice gave him financial independence. “If I should join them,” he said, “I would do exactly what they were telling me to do, even though I would have a separate lab.” When he refused their offer, these well-established cancer researchers turned against him and began to make life difficult. … Although he received an impressive certificate for meritorious service … his Chairman’s last words were not auspicious: “Just wait, Burzynski. They’re going to kick your ass.”7

Because this was such a turning point, I asked Burzynski to review the reasons why he made this decision. Burzynski said that he was enjoying an ideal situation in the department of anesthesiology with a larger laboratory space than he would have had in the department of pharmacology; he had applied for a new $30,000 grant to continue his research; and, there was, he hoped, the prospect that he could develop a semiautonomous research unit loosely affiliated with the department of anesthesiology. By contrast, he said the reputation of the department of pharmacology was that it was autocratically run. He feared he would be unable to pursue his research. Burzynski says that at the time he refused the department of pharmacology offer he had no intention of leaving Baylor, and he expected to stay in his position with the department of anesthesiology. At a minimum, he misjudged the politics of the university. The situation “deteriorated,” he said; his grant was not funded and he had to leave.

Reviewing his decision to leave Baylor, Burzynski offered his perspective:

Regarding the issue of remaining at Baylor, I still believe that this would have led to disaster. One of the misfortunes of this country is that there is a big gap between basic scientists and clinicians. Contrary to the European situation, basic science researchers are despised by M.D.’s, and practicing physicians in medical schools are hated by basic researchers. A number of breakthroughs in medicine were done through the combination of basic science and clinical research in the same person. Well known examples are Louis Pasteur and Jonas Salk. When I initially came to the United States, I did not have any intention of practicing clinical medicine. I dreamed of being involved just in pure basic science. My enthusiasm quickly vanished after I noticed how biochemists are treated by clinicians. If I had stayed at Baylor after 1977, I would have had to rely on the mercy of proud clinicians using the same approach to Antineoplastons as to conventional chemotherapy. Such an approach would never have worked and the whole project would have been destroyed after the first unsuccessful clinical trial. I must admit, on the other hand, that it was my error to take “American democracy” too seriously. Coming from Poland, where everybody has a saintly opinion of the United States, I was convinced that this would be the country where democracy is everywhere. This may be true, but ultimately you have to prove this in court.8

Burzynski Attacked by the Medical Establishment

Moss describes in detail the extraordinary story–which will become scientific legend if antineoplastons ultimately prove to be a significant scientific discovery and the basis for an anticancer therapy–of how Burzynski set out virtually alone to develop, test, and win FDA (Food and Drug Administration) approval for a new anticancer drug–an effort that takes major pharmaceutical companies in good standing a decade and costs up to $100 million.9 Burzynski undertook to do this with the cancer establishment pitted against him and with total assets of only $5,000. Historically, it is important to recognize the undeniable reality that he could have gone the other way, avoiding all of the massive opposition he has subsequently encountered. Whether the internal obstacles that the cancer establishment places before its own researchers would have proved greater than those Burzynski was to face as a maverick outsider, history cannot tell us. My own instinct–based on the strength of the scientific evidence that antineoplastons have promise and the entrepre-neurial skills that Burzynski has subsequently demonstrated–is that he could have done better inside the American scientific establishment than outside it. Burzynski’s assessment was that he would have been trapped by the bureaucratic and autocratic characteristics of the department and left without the freedom to pursue his research.

Having been forced out of Baylor, Burzynski faced a double-bind. Antineoplastons are largely species-specific. So, as Moss points out:

Thus he was caught in a classic catch-22 situation. If he tested Antineoplastons in humans, the FDA was sure to come down on him eventually. But if he didn’t so test them, he could never win FDA approval, since Antineoplastons, being species-specific, are not generally effective in animal treatment experiments [which the FDA was then using as a screen before authorizing human experiments]. His decision was thus to start treating patients, build up good records, let patient fees finance the future development of the drugs, and deal with the FDA later.10

Moss also notes that, “Financial considerations could not help but play their part. It escaped no one’s notice that an effective treatment, locked up in patents, would be worth a fortune” [emphasis added].11

Had Burzynski gone inside the cancer establishment, on the other hand, I believe that there is a high likelihood that he could have found his way past the obstacle of the animal screen that FDA uses for anticancer drugs and made his way into human trials more quickly than he was able to on the outside. Once he had persuaded the FDA to allow him to conduct clinical trials, then by the curious logic of American cancer research antineoplastons would have been a legitimate “experimental cancer therapy” instead of a dangerous unproven “alternative therapy” precisely because it was being conducted under the auspices of a recognized scientific institution instead of under the auspices of an independent center that had challenged the establishment. He would have had the power to move the FDA around the problem of an ineffective animal screen precisely because he was developing a patentable therapy in conjunction with a major pharmaceutical house and a major university cancer center. All three players would have stood to benefit financially from his success. In case the FDA slowed clinical trials, he would also have been better placed to have antineoplastons tested in humans outside the United States because of his standing in the medical establishment and because of the standing and clout of his pharmaceutical industry partner.

Having made his decision, Burzynski proceeded to experience, as his Baylor department chairman had predicted he would, the full-scale legal and regulatory terror of county, state, and national authorities. He was investigated by the Board of Ethics of the Harris County Medical Society on the charge of using unapproved medications; he was refused research money by mainstream funders who had previously funded him.12 Subsequently, he was to have his offices raided by the FDA, which seized 200,000 medical files and documents, and he was placed on the American Cancer Society’s “unproven methods” list.13 He was sued by an insurance company and investigated by a Federal grand jury.

