Trials of a Cancer Doc
Experimental drugs and a 20-year fight with the FDA
To thousands of cancer patients who have failed to benefit from standard medical treatments and who have reached the end of hope, Dr. Stanislaw Burzynski possesses the cure of last resort. Burzynski runs a cancer clinic in Houston, where he has treated at least 3,000 cancer patients with experimental drugs he developed more than 20 years ago. Patients come from as far away as Turkey and Chile for his treatments, and pay up to $14,000 a month, hoping for a cure.
It's a hope that Burzynski himself has fueled assiduously. In appearances on television shows like 48 Hours he talks of his drugs curing cancer; his Web site claims few side effects. His supporters claim that his drugs can reduce a brain tumor from "the size of a grapefruit to smaller than a pea."
In interviews with the New York Times and U.S. News, and according to Justice Department papers, the doctor has said his experimental drugs, known as antineoplastons, can help patients with not only a wide array of different cancers, such as prostate, brain, breast and lung, but also a host of other diseases such as multiple sclerosis, arthritis, Parkinson's disease, HIV, and even baldness.
Loyal following. Patients who have taken Burzynski's antineoplastons and gotten better--and there are hundreds of them, he says--compose an intensely loyal following. Pam Murphy, of St. Peters, Mo., believes that antineoplastons have relieved the debilitating symptoms of a rare connective-tissue disease that afflicted her. The parents of 7-year-old Dustin Kunnari from Aurora, Minn., credit antineoplastons with curing their son's medulloblastoma, a type of brain tumor. Mary Jo Siegel, of Pacific Palisades, Calif., believes that antineoplastons cured her lymphoma in 1993. She is now his most vocal supporter. "Dr. B. is a miracle worker," she says. Without him, she says, "I wouldn't be here today." Some doctors also have been impressed by the apparent results of the treatment. Dieter Schellinger, a neuroradiologist at Georgetown University Medical Center, said he was "surprised" by some responses he saw when he reviewed the MRIs of nearly 40 patients whose brain tumors, Burzynski claims, responded to his drugs. "I don't know of any active agent that produces these results," said Nicholas Patronas, a radiologist at the National Cancer Institute, after reviewing five of Burzynski's brain-tumor cases.
Given such testimonials, the U.S. Food and Drug Administration, the agency that approves drugs for treatment and sale, would like to know if Burzynski is really on to something. But it has yet to see convincing scientific evidence that antineoplastons are either safe or effective. The FDA has spent 20 years and $2 million trying to force Burzynski to put his antineoplastons through standard testing regimens, called clinical trials, which are required to gauge whether drugs work.
These struggles have cost the doctor. The Justice Department prosecuted Burzynski in 1997 on a 75-count indictment for selling and administering antineoplastons without FDA approval. Texas medical authorities threatened to revoke his medical license for illegally marketing his drugs. But Burzynski has fought back with persistence and skill. With supportive patients lining courthouse steps and entreating judges on his behalf, he has beaten federal prosecutors, fended off the state of Texas, and held off the FDA. With new patients walking in the door nearly every day, Burzynski has become the most visible purveyor of an unapproved cancer treatment in the nation, with his clinic grossing, he acknowledges, an amount that "could" approach $9 million last year alone. (He says all revenues are used to run the clinic, and it ran at a loss last year.) Burzynski, says Michael Petty, a former FDA lawyer who now practices in Washington, D.C., "has beaten the system."
But the doctor's long string of successes is now in jeopardy. In August, a Houston judge awarded Provident Life & Accident Insurance Co. $233,044 plus interest in a billing dispute with Burzynski. The judge said Burzynski had "failed to inform Provident of the nature of his treatment, its extraordinary character, or its illegality" and said that it was "unconscionable for him to retain the funds." Burzynski says there was no finding of fraud and he has filed a notice of appeal.
He has also been hit with his first patient lawsuit: A New York State couple has filed a negligence, fraud, and wrongful-death suit regarding treatment of their 11-year-old daughter, who died in June 1996. "In the opinion of our lawyers, this is a frivolous lawsuit," says Burzynski. Another couple complains clinic doctors misled them about the progress of their 26-year-old daughter, who died on a plane returning from Burzynski's clinic. Burzynski says she died of a stroke and that he had no way of preventing her death.
