History

Malignant Law Enforcement

The FDA's war against an unorthodox cancer doctor

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Mary Michaels is pleased to tell me that her 16-year-old son, Paul, is in front of their house in Troy, Michigan, washing the car. More pleased than you might imagine. Nearly a dozen years ago, the 4-year-old Paul was diagnosed with an astrocytoma, the most common type of brain tumor. The prognosis was grim. Doctors warned that the tumor was too entwined in Paul's brain and optic nerve for debulking surgery–where doctors saw through the skull and physically remove the cancer–to do any good.

More bad news followed. Radiation and chemotherapy are the main weapons of mass destruction in the cancer doctor's arsenal. But they've got their own problems. Chemotherapy powerful enough to annihilate Paul's tumor would probably kill him too. The radiation would likely blind him, stunt his growth, and make him incessantly nauseated, and in the end might not give him much more time.

"We started thinking, 'Why would we do this?'"Mary says. "It would be torturing Paul. But the doctors' attitude was, 'You have to treat it somehow.'" So the Michaelses turned to the grab bag of alternative cancer treatments popular among those mistrustful of standard medicine: "We became macrobiotic. The tumor grew. We went to Mexico for Laetrile. The tumor still grew. As crushed as you are coping with a malignant, inoperable tumor, it takes a lot for hope to die."

Then Mary saw an episode of the Sally Jesse Raphael show about a Polish-born cancer doctor named Stanislaw Burzynski. Some of Burzynski's patients had suffered from astrocytomas that apparently had been cured by his unorthodox treatment. "The people on the show were so healthy-looking, "Mary recalls. "I couldn't believe they were cancer patients." By that time, doctors at the prestigious Mayo Clinic had given Paul a definite death sentence. He had the largest brain tumor they'd seen in anyone that age, they said, larger than an egg. In a last-ditch effort, Mary sent Dr. Burzynski her son's scans and medical history. Burzynski thought he might be able to help.

That was more than a decade ago. Whether Burzynski in fact helped Paul remains an open question. But after years of treatment with Burzynski's patented substances, antineoplastons, Paul is 16. Back in the early years of his cancer, every doctor assured Mary that Paul wouldn't make it past the age of 10. The egg-sized tumor is now pea-sized, and, unlike heavy users of chemotherapy and radiation, Paul is neurologically normal and healthy in every way. The cancer, for now, seems under control. (Tumors are often known to return with a vengeance after seeming remission, however.) The biggest danger now to Paul's continued life and health, his mother says, is the U.S. Food and Drug Administration.

The FDA has a long-standing dispute with Stanislaw Burzynski that has embroiled both parties in legal maneuvers for 14 years and earned the agency the ire of Burzynski's patients. As Paul Michaels is wont to tell the press, echoing many other patients, "The government is trying to take away the only weapon I have against cancer." In the struggle for the hearts and minds of politicians and journalists, Burzynski and his patients are at least holding their own. Rep. Joe Barton (R-Tex.), chairman of the House Subcommittee on Oversight and Investigations, has led sympathetic congressional hearings. While the print press tends to be skeptical, Burzynski has been presented in a mostly positive light on TV shows ranging from Hard Copy to CBS's 48 Hours and ABC's Nightline.

Depending on your point of view, the FDA officials battling Burzynski are mindless bureaucrats blocking access to a promising new therapy or dedicated public servants trying to prevent a quack from taking advantage of desperate patients. The truth probably lies somewhere in between. On the one hand, the government has been hard pressed to cite any serious danger from allowing Burzynski to continue treating patients with antineoplastons. On the other hand, despite numerous dramatic anecdotes and testimonials, the effectiveness of Burzynski's approach has yet to be demonstrated. Whatever the merits of antineoplastons, Burzynski's dispute with the FDA starkly poses the question of who should get to weigh the costs and benefits of experimental treatments: the patients whose lives are at stake or regulators acting on their behalf.