Despite Burzynski’s fall from grace, the NCI agreed to test three of his antineoplastons in its standard drug screen. Moss reports:

Burzynski cooperated but was dubious. There were several test-tube and preventative models in which the drug had showed effectiveness, but the standard NCI pre-screen, P388 mouse leukemia, was not one of them. … As he had predicted, these compounds were not effective against mouse leukemia. Burzynski reasonably suggested that NCI try the Antineoplastons in cell-culture assays that were similar to “human solid tumors, especially adenocarcinoma of the breast” …

Burzynski was hardly alone in voicing doubts about the P388 mouse model. Ironically, it was Dr. John Vendetti himself, chief of the NCI’s Drug Evaluation Branch, who in 1983 coauthored an article in which he argued the limitations of this very mouse system. Scientists at NCI had found, for instance, that of seventy-nine drugs which had previously been judged negative in the P388, fourteen showed “significant activity” when retested in cell culture assays.14

Evaluating Antineoplaston Therapy

In 1982 a Canadian oncologist, David Walde, M.D., visited Burzynski in Texas. Moss quotes Walde’s account:

“I had no idea what to expect upon my arrival there and would not have been surprised to find a backdoor operation directed to the exploitation of patients for financial gain without the benefits of any therapeutic activity of the program. I also thought that documentation of the clinical cases would be poor and incomplete, making evaluation difficult if not impossible.”

What he found “was beyond my wildest expectations, and I had to rapidly backtrack on all my preconceived concepts of the situation.”

Moss continues:

Walde first visited the new antineoplaston production plant that Tad Burzynski [Stanislaw’s brother] was busy assembling in a block-long building in nearby Stafford. The manufacturing guidelines were those suggested by the FDA and the entire operation–with its highly sophisticated, computerized control panels, its safety codes, and its sterile precautions–was the same as one might find in any modern pharmaceutical plant.

“It was beyond my conception that an individual, without massive cash-flow funding from either commercial or governmental sources, would be able to single-handedly put together this sophisticated production capability. My impression was that the entire program, both research and production, was built on the financial backs of the patients, supplemented by large personal bank loans by Dr. B” [emphasis added].

Walde’s conclusion [Moss said] was that urinary peptides were an extremely promising area of cancer research.15

Stimulated by Moss’s citation from the Walde report, I looked up the original and found it to be a model of the kind of balanced effort at evaluating an unconventional therapy that is so rarely found in the literature. Walde did say the positive things that Moss quoted, but he also said some other revealing things. For example:

Dr. Burzynski does not and never has claimed he has the cure for all cancer. He never made any such claim to me. He tends to be readily and enthusiastically carried away about the possible theoretical extensions of his work, the ultimate of which hopefully being the use of peptides in not only cancer therapy, but in a variety of biological modulations. He can easily be misinterpreted as representing that he has the “cure” [emphasis added].

He was very frank in his discussions. With malignancies such as ovarian, oat cell bronchogenic carcinoma and acute leukemias he has had dismal results. He cannot explain why hepatic and bony metastases of breast carcinoma seem to show a response, yet pulmonary and soft tissue disease often progresses even while the former regress, a situation contrary to standard chemotherapy, raising of course the later possibility of combined therapy once the role, if any, of peptides in cancer therapy is established.

The cancer processes that seem to respond best to chemotherapy show little response with his peptides. A good example is Hodgkin’s, yet dramatic responses in some cases have been obtained with non-Hodgkin’s lymphomas. The response data is far from complete, and preliminary. …

I had a chance to review a number of his charts, about 60 in all. Those that I was shown had excellent documentation. … The degree of response, complete remission, partial remission, stable and progressive disease has been assessed again by standard criteria. Here I was in disagreement with a number of the assessments. Undoubtedly some interesting responses were occurring with a strong suggestion of an alteration in the natural history of the disease [emphasis added]. However, I was only shown a small number of the total case load treated. Many were what I would term “dirty cases”; namely cases treated by some modality other than peptides, leaving open the criticism that the response was due to the prepresentation treatment, rather than peptides.16

Walde also noted that there was “an extremely high patient fallout” due to many factors, including high mortality related to the advanced stage of cancer in many patients; the fact that “the cost of the therapy is prohibitive, except for the very rich”; the fact that some patients become demoralized and leave treatment; the problems raised by assessing the effects of pretreatment; and the short duration of follow-up. Walde said: “One arrives at the situation of a major dilution in the number of assessable cases. The Phase II equivalent trials are therefore totally statistically invalid.”

Walde also recorded telling conversations with Burzynski about Burzynski’s use of the popular media to publicize his cause:

Politically, the situation at the moment over there is bordering on the impossible. I have stressed to Dr. Burzynski that he should avoid using the media to publicize his data. Dr. Burzynski retorts that this has been the only channel left open to him by repeated rejection of his submissions to journals and meetings. … He has to have patients to continue his research, as these fund the ongoing program. Public attention is therefore mandatory to maintain patient flow. This publicity inevitably ends up with the media Fed-bashing or accusations of a cancer-cure cover-up against the cancer societies. … I can appreciate the reasons for the criticisms launched against Dr. Burzynski. He proceeded with patient therapy, not only to investigate his peptides, but also to use the patients as a funding source.17

Yet with all these criticisms, Walde also came away with the powerful positive impressions that Moss cited above. He had looked up and read numerous articles in the historical and modern literature on urine therapy and concluded:

The whole concept of peptides in the modification of biological processes has been firmly established. … There is now enough data to necessitate us to look more closely at the nature and action of these peptides, as has been advanced by Dr. Burzynski, even if these eventually do not result in any major therapeutic advances” [Walde’s emphasis].17

Whatever the faults of Walde’s analysis, it was a genuine effort at a balanced assessment of an unconventional cancer therapy. Walde was a founding member of the investigatory drug subcommittee of the National Cancer Institute of Canada. He is also the leading Canadian authority on Essiac, a native Canadian alternative cancer therapy. I find him to be a remarkable example of an oncologist with a balanced and open interest in effective assessment of unconventional cancer therapies. As we shall see, the next Canadian delegation arrived with less open minds.