Whatever the case, Burzynski is enrolling patients in clinical trials per a court order, and has reported interim results to the FDA. This week, however, a respected cancer newsletter plans to publish a review of Burzynski's clinical trials saying that they won't provide meaningful results. The FDA, in a preliminary look at the data, sees no evidence of improvement in eight types of cancer and a response rate in brain cancers that is too small to determine if patients are being helped. The agency also contends that side effects of the therapy contributed to the deaths of seven patients. The doctor challenges the FDA's findings and counters that the agency "doesn't have any evidence that patients died from side effects."
Charismatic. How a Polish-born doctor who came to the United States with $20 in his pocket managed to become so successful is an intriguing tale. Charismatic and self-confident, the stocky, 55-year-old physician posseses a messianic faith in the powers of his drugs. He compares himself to scientific giants like Copernicus and Galileo, whose ideas contradicted accepted axioms of the time. One day, Burzynski boasts, "antineoplastons will be accepted everywhere."
When he arrived in the United States in 1970, Burzynski had a medical degree from Poland's Lublin Medical Academy and an untested idea. From his research, Burzynski believed that bits of protein found in human urine could fight cancer. He called these proteins antineoplastons, a word derived from neoplasm, Greek for tumor. By 1978, he was extracting them from human urine in a lab set up in a Houston garage and using the drugs in his own cancer clinic. Word of Burzynski's experimental drugs spread quickly; patients soon flocked to his door.
When the FDA got wind of what he was doing, it tried to get him to submit the drugs to clinical trials. In such a regimen, experimenters first give a new drug to animals to test its toxicity. Only after such tests can a drug be given in the clinic to human patients, whose histories are documented closely to determine if the drug's cure rate is as good as or better than existing drugs. "Any doctor can line up a few successes," says Robert DeLap, who runs an FDA office of drug evaluation. Carefully designed trials are crucial for cancer drugs, because tumors can swell or shrink of their own accord, and cancers can spontaneously go into remission--making it difficult to know if a treatment is actually responsible for a patient's improvement.
In dealing with the FDA, Burzynski took several tacks. Responding to the agency's demands, he sent voluminous letters in reply. He did some pre-clinical animal tests, but they never met the agency's requirements. When the FDA took Burzynski to federal court in an attempt to close down his clinic, a horde of loyal patients pleaded with the judge on their doctor's behalf. The judge allowed the clinic to remain open if Burzynski would test his drugs according to standard FDA procedures. After more wrangling, Burzynski finally was granted permission in 1989 to conduct clinical trials; he sent out press releases announcing that he would begin testing antineoplastons on women with advanced breast cancer. But he never did. "After we got permission, we didn't have the money," Burzynski told U.S. News. "It costs millions of dollars to do clinical trials."
While the fight dragged on, Burzynski's business grew, nourished by his flair for publicity. Burzynski distributed fact sheets to patients and owners of his penny stock, attacking the FDA and touting his company's product. An appearance on Sally Jessy Raphael in 1988 brought a flood of new customers. Stories about Burzynski soon followed in such publications as Good Housekeeping, The New York Times, and the Washington Post, many of which portrayed him as a victim of an overzealous FDA. Three members of Congress, two critical of the FDA, took up his cause.
Burzynski also benefited from a shift in the regulatory mood. When thousands of frustrated AIDS and cancer patients besieged Congress, the FDA accelerated the process for approving new drugs and allowed patients broader access to experimental medicines. At the same time, the National Institutes of Health began programs for testing alternative drugs. In 1991, the National Cancer Institute sent six scientists, including its own Patronas, to review a "best-case series" of seven of Burzynski's brain cancer patients. In five of those, Patronas said, radiographs indicated that a large tumor had disappeared after treatment by Burzynski. The NCI offered Burzynski the chance to do free trials. He had only to provide the drugs and approve the experiment's design. By the end of the year, however, the collaboration had dissolved in acrimony, and the NCI halted the research.
By 1997, the FDA's struggle with Burzynski reached the federal criminal courts. That year the Justice Department prosecuted him on behalf of the FDA, charging him with, among other things, mail fraud for illegally shipping his drug across state lines. The trials resulted in acquittal on 35 counts, and the prosecution dropped the other 40.
Still, the FDA got one thing from the court. In 1996, U.S. District Judge Simeon T. Lake III had ordered Burzynski to begin testing his drugs in clinical trials as a condition of his pre-trial release. Burzynski began submitting records for patients already under his care, and he placed new cancer patients in 72 clinical trials.