Ever since the late 1970s, when Burzynski was a researcher at Baylor College of Medicine in Houston and just beginning to work out his theories on antineoplastons, the FDA has been skeptical of this foreign doctor who dared suggest he had discovered something that might–sometimes, under some circumstances–be a cure for cancer. The FDA has protocols for bringing new drugs to market, and Burzynski has a long history of flouting them. He left Baylor in 1977 amid controversy over antineoplastons–amino acid chains that he believes can communicate "switching off" signals to multiplying cancer cells. Without jumping through the FDA's hoops, Burzynski began treating patients with the substances. He originally extracted his antineoplastons from human urine–a point that has proven priceless for those seeking to cast Burzynski and his drugs in an uncharitable light–but now synthesizes them in a lab.

Burzynski gives me a rough explanation of his theory by telephone during a day off from the latest battlefield of his war with the FDA: a federal courtroom in Houston where he faces up to 290 years in prison for allegedly violating FDA regulations concerning experimental drugs and misinforming insurance companies about the nature of his therapy. "The theory is that cancer is a disease of information processing," he says. "The cancer cells are mistakenly programmed to multiply all the time. There are various switches where you can turn them off, that define 'to be or not to be' for cancer cells. All the antineoplastons do is turn off that switch. But different cancers are caused by different oncogenes. Our switches seem to work on two different genes, and we can't help everyone."

The conflict between the FDA and Burzynski is complicated by the fact that both sides are now sleeping with the enemy. Even as Burzynski fights to stay out of prison for breaking the agency's rules, the FDA has approved and is supervising a set of Phase II clinical trials to test the value of antineoplastons. (Technically, the FDA does not supervise clinical trials, as they will tell you. In practice, however, anyone who wants the approval of the new drug application at the end of the trials will take the advice and obey the whims of the FDA, says former FDA Chief Counsel Peter Barton Hutt.) As a result, Burzynski might get the equivalent of a life sentence for technical violations of FDA regulations, while simultaneously being hailed for discovering a powerful cancer-fighting tool.

The FDA first tried to stop Burzynski from treating patients with antineoplastons in 1983, when it filed a lawsuit against him in federal court. U.S. District Judge Gabrielle McDonald ruled that, while interstate trafficking in the unapproved drugs would have to stop, Burzynski could still treat patients in Texas without FDA interference. During that case, the government side made a motion for summary judgment, which read in part: "If the court declines to grant this injunctive relief sought by the government, thus permitting continued manufacture and distribution of antineoplastons, the government would then be obliged to pursue other less efficient remedies, such as actions for seizure and condemnation of the drugs or criminal prosecution of individuals. "This court would then be faced with ordering the government not to undertake such enforcement actions, if the court truly desired to allow the continued manufacture and distribution of the drugs."

The government has followed through on that threat, illustrating a tendency cited by former FDA Chief Counsel Hutt: "When the FDA loses a case, it has a mind like an elephant…. Once the FDA makes a collective decision, trying to make it let go is almost impossible. These are 'FDA crusades.' In a real sense, they're vendettas." (See "Clinical Trials," May 1995.)

In 1985, the FDA got a search warrant for Burzynski's office, based on the suspicion that he was violating the ban on interstate distribution of antineoplastons. Agents confiscated thousands of patient records. FDA officials allowed Burzynski access to the records only at their convenience and forced him to photocopy them before leaving the agency's Houston office. Burzynski, various patients, and several of his associates were called up before three different grand juries from 1985 to 1994. No indictment followed.

These repeated attempts to incriminate Burzynski led Rep. Barton to send an angry letter to Attorney General Janet Reno in September 1995. "It would appear," he wrote, "that the FDA and the Justice Department are abusing the grand jury process to harass and punish Dr. Burzynski for persuading a federal judge that he is not violating the law by practicing medicine within the State of Texas." Barton asked for all the documentation regarding the government's fight against Burzynski. With political pressure on the FDA regarding more than a decade of harassment with no criminal indictment, in November 1995 a fourth grand jury finally gave the government what it wanted: a 75-count indictment against Burzynski, which led to the trial in Houston.

The government tried to prove that Burzynski treated out-of-state patients with antineoplastons, in violation of Judge McDonald's order. It also alleged that he committed mail fraud against insurance companies by not informing them about the experimental nature of the work for which he was charging them. Most of the patients I interviewed said their insurance companies didn't pay for antineoplaston treatments anyway. Burzynski has been in court many times in civil suits with insurance companies over payment for his treatment. He's won some and lost some.