Walde made his report to the health protection branch of Health and Welfare Canada. The Ontario Ministry of Health requested an assessment from two leading physicians of whether or not it should waive its standard practice of not paying for experimental cancer therapies in the case of antineoplastons. Bureaucratically, this would have been a major move for the Ontario Health Insurance Plan, and the fact that the possibility was entertained speaks volumes for the difference between the cultures of medicine in the United States and Canada.

Two Toronto physicians, Martin E. Blackstein, Head of the Division of Oncology at Mount Sinai Hospital and Daniel E. Bergsagel, Chief of Medicine at Princess Margaret Hospital, visited the Burzynski clinic and research institute on November 15, 1982. Both were powerful and respected physicians in the Canadian cancer establishment. They returned with a blistering, critical report. The essence of their critique was that (1) there was little evidence that antineoplastons had significant antitumor activity against human or animal cancers; (2) Burzynski’s argument that antineoplastons were species-specific created a line of argument for avoiding standard animal and tissue culture screens and required testing in humans; (3) four patients whom Burzynski had described as achieving remission in a test of antineoplastons had in reality not benefited from the treatment; and (4) a review of 12 of Burzynski’s “best cases” showed results that were very questionable. They reported:

We were surprised that Dr. Burzynski would show us such questionable cases. We were left with the impression that either he knows very little about cancer and the response of different tumors to radiation and hormonal measures, or else he thinks we are very stupid, and he has tried to hoodwink us. As we look back on the cases we were shown, we are left with the impression that the only patients who were still alive either had slowly growing tumors, or had received effective treatment before being referred to Houston.18

Moss has devoted a large section of his chapter on Burzynski to refuting this report, saying that the Canadian physicians’ critique of Burzynski’s cell line experiments was entirely inaccurate; that species-specificity is well-known in nature and independently demonstrated with respect to antineoplastons; and that they misread and misrepresented their review of Burzynski’s 12 cases. Burzynski also wrote a detailed critique and called their visit “a complete failure and a great disappointment.” He concluded:

It is clearly evident that Dr. Bergsagel and Dr. Blackstein completely distorted the research, production and clinical data presented to them in Houston. One could expect that people who are medical doctors and who occupy important positions in two large hospitals in Toronto should not give false statements to their own countrymen. They should know that the delay in the approval of experimental treatment which they will create will cost lives of Canadian cancer victims.19

Reading the Blackstein-Bergsagel report and the refutation by Moss and Burzynski side by side is like listening to opposing attorneys offering drastically different accounts of the same event. Judging from the documents, the case made by Burzynski and Moss is somewhat more persuasive, particularly with the benefit of having read Walde’s account of his visit to Burzynski, which is far closer to a model of how an open-minded oncologist can assess an unconventional cancer therapy than is the Blackstein-Bergsagel report.

On the other hand, I would emphasize that Burzynski–having gone to the public media to protect himself from medical regulatory opposition in Texas and to develop the patient flow he needed to finance his research program–made public various details of the negotiations for clinical testing of antineoplastons in Canada, to Walde’s dismay and to Bergsagel and Blackstein’s disgust. Although Walde presented a much more sophisticated, balanced, and thorough report than his colleagues, it is hard to fault mainstream oncologists for distrusting a practitioner who was breaking all the basic rules of the game in using an experimental therapy on patients without an FDA Investigational New Drug (IND) permit in either the United States or Canada, and whose public relations person was continuously going public on research issues that should not have been discussed. The fact that at that time it was still technically legal in Texas to use a new cancer drug with patients within the state without an IND permit is often pointed out in Burzynski’s defense. But this legality did nothing to lessen the shock of such a practice to the medical establishment.

Moss describes Burzynski as having “moved swiftly” to follow Walde’s suggestion to apply for IND clearance in Canada so that further studies could be undertaken there. He suggests that the Blackstein-Bergsagel report was the event that ended the prospects for any trials in Canada.20 Walde remembers the events differently, and this is a critical point. According to Walde, the reason the studies did not go forward was that Burzynski refused to cooperate. The reason for Burzynski’s failure to cooperate, according to Walde, was that “he was concerned about protecting his patents–that they would not be fully protected.”21

If Walde’s account is accurate, Burzynski again had an opportunity to reenter the research mainstream. He had a new chance to have antineoplastons tested and evaluated by independent researchers. It is clear from his subsequent record that Burzynski really does want independent evaluation of antineoplastons. But at this point, according to Walde, despite the tremendous pressure he was under in the United States, Burzynski was more concerned with protecting his patents than with moving forward as quickly as possible toward an open evaluation of his therapy. We can look at this from two points of view. If we view Burzynski as a pharmaceutical entrepreneur protecting patents that could result in a vast fortune for his future research and himself, he was doing what many pharmaceutical companies would have done under the circumstances. If we accept the view of Burzynski as an embattled champion seeking to get a promising cancer therapy out to cancer patients as quickly as possible, one could certainly argue that the risk to Burzynski’s patents was not too high a price to pay to regain mainstream research credibility. Here, after all, was a man who told alternative cancer therapy reporter Gary Null: “I’m going to fight no matter what they do, because I believe I’m doing the right thing. I believe that this is our obligation to the people. If you find something that’s valuable, you must continue and I believe we’ve found something that may be able to save lives.”22