These trials, if done properly, might finally answer the question of whether antineoplastons really work. Burzynski sent a complete copy of the report he submitted to the FDA to the Cancer Letter, a newsletter for doctors, researchers, and patients. Editor Paul Goldberg sent the data to three nationally known oncologists-the first examination of Burzynski's data by independent experts. Among their conclusions: Burzynski's protocols, or trials, are poorly designed, making data impossible to interpret. "They have all the trappings of protocols," Howard Ozer, director of the Allegheny Cancer Center in Philadelphia, told U.S. News, "but they cannot be analyzed statistically." They also noted that many patients withdrew from the study, even though Burzynski reported that they were responding to treatment. "Usually, people keep taking medicine that's working," says Peter Eisenberg, a Marin County, Calif., oncologist, "unless the side effects outweigh the benefit."
According to the FDA's analysis of the data, the therapy contributed to the deaths of at least seven people through its most common side effect, hypernatremia--a potentially life-threatening condition associated with high levels of sodium in the therapy. The FDA reported that 65 percent of Burzynski's patients had hypernatremia, a finding seemingly at odds with Burzynski's claim on his Web site that his drugs are "normally free from serious side effects." Burzynski contends the patients died of other causes and when there was hypernatremia, it was "due to the fact that the patient wasn't drinking fluids."
Not all of Burzynski's patients are comfortable with his representations. On July 9, the parents of 11-year-old Christina Bedient, of Lockport, N.Y., filed suit against Burzynski and his clinic, claiming the doctors made misrepresentations to them about the efficacy of antineoplastons, "the effect that the treatment was having on Christina's tumor, and about her prognosis." They say they believed the alleged misrepresentations. They also claim that Burzynski and his clinic were negligent and treated their daughter "in a manner that violated the standards of acceptable medical practice." Christina died June 17, 1996. Burzynski says the lawsuit is frivolous. "The bottom line is they signed an informed consent form," he says. He says Christina's "big tumor was decreasing" but that another tumor was increasing. "There was no negligence," he says. "There was no treatment for this child. At the same time, we have patients who respond and whose tumors disappear completely, so there was [a] chance."
Another couple has raised similar complaints. In June 1997, 26-year-old Andrea Walsh, a registered nurse from Jordanville, N.Y., was diagnosed with a glioblastoma, the same cancer Christina Bedient had; after surgery, her doctors said chemotherapy and radiation might extend her life a few months, but no more. That August, Andrea's mother, Jean, and her brother Bill took Andrea to Burzynski's clinic, where they say they were told by a clinic doctor that antineoplastons could cure a third of glioblastomas. The Walshes borrowed $16,000 to start treatment.
High fevers. Over the following six weeks, Jean Walsh says, her daughter suffered side effects ranging from disorientation and high fevers to constant thirst. She and her husband, Tom, repeatedly complained to clinic personnel. Each time, she says, "the nurses were jubilant. They said this [side effect] was a sign the tumor was breaking up." On September 22, an MRI scan showed that Andrea's tumor had doubled in size, says her local neurosurgeon, Frank Boehm. He told the parents that Andrea had very little time left. Still, the parents say, a Burzynski clinic doctor insisted that the young woman come to Houston to be examined or she would have to be dropped from the clinical trial. Burzynski says that the clinic has no record of such a conversation. Andrea left on September 28 in the company of Mary Briggs, her best friend.
After they arrived, according to Briggs and the Walshes, another doctor at the Burzynski clinic told the two women the tumor was dissolving. That doctor called Andrea's parents on September 29, telling them the tumor was shrinking and their daughter would be back to work. "I can't tell you how happy we were," says Jean. She and Tom ran up their credit cards to come up with the $7,000 for the next month's treatment.
Andrea never made it home alive. On October 1 her brain swelled massively, just as her flight home was beginning its descent. Henry Friedman, a neuro-oncologist at Duke University, and Victor Levin, a brain-tumor specialist at MD Anderson Cancer Center in Houston, and Boehm, her neurosurgeon, say she should never have traveled any distance from a hospital emergency room. Counters Burzynski: "It's not up to us; that's up to the local physician." Boehm says the Burzynski clinic never called to ask him whether Andrea was fit to fly.
The patient history in Burzynski's report to the FDA states that Walsh did not die under his care. She is listed as having withdrawn from treatment September 30, two days before she died. But according to the Walshes and Briggs, a nurse, Andrea was still receiving antineoplastons just before she boarded the plane. "If she withdrew, why was she carrying a suitcase full of the medicine?" Tom asks. Burzynski says he listed Walsh