During the criminal trial, a postal inspector testified that she followed one of Burzynski's employees to a shipping store, where he tried to send a package containing antineoplastons to an out-of-state patient. The employee swears he did this on his own time and without Burzynski's knowledge, out of compassion for the patient. The son of an elderly Burzynski patient testified about harassing and frightening visits from FDA agents, who told them Burzynski was a fraud.

Burzynski's lawyers weren't allowed to present any evidence regarding the possible efficacy of antineoplastons. That's because the government's case has nothing to do with whether the drugs save lives, whether Burzynski's treatment is medicine or quackery. Whatever antineoplastons are, they aren't FDA-approved. Yet the agency saw enough promise in Burzynski's approach to authorize clinical trials. The act for which Burzynski might spend the rest of his life in prison–shipping antineoplastons across state lines–he is now doing openly and gleefully, because the FDA has told him it's OK in the context of the clinical trials.

Another apparent sign of flexibility came in March 1996, when the Clinton administration announced plans to speed up the approval process for cancer drugs whose tumor-shrinking potential has been demonstrated. Clinton vowed that "dozens of [new cancer drugs] will get to the market sooner." Secretary of Health and Human Services Donna Shalala swore the new system would go into effect immediately. The Dallas Morning News reported that, under the new rules, "all a company has to show is that the drug can measurably shrink the size of a tumor, even for only a short time," and speculated that the reform might affect Burzynski's case.

Burzynski thinks the preliminary results of the clinical trials already satisfy the tumor-shrinking requirement. But when he petitioned the FDA in September for approval under the new guidelines, the agency wrote back that "[t]he Cancer Initiatives announced in March of this year by President Clinton and Vice President Gore did not set aside any laws or regulations related to approval of new drugs for cancer treatment. "We have not changed or lowered our standards for the approval of such drugs." Which makes you wonder what the point of the March 1996 announcement was, aside from making Clinton seem compassionate.

All that Burzynski's patients–and potential patients–care about, of course, is whether antineoplastons work, not whether the FDA has approved them. But according to Assistant U.S. Attorney Mike Clark, who is prosecuting Burzynski, even raising that issue would be "irrelevant, emotional, prejudicial, and misleading." From the government's perspective, that's undoubtedly true. But for someone who wants or needs an unapproved treatment, the FDA's devotion to regulations is the real crime.

Young Paul Michaels is not Burzynski's only success story. There are dozens more among the 2,500 or so patients he has treated, all telling similar stories of cancer diagnoses, medical death sentences, and desperate searches in the realm of alternative therapies that ended in Burzynski's healing arms. Their dedication to Burzynski, whom they call "Dr. B," is such that they've formed a pressure group to lobby on his behalf. The Burzynski Patient Group is run by Steve Siegel, the head of a Los Angeles-area marketing company and the husband of patient Mary Jo Siegel. "We've become another pain in the ass to the FDA–we're like a new AIDS lobby," he says with a proud laugh. Siegel and his wife spend 100 hours a week running the Burzynski Patient Group, answering questions, referring people to other patients, lobbying Congress and the FDA. Through the efforts of the group, Barton held a series of congressional hearings from August 1995 to February 1996 to discuss Burzynski's treatment and the FDA's treatment of Burzynski.

A series of patients and their relatives appeared before Barton's subcommittee. Some said Burzynski's treatment saved them or their loved ones. (One patient who testified about the wonders of Burzynski's treatment, Doug Wagner, has since died.) Some witnesses acknowledged that antineoplastons didn't keep their relatives alive but said Burzynski should be allowed to continue his research and treatment anyway.