Burzynski gives his own version of his reasons for not moving forward with the Canadian research:

Dr. Walde is seriously mistaken regarding the approval in Canada. … I did not consider at all the problem of patent protection versus filing of an IND. First of all, the patent application was filed already, and, on the other hand, I did not have any reservation in sharing the details of production and treatment with Antineoplastons with any researcher all over the world. For instance, I gave the procedure on how to synthesize Antineoplaston A10 to the People’s Republic of China, where I do not have any patent protection. In the future, the People’s Republic of China could be a huge market for pharmaceuticals, much bigger than Canada. As a result of such sharing of technology, the doctors in China were able to synthesize Antineoplaston A10 a few years ago and are using their product successfully in their research.

The initial approach from the Canadian authorities was extremely cooperative, but all of a sudden it changed, perhaps as the result of the second [Blackstein-Bergsagel] Canadian visit. Ultimately, Canadian authorities requested tremendous amount of data which were impossible to produce with our limited laboratory type production of Antineoplastons A2, A3, and A5. To deliver such data and comply fully with the requirements, it was necessary to have fully automatic urine processing plant. … This plant would have been finished many years ago, but it’s not even finished today due to extreme expenses for our defense. Even today, we are not able to comply with Canadian requests.23

I believe in Burzynski’s sincerity in saying this. The directors of many pharmaceutical companies would also speak truly of their desire to save lives. But when one enters the big business of cancer pharmaceuticals, either in mainstream or alternative medicine, the same business considerations are operating. No prudent pharmaceutical entrepreneur would move for more rapid studies and trials if he thought that the process might endanger his patents. Yet by Burzynski’s account, patent problems had nothing to do with why the Canadian research opportunity did not go forward.

A Visit to Japan–My Own Reawakened Interest in Burzynski

The Blackstein-Bergsagel report cooled my interest in Burzynski for years, because I assumed that report was a good faith and neutral assessment. I had not yet read Walde’s assessment. As a result of the Blackstein-Bergsagel report, I relegated Burzynski to the large category of alternative cancer therapists that I frankly did not know what to make of. The evidence of my senses had told me during my visit to Burzynski that he looked like a qualified scientist and that the therapy looked like perhaps the most promising and scientifically open of the pharmaceutical therapies I had seen. But how, I asked myself, could my reportorial assessment of Burzynski match a medically knowledgeable assessment by two very distinguished Canadian physicians who had made a site visit, examined case records, and seemed qualified to assess the science? But in more recent years, the consistent focus of some of the most knowledgeable advocate-analysts of alternative therapies on Burzynski, notably Ralph Moss and Robert Houston of New York, brought me back to take another careful look at Burzynski.

I had three personal friends who had come through the Commonweal Cancer Help Program and believed strongly that Burzynski “had something.” One was a woman from New York with colon cancer with liver metastases. Her liver metastases had stabilized on the Burzynski program and she seemed to be enjoying life extension as a result of the therapy, but she was profoundly embarrassed by a side effect of the treatment that some patients experience which is not often cited by advocates: a strong odor that some people are sensitive to and others (like me) are not. The odor in her case was reminiscent of the odor of urine and made her feel like a “bag lady” who had soiled herself. In at least one instance, she was asked to leave a restaurant because of the smell. Because of the deeply isolating experience of cancer itself, this side effect was very troubling for my friend, even though she believed she was benefiting from the therapy.

A second friend from nearby Stinson Beach had gone to Burzynski with metastatic breast cancer: the therapy had not helped her, but she came away thoroughly convinced that other patients were being helped. A third friend from another nearby town had gone to Texas with metastatic prostate cancer and high hopes, since prostate cancer is one of the cancers for which the Burzynski therapy appears most promising. But he was not helped and subsequently died. A fourth friend and colleague, a health educator like myself with a strong professional interest in objective assessment of alternative cancer therapies, also visited Burzynski and came away with positive impressions.

My real sense that Burzynski possibly did “have something” came when, at the suggestion of Robert Houston, I visited Hideaki Tsuda, M.D., an anesthesiologist, and his colleague, a surgeon named Hiroshi Hara, M.D., at Kurume University School of Medicine in Fukuoka Prefecture. Tsuda had known Burzynski when they were both at Baylor University and had a friendly regard for him, but it was the high regard of a medical colleague, not a “true believer.”

Tsuda had subsequently decided to do an independent evaluation of antineoplastons in both animals and humans. He and Hara and almost a dozen other colleagues formed the Antineoplaston Study Group. He had the advantage that Burzynski had foregone: a hospital setting and a university appointment. He had the further advantage that Japanese law made it much easier for him to proceed from animal studies to patient studies. Japan’s enlightened universal system of health insurance pays for most of the costs of patient care for virtually all Japanese, so that the costs of observing a clinical series of patients given antineoplastons is relatively modest.

In the course of two studies with mice, in which they demonstrated both tumor prevention and inhibition, Tsuda, Hara, and their colleagues resolved to their own satisfaction the issue that had troubled the Canadian oncologists with respect to animal studies of antineoplastons. They acknowledged the discrepancies between animal and human studies regarding the effectiveness of antineoplaston 10, but speculated that the explanation might rest in the fact that antineoplastons are not naturally occurring in mice and are excreted rapidly, whereas humans show longer retention times. They therefore adopted a more frequent and larger dose in the athymic mouse study, reasoning that the smaller doses might be sufficient to prevent tumor formation, but not to inhibit an existing tumor. They concluded:

Antineoplaston A-10 and A-10 Injection are quite different from conventional chemotherapeutic agents because they are both naturally occurring in humans though not existing in animals. Accordingly in experimental studies, a larger and more frequent dose will be required to demonstrate quantitative antitumor effects. An improved effect can be expected in clinical cases from the same dosage levels as evaluated experimentally” [emphasis added].24

In other words, Tsuda and his colleagues were suggesting that the improved anticancer effects that might come with larger doses of the antineoplaston agents would go beyond tumor prevention to stabilization or reversal of existing cancers. The amounts required might seem very large by mainstream cancer therapy standards, but the substance involved was naturally occurring in humans and generally nontoxic.

Tsuda then presented to us ten case reports of patients with whom he and his colleagues had tried the antineoplaston therapies. These case presentations, which, with one exception, have not yet been reported in scientific journals, were what truly awakened me to the possibility that antineoplastons worked.

One case, which Tsuda has reported publicly, was described in the July-August 1990 issue of Oncology News. At a Swiss conference that is discussed further below, Tsuda reported that his group had administered a combination of cisplatin and antineoplastons to an elderly woman with inoperable metastatic ovarian carcinoma with massive ascites:

At the second-look operation following this therapy, the ovarian tumor could be removed easily, Dr. Tsuda reported. … “She is now enjoying her life very much without any side effects,” he added. “By this kind of combination, we were able to augment the anticancer effect and reduce the miserable side effects of anticancer agents and keep up the patient’s quality of life.”25

Obviously, my report of the work of Tsuda and his colleagues at Kurume University Medical School in Japan is a reportorial account of the clinical impressions of a physician who was summarizing some early and limited case data. But in all my years of doing this work, rarely has an account had such an impact on me. Tsuda has, to the best of my capacity to discern, the kind of impeccable integrity that surrounds the Japanese cultural approach to science and many other things. He made clear that he had taken absolutely no money from Burzynski to conduct this research, nor would he, in part, he explained only half-jokingly, because he was a little older than Burzynski and in Japan the older colleague would never put himself in such a relationship with a younger colleague. Tsuda, like Burzynski, welcomed outside evaluation of his research, and asked me specifically to convey to Japan Cancer Institute authorities in Tokyo how much he would welcome constructive guidance.

Independent Laboratory Research Supporting Burzynski

In the United States, a review of independent assessments of antineoplastons by scientific researchers further deepened my sense that the promise of antineoplastons should be pursued. Oncology News for July-August 1990 gave a flavor of the excitement that the antineoplaston research was generating. Under headlines that read Trials Underway at Several Research Centers and Antineoplastons: New Antitumor Agents Stir High Expectations, an article led off with a report on independent studies by Dvorit Samid, Ph.D., at the Uniformed Services University of Health Sciences in Bethesda, Maryland, that had shown that “AS2-1 [the antineoplaston] profoundly inhibits oncogene expression and the proliferation of malignant cells without exhibiting any toxicity toward normal cells. … The Antineoplaston can actually induce terminal differentiation [reversion toward normal cellular structure]. … `Such a dramatic phenomenon is seldom seen,’ Dr. Samid noted.”26

At the same conference, Burzynski reported a phase II trial of the antineoplaston AS2-1 (that Samid had tested) combined with the hormonal regimen DES (diethylstilbestrol) in 14 patients with prostate cancer resistant to hormonal therapy: “`By the end of the study we were able to identify two cases of complete remission, three partial remissions, seven objective stabilizations, and two cases of progressive disease.’ Clinical improvement was accompanied by a drop in prostate cancer markers and by improvements in bone scans.”27

Burzynski has now also completed but not yet published a phase II trial with astrocytoma (a nerve tissue tumor). While he is not free to talk of the trials until they are published, patients report that he is having really remarkable success with stage IV astrocytomas in children, and some interesting results with astrocytomas in adults, and with glioblastomas (brain cancers) in children and adults.

On March 16, 1989, the FDA approved Burzynski’s request for an IND permit to do a phase II clinical trial at an independent institution on the effect of antineoplaston therapy on 15 patients with metastatic breast cancer.28 But as of April 1991, the trial had not begun because Burzynski had not yet found the right pharmaceutical industry partner to underwrite the $400,000 to $600,000 cost of the trial.

The growing interest on the part of mainstream cancer researchers in Burzynski’s work and increasing evidence of antineoplaston’s effectiveness in some cancers led to the announcement in December 1991 of NCI-sponsored phase II trials of antineoplastons with brain cancer patients. On October 6, 1991, an NCI site visit team reviewed a best-case series of results using antineoplastons with brain tumors at the Burzynski Research Institute. As a result of this review, the NCI decided that phase II trials were indicated in order to confirm tumor shrinkage documented in this series and to determine the tumor response rate. The NCI will be conducting four independent phase II trials of antineoplastons A10 and AS2-1 with brain tumor patients having glioblastomas, astrocytomas, pediatric brain tumors, and low-grade astrocytomas.29

Burzynski’s Current Impressions of the Effectiveness of His Therapy

Asking the developer of a new unconventional therapy for his own estimate of the efficacy of that therapy is a delicate but important step in assessing unconventional cancer therapies.