One subcommittee staffer who works on the Burzynski matter wonders aloud about the FDA's goals in this case. Three grand juries without an indictment, he says, is "unheard of. Ask any career federal prosecutor with no dog in this fight, and you'll learn it's extremely unusual to have a succession of grand juries without an indictment being issued. Grand juries will generally indict a ham sandwich if a prosecutor asks them to. The underlying charges are not that complicated. The drug's not approved–was the doctor distributing it? It shouldn't take 10 years to get an indictment. We still don't really know why it is that the federal government and the U.S. Attorney's Office in Houston–a large city with lots of federal criminal issues–are spending all this time going after Burzynski, what there is about this case that merits this kind of devotion of resources, when there doesn't seem to be any compelling evidence of a threat to public health. We asked the FDA when [Administrator David] Kessler testified before Barton's committee: Where's the harm? Where are injured people?"

Kessler didn't have the answer. He referred Barton to Robert Spiller–who, as the FDA's lawyer in the 1983 case, had vowed vengeance against Burzynski more than a decade earlier. Spiller related the tale of two patients he had read about in a report from the Canadian government. Both had died of a blood infection from the subclavian catheters through which some patients administer their antineoplastons. (For lower dosages, the drugs are taken in pills.) "That has nothing to do with antineoplastons per se, just the way they were administered," the subcommittee staffer observes. "That could happen with any kind of medicine administered through intravenous catheters."

Congressional pressure, the staffer maintains, pushed the FDA into allowing all of Burzynski's previous patients to be treated in the ongoing clinical trials, even if they didn't meet the usual criteria for participating in such research. "The subcommittee broke the logjam," he says. "Burzynski is getting farther along in the approval process. We'll soon have data on hundreds of patients under many different protocols. People want to know if this stuff works, if this is a breakthrough, or if Burzynski has nothing and is just stealing people's money. But with no accomplishments, there would be no following. If he's a con man, where [are] the victims? I've gotten a few complaints from disgruntled relatives, but I can count them on one hand. And none of them would talk to me face to face so I could question them. Even where it doesn't prove ultimately successful and the patient dies, I have relatives telling me they still swear by the guy, and their mom loved him, and they think she got some time she wouldn't otherwise have had and at least wasn't made sicker by the treatment."

The FDA isn't Burzynski's only enemy. The Texas Board of Medical Examiners has also been fighting him for years. "There's always something new going on with Burzynski," says Tony Cobos, the board's general counsel, when I ask him to sum up the story of Burzynski's troubles with state regulators. He suggests I order the relevant pile of documents. Nearly 100 pages arrive, chronicling how the medical board snatched victory from the jaws of defeat in their struggle to divest the doctor of his license. First an administrative law judge decided that all Burzynski had to do was change some of his advertising. Then the board decided to revoke Burzynski's license despite the judge's decision. The case entered the courts, where a district judge reinstated Burzynski's license and an appeals court reversed that decision, upholding the license suspension. The issue is now awaiting possible consideration by the Texas Supreme Court.

How the board got on Burzynski's case isn't clear. "Our investigative files are confidential," Cobos says. "We don't say if people filed a complaint or where it came from. But obviously a complaint was filed." Burzynski does have some unsatisfied customers. Take Andy Cayon, a patient from Connecticut who started out as a Burzynski booster but, after six months of treatment and no apparent shrinkage of his brain tumor, now complains that the doctor's office won't even take his phone calls. Whether antineoplastons are worth a damn is still an open question; that they are not a miracle cure-all is clear to everyone, even Burzynski.

Burzynski's man in charge of managing the bureaucratic end of the clinical trials, Dean Mouscher, acknowledges that up to one-fourth of the brain cancer patients in the study so far are showing no response at all. Of course, conventional cancer treatments can't promise miracles all the time either–and often have horrific side effects to boot. The worst any user of antineoplastons can tell me about them is that they made him very thirsty all the time. In fact, the FDA let Burzynski skip Phase I of the clinical trials for antineoplastons–the test for toxicity–because there was already ample evidence of long-term safety.