In our interview, Burzynski said he was clinically impressed, especially with the results with prostate cancer (which he has reported a phase II clinical trial on, described above) and brain cancers, specifically childhood gliomas, which he plans a phase II study on. After that, he said, in descending order, he is impressed with results with non-Hodgkin’s lymphomas (80% of tumors reduced by 50%) and pancreatic cancers (70% of tumors reduced by 50%). Breast cancer follows, with lung cancer and colon cancer further down the list. I asked if he was referring to brief or sustained responses, and he replied that he was referring to sustained responses. Did this mean that the patients were cured? No, he said, the cancers could certainly recur. These figures, he added, were for responses using his synthetic antineoplastons. He is in the process of completing a production plant for production of a natural antineoplaston derived directly from urine. With the natural product, he said that he expected better responses with mesotheliomas and lung, bladder, breast, and colon cancers.

I pointed out that Tsuda in Japan had said that antineoplastons seemed to be more effective in keeping a tumor from growing, and therefore sustaining life, rather than in reducing the size of the tumor, which Burzynski was clearly describing in his estimates of objective responses described above. He replied that Tsuda had been largely limited to oral antineoplastons and that the injections were more effective. Burzynski then offered an interesting insight into the way he believes antineoplastons work:

In humans, normal cells die off after 20 to 60 divisions. They enter a terminal differentiation at that point and die. In animals, they can sometimes revert back, but not in humans. Cancer cells become essentially immortal: they do not die, and so with the process of division the tumor grows. In highly malignant cells, those 20 to 60 divisions can happen very fast. So if you can force differentiation, they will die much more quickly and you will see the tumor reduction more quickly. That is why we can see better results with glioblastomas or pancreatic cancers, which are fast-moving cancers. In slower-growing tumors, like breast cancer, it can take longer to see results, unless you add some chemotherapy or interferon, which can shorten the course.30

This issue of the difference in efficacy between the synthetic and natural antineoplastons is one that troubles some of Burzynski’s critics. Burzynski’s early work with cancer patients used the natural antineoplastons, and they may be more effective. One scientist who stayed very close to Burzynski’s research from its inception through 1985 reported:

When Burzynski was using the natural product, I felt he had stumbled onto a really important discovery. The early cases suggested that. But when he started using the synthetic, my friend who worked there would say to me that the patients were dying. I think he had to make the move to synthetics for economic reasons–the natural product was too expensive to produce.

This scientist, who spoke on condition that I not identify him, had excellent access to the opinions of a dedicated staff member within the Burzynski Research Institute who did like and support Burzynski. This opinion of a loyal and longtime Burzynski staff member regarding the difference in efficacy between synthetic and natural antineoplastons, which parallels Burzynski’s view that the two products work differently with different cancers, is thus worth recording. Burzynski offers a different view:

The synthetic Antineoplastons are not less important than natural ones. They simply have a different spectrum of activity. … This could lead to misconceptions for somebody who has been associated with us for only a limited period of time. Knowing which types of cancer respond the best [to synthetic vs. natural antineoplastons], we are trying to make an appropriate selection of patients. We are trying to limit the admissions of patients with adenocarcinoma of the lung, because our production capacity of Antineoplaston A2 is very small. Once we have the automatic urine processing plant ready, we will be happy to accept a large number of lung cancer patients.31

Finances and Pharmaceutical Agreements

Two other subjects of concern that are often raised by critics of Burzynski are the financial workings of the Burzynski Research Institute and the question of whether Burzynski will ever consummate an effective agreement with a major pharmaceutical corporation that will take antineoplastons through the hugely expensive process of clinical trials and, if appropriate, commercial marketing for all cancer patients.

With regard to the question of whether or not the finances of the institute have been professionally run, Burzynski said quite candidly in our interview that the finances had been handled properly in the early years when he was personally responsible, from 1977 to 1981, but as the institute grew he hired “a succession of bad people” and only achieved what he regarded as good financial management 2 years ago.

But a more important financial question is an ethical one. I asked Burzynski whether or not he had always been careful to tell patients what he believed he could and could not offer, given the substantial patient flow he needed to finance the research program and the large and often unreimbursable burden that using the therapy imposed on patients.

Burzynski replied that in the early years, he did not know which cancers would respond and which would not, so in his view–and in the view of colleagues with whom he discussed this issue–the decision was properly on the patient’s side. In later years, he said he has been more able to describe to patients whether or not he has had success with their kind of cancers, although some want to try the therapy even when the probability of success is not high (the same dilemma is well-known to conventional oncologists). He also responded that, in many cases, the effectiveness of the therapy can be determined within 6 weeks, which somewhat limits the financial burden to the patient.

The broader question of whether Burzynski will actually enter a working partnership with a major pharmaceutical firm to develop and market antineoplastons is also a question for critics and friends who note Burzynski’s long-time distrust of large bureaucracies and organizations that he sees as trying to restrict his freedom.

The Burzynski Research Institute announced with some fanfare that Burzynski had signed a letter of agreement with Sigma-Tau, Italy’s largest pharmaceutical company, authorizing them to conduct laboratory, preclinical, and clinical studies on A10 and AS2-1 in Europe.32 But in August 1990, Burzynski’s public information officer, Le Trombetta, expressed her frustration with the very slow pace of these announced studies and clinical trials. Sigma-Tau was also, Burzynski reports, originally interested in funding the FDA-approved phase II trial with metastatic breast cancer patients, but has since backed away from the agreement. Burzynski is said to be near agreement with another pharmaceutical firm that will finally take antineoplastons into the legal and commercial development that has eluded the agent for so long. The question is whether the successful agreement will always be around the next corner.

A Pattern of Distrust

Does Burzynski have a real pattern of distrust of large organizations, and has this pattern hampered the development of antineoplastons? This is a significant question.

It may be that Burzynski’s early experience with the authorities in Poland provides an explanation of his aversion to bureaucracy. Burzynski was one of the youngest and most promising graduates of the Medical Academy of Lublin, but he refused to join the Communist Party, which Moss tells us was “the perquisite for academic achievement.” As a result of this and the jealousy of classmates, “Burzynski suddenly found himself drafted in the Polish army–one of only two doctors from the academy drafted that year. … Finally, with the help of influential scientists, he was able to emigrate.”33 That was surely a formative experience for a brilliant young scientist: that his innate instinct to avoid the Communist Party bureaucracy resulted in a highly punitive military assignment.

He then came to America and became again an extraordinary academic success at Baylor as he had at the Medical Academy of Lublin. But when he was asked to do the equivalent of joining the Communist Party at Baylor–again a prerequisite for advancement–he again balked and went his own way. And, just as was the case in Poland, anyone could have told Burzynski that the consequences of what he planned to do would be as severe in the United States as they had been in Poland.

In more recent years, the pharmaceutical partnerships Burzynski has explored in the United States and abroad remain, for whatever reasons, just around the corner. The research and clinical studies continue to move forward simply because of the apparently compelling nature of the science that Burzynski has uncovered. The question for the coming years is whether or not Stanislaw Burzynski will actually sign up for and complete the major pharmaceutical agreements that are prerequisites for the large controlled clinical trials that will definitively evaluate the promise of antineoplastons. Skeptics claim that he does not actually want these trials. I believe that is entirely incorrect. But who knows whether he will, on the one hand, overcome a lifetime of distrusting partnerships with large bureaucracies and join forces with a pharmaceutical company or, on the other hand, ultimately try to win scientific acceptance of antineoplastons on his own?

I asked Burzynski whether there was anything I had not asked him that he would like to say:

You have to understand all the attempts I have faced since Poland that were aimed at limiting my freedom. For the past fourteen years, I have been working under severe conditions with formidable enemies. The State Board of Medical Examiners, the FDA, the insurance companies … this has not been the ideal way to do research.34

Burzynski, like Max Gerson and Emanuel Revici–all great innovators in unconventional cancer therapy who immigrated to the United States as refugees from Central Europe–came from a medical tradition which tolerated and protected the integration of science and clinical practice. Permitting physicians to conduct extended clinical programs of experimentation with cancer patients, which the European system essentially allows physicians to do, brings both great risks and the potential of great benefits. Gerson, Burzynski, and Revici came to America believing that this was the land of freedom, and each was already trained in the permissive culture of European medicine. One can add to this that Burzynski, at least, like many scientists, was also by character antiauthoritarian. So all three pursued their unconventional cancer therapies in this new land, where perhaps they did not fully appreciate the cultural clues about the practice of medicine. And each faced the full force of opposition that organized medicine often visits on those who contravene the established norms–developed both for patient protection and for the trade-union interests of organized medicine–especially in the cancer field.

Practitioner, Therapy, and Service Delivery

In trying to find one’s way through the morass of conflicting evidence on Burzynski and antineoplastons, let us return to the distinction between the practitioner, the therapy, and the service delivery.

The practitioner: Burzynski himself, most observers agree, is a brilliant man with a genuine scientific interest in the open assessment of his antineoplaston research, an attitude which is relatively rare among major practitioners of alternative pharmacological cancer therapies. Burzynski’s commitment to open science in the face of extensive opposition deserves commendation. And yet, while Burzynski is unquestionably brilliant, while his commitment to science is profound, and while (most important) he may have made an important discovery in cancer therapy, legitimate questions persist about the wisdom of the decisions he made to go outside of mainstream institutions instead of inside them, decisions which have profoundly affected the course of the development of antineoplaston therapy and the prospects for its availability, proven and at reasonable cost, to cancer patients. Burzynski and his supporters have one view of these choices; his critics have another; the question remains an important and fair one.

The therapy: Antineoplastons as therapy–based on the current scanty research and clinical evidence–may well be recognized in coming years as having considerable benefit in the treatment of some cancers. The extent of this benefit is not yet clear, but it could be significant. More important, Burzynski has only been able to explore a small corner of the research that antineoplastons open up. At present, antineoplastons bring benefit to only a portion of patients seeking help at the Burzynski Research Institute, most of whom are suffering not only from advanced disease but also from the toxic side effects of previous treatment. Despite the laboratory evidence, the phase II clinical trials that Burzynski has reported, and the independent clinical studies of Tsuda in Japan, the history of science warns us emphatically that there is a long road to travel before we know the extent of the actual benefit of antineoplaston cancer therapy.

The service delivery: The largest legitimate doubts about antineoplaston therapy could be said to lie in the area of service delivery, broadly construed. I have already asked whether Burzynski made the right choice–from the point of view of the well-being of cancer patients–in turning down the offer to join the Baylor College of Medicine Department of Pharmacology to pursue his warmly welcomed line of research within the establishment. Was the extraordinary choice to build a major research program on high medical fees the ethical and necessary decision? Was Burzynski right to court major media attention to fend off the assaults on him and to keep the flow of patients coming in the early years so that the research could be continued? How strenuous an effort has the Burzynski Research Institute made to discourage cancer patients who are unlikely to benefit from antineoplaston therapy but who wanted to come out of desperation and who have the money to pay for the therapy?