Whether or not antineoplastons work, Burzynski makes it clear that he offers no guarantees. All patients sign a consent form which states that antineoplastons are "purely experimental" and that "the use of such substances as a prescriptive drug has not yet been approved by any State or Federal regulatory agency, nor has its use been approved or adopted by any single or group of Medical Institutions at this time." The form also says that "[n]either [Burzynski] nor anyone associated with him is permitted to ship his medication out of the state. Transportation of Antineoplastons out of the state of Texas by a patient or his or her agent could be viewed as a violation of the federal Food and Drug Laws. No assurances have been made to the PATIENT by anyone as to any results expected to be obtained from such treatment….[N]either success nor improvement in PATIENT's condition is warranted or guaranteed by such treatment. PATIENT understands that the reverse may be true and that PATIENT's condition may worsen as a result of the treatment….Dr. Burzynski makes no claims that Antineoplaston drugs will cure or stabilize cancer….[T]he U.S. Food and Drug Administration and the National Cancer Institute claim that this drug is not yet proven to be effective in the treatment of cancer."

The default assumption when someone jumps up claiming to have cured otherwise incurable diseases is: The guy must be a quack. Is Burzynski? The sort of rough empiricism most of us use in making these judgments (how many of us have done, or carefully studied the results of, double-blind, long-term studies of health claims on which we hold opinions?) gives evidence for both sides.

Despite all his FDA troubles, Burzynski hasn't set up shop in Mexico, where desperate patients in search of alternative treatment already go for such supposed cancer cures as Laetrile. There, he could charge whatever he wanted, with no hassles from the authorities. And though he seems to be making a tidy living at his practice, Burzynski doesn't make wild promises to every patient who comes his way. He readily admits that there are certain cancers for which his treatment just doesn't seem to work. Mary Jo Siegel–who is now in total remission from a low-grade non-Hodgkin's lymphoma, a recovery for which she credits antineoplastons–learned about Burzynski from a fellow cancer support group member with ovarian cancer, who had gone to Burzynski for help but was turned away. Burzynski told her he'd had little luck with ovarian cancers.

Nor is Burzynski's treatment approach utterly implausible. In addition to radiation and chemotherapy, the National Cancer Institute recognizes "biological treatment" for cancer, which "uses materials made by your body or made in a laboratory to boost, direct, or restore your body's natural defenses against disease." In the 1980s, NCI scientists were interested enough in antineoplastons to check out some of Burzynski's best cases, and they were impressed enough by what they saw to begin trials, which ended inconclusively after Burzynski disapproved of their methods. The NCI shifted from a protocol on which both parties had agreed, Burzynski says, and began including patients whose tumors were too big and had spread too far for the doses of antineoplastons used. But NCI researcher Nicholas Patronas, a member of the team that looked into Burzynski's treatment, nevertheless testified during the Texas medical board hearings that the results he saw were "amazing." Antineoplastons seemed to have eliminated at least five patients' brain tumors, he said, and "they are not handicapped from the side effects," whereas "side effects of the most aggressive previous treatments are worse than the tumor itself. I think it is impressive and unbelievable."

The NCI is even now studying a substance called phenylacetate, chemically similar to antineoplastons but not patented by Burzynski. One scientist who has worked with the NCI and the National Institutes of Health says he has done enough in vitro work to feel confident that Burzynski is on to something–that it is the particular combinations of amino acid chains in the antineoplaston treatment that work, much better than phenylacetate alone. Yet he insists that higher-ups in the NIH had no interest in pursuing this work because of animus against Burzynski, or at least because of the unsavory reputation his ongoing FDA fights have given him.

Several factors have made it hard for Burzynski to improve his reputation. He has a disturbing tendency to compare himself to Galileo and Pasteur, in classic quack style. He and his supporters tend to blame huge, vague, overarching conspiracies on the part of the establishment for his troubles. Because he has patented the antineoplaston formulas, Burzynski stands to profit even more handsomely than he has so far if the drugs pan out in clinical tests. He makes sweeping statements about the potential of antineoplastons, not only for treating various types of cancer but for curing ailments such as AIDS, lupus, multiple sclerosis, arthritis, and skin diseases–all of which he attributes, like cancer, to information processing failures.

Burzynski's public relations apparatus also engages in some questionable practices, such as circulating a letter, supposedly from the chairman of the Department of Neurosurgery at the University of Texas M.D. Anderson Cancer Center, recommending antineoplastons for a prominent Houston TV news anchor. The doctor is adamant that he never signed or approved the letter, which he says his secretary wrote at the patient's insistence. Finally, while it probably shouldn't be held against him, Burzynski's Polish-inflected speaking voice is eerily reminiscent of Dr. Nick Riviera, the recurring quack-for-all-seasons on The Simpsons.

In the end, none of this should be relevant. However disreputable Burzynski may seem, however much money he's making, the scientific issue of whether his treatment works still has to be resolved. By the same token, the anecdotes and testimonials from his patients do not count as proof. Most of Burzynski's patients see other doctors in their hometowns. Mary Jo Siegel is a patient of Dr. Peter Rosen at UCLA, who remains resolutely unimpressed by her remission. Spontaneous remissions, however rare, do happen, and he thinks that they explain Burzynski's apparent successes.

Dr. Carl Ott of the Memorial Clinic in Olympia, Washington–another doctor with a brain tumor patient, Richard Gribben, who has improved under antineoplaston therapy–still doesn't recommend it to patients generally, mostly because "people don't often come to me for advice on unconventional therapy. If [Gribben] had any real hope with conventional therapy, I would have tried hard to talk him out of [using antineoplastons]. Our oncologist on staff thinks Burzynski is a quack preying on the desperate. When he saw the results [with Gribben], he just kind of shrugged his shoulders." Might Gribben have undergone a spontaneous remission? "I've been in practice 10 years," says Ott. "I see 10 people a year with similar problems, and I've never seen a spontaneous remission comparable to this. Some do better than others, but I've never seen advanced tumors disappear spontaneously like this. I hope they don't shut Burzynski down. He's been remarkably effective and nontoxic for my patient."

But if Burzynski is imprisoned, the research will come to an end. Steve Siegel asked U.S. Attorney Mike Clark whether the government had a contingency plan for the patients should Burzynski go to prison. "He said, 'That's not my responsibility,'" Siegel recalls. "He would assume that responsibility is with the FDA. But the Patient Group had a meeting with the FDA in early December. They said it wasn't their responsibility if the clinic closes down, either." Says Burzynski, almost nonchalantly, "If I am in prison, this whole thing would collapse and people will die."

Burzynski isn't going to prison–yet, anyway. Just before press time, the jury in his Houston trial announced that they couldn't reach a verdict. Six jurors wanted to acquit and five wanted to convict on all 75 counts. One juror was undecided. The judge then dismissed the charges of insurance fraud. U.S. Attorney Clark vowed to bring Burzynski to trial again in May on the FDA-related charges.

Burzynski's satisfied patients and would-be patients don't care whether he has dotted all his is to please FDA bureaucrats. They are sick; they are dying; in most cases, they've been told by a bevy of the best conventional doctors that they are as good as dead. (The path to Burzynski, for the patients I talked to, wound its way first through places like Stanford, UCLA, the Mayo Clinic, and the Dana Farber Cancer Center.) What possible harm, they wonder, is the FDA trying to prevent by keeping us from taking the substances we want, that we think give us some hope?

The FDA is not completely deaf to such appeals. Washington, D.C., lawyer Jonathan Emord represents patients petitioning the FDA's Center for Drug Evaluation and Research for "compassionate use" exceptions or slots in the clinical trials. Sometimes he succeeds; sometimes he fails. Emord is eagerly awaiting the Supreme Court's response to last year's rulings by two circuit courts declaring a right to die in certain circumstances. If upheld, those decisions could open the door to new arguments about the right of dying patients to take any treatment they choose.

When faced with a face, a name, a case, a specific impending death, the FDA often is capable of bureaucratic mercy. But patients wonder: Why must they make it so hard? Why must we come before them as supplicants? Why not just let us pay our money and take our chances?

"Sure, there's the safety valve of the compassionate use exception," says the congressional staffer who works on the Burzynski case. "And the agency has told me off the record that they've approved 90 percent of compassionate use requests from Burzynski. But what about the 10 percent? I knew one 67-year-old man with prostate cancer and a heart condition. The FDA at first refused him, saying surgery could still work for him. The man didn't want surgery. He was afraid of dying on the table because of his heart condition. The patient didn't consider surgery a viable option for himself–but the FDA did. He eventually got his way, because the FDA wondered if they could defend this decision before Congress.

"That's a hell of a way to make decisions for terminally ill people."

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