There are no final answers, only the questions. I was impressed by Burzynski’s frank advice to a friend of mine with metastatic ovarian cancer that she probably would not benefit from his therapy. Several other friends of mine have also been told not to come, and that is greatly to Burzynski’s credit. One of the marks of less credible unconventional cancer treatment centers is that practitioners tell everyone to come who has money to pay for services.

Burzynski is far from alone in facing the issue of whether or not he should give expensive treatments to desperate patients who may not benefit. It is a perennial problem for practitioners of both conventional and experimental therapies. In experimental mainstream cancer therapies, the dilemma was most poignantly clear at the for-profit Biotherapeutics program in Franklin, Tennessee, which was run by former NCI researchers, who, like Burzynski, also charged very high fees for individually designed experimental research pharmaceuticals. (Now reorganized and called Response Technologies, the firm is no longer involved with experimental protocols, but now focuses on the outpatient delivery of “intensive” cancer therapies normally available only through hospitals.) Biotherapeutics had also faced the charge that it allowed patients to come who were very unlikely to benefit. Within the cancer establishment, outrage was expressed about the tactics of Biotherapeutics in offering experimental therapies for wealthy patients (often themselves physicians), both on the grounds that this contributes to a two-track medical system for the rich and the poor, and on the grounds that patients are paying for unproven therapies. Even if the credentials of the Biotherapeutics team were impeccable, the fear existed that the pay-for-individual-research scheme may allow the unwashed hordes of unconventional cancer therapy practitioners to breach the cordon sanitaire so carefully constructed to prevent cancer quackery in the United States.

There are currently many examples in mainstream medicine–coronary bypass surgery for patients who are unlikely to benefit is one of the most egregious–of expensive therapies that will help some patients, but are offered to many others who are unlikely to benefit. That Burzynski is asking patients to pay huge fees for an experimental therapy must be assessed in this context.

Presently, the Burzynski therapy costs $3,000 to $5,000 a month–or $36,000 to $60,000 a year–depending on the specific medications a patient is taking. Most patients cannot get their insurance companies to reimburse these expenses. And even if an insurance company is willing to pay, that raises the larger question of whether or not all patients in that insurance system should ultimately pay a higher premium for a very expensive therapy, the benefits of which may accrue largely only to certain subsets of cancer patients. Again, this is as real an issue for coronary bypass surgery as it is for antineoplaston therapy.

But ultimately, all the issues we have discussed will fade away. The awesome beauty of science is that in the end Burzynski is either right or wrong about antineoplastons. If he is right, he probably deserves at least a share of a Nobel Prize, because a nontoxic chemotherapy for cancer, even if only partially effective, would be a profound service to mankind. If he is wrong, if his phase II trial results do not hold up in more extended controlled trials, then this will be simply another tortured and unfortunate chapter in the long and often unedifying history of both conventional and unconventional cancer therapies.

Notes and References

1 Ralph W. Moss, The Cancer Industry (New York: Paragon House, 1989).

2 Another less favorable extended account of Burzynski’s work is included in the Report on Unconventional Cancer Therapies by the Office of Technology Assessment.

3 Moss, The Cancer Industry, 289-91.

4 Ibid., 292.

5 Stanislaw Burzynski, personal communication with the author, 13 November 1990.

6 Moss, The Cancer Industry, 294-5.

7 Ibid., 295-6.

8 Burzynski, personal communication with the author, 13 November 1990.

9 Moss, The Cancer Industry, 297.

10 Ibid.

11 Ibid., 296.

12 Ibid., 298.

13 Ibid., 287-8.

14 Ibid., 299-300.

15 Ibid., 300-1.

16 David Walde, unpublished memorandum, 22 November 1982, 7-8.

17 Ibid.

18 M.E. Blackstein and D.E. Bergsagel, “The Treatment of Cancer Patients with Antineoplastons and the Burzynski Clinic in Houston, Texas.” Report to the Ministry of Health, Province of Ontario, undated.

19 S.R. Burzynski, “Response to Site Visit to Burzynski Research Institute on November 15, 1982 and Report by Drs. M.E. Blackstein and D.E. Bergsagel,” Burzynski Research Institute, undated.

20 Moss, The Cancer Industry, 301.

21 David Walde, telephone conversation with the author, 30 August 1990.

22 Gary Null, “This Man Could Save Your Life, But He Can’t Get the Money to Do It,” Our Town 13-19 May 1979. Quoted in Moss, The Cancer Industry, 297-8.

23 Burzynski, personal communication with the author, 13 November, 1990.

24 K. Hashimoto et al., “The Anticancer Effect of Antineoplaston A-10 on Human Breast Cancer Serially Transplanted to Athymic Mice,” Journal of the Japan Society for Cancer Therapy 25(1):1-5 (1990).

25 Oncology News 16(4):1,6 (1990).

26 Ibid., 1.

27 Ibid., 6. For details, see abstracts in proceedings of 9th International Symposium on Future Trends in Chemotherapy, Geneva, Switzerland, 26-8 March 1990.

28 Moss, The Cancer Industry, 334.

29 Press Release, Burzynski Research Institute, 6 December 1991.

30 Stanislaw Burzynski, personal communication with the author, 13 November 1990.

31 Ibid.

32 Moss, The Cancer Industry, 333-4.

33 Ibid., 290-1.

34 Stanislaw Burzynski, personal communication with the author, 13 November 1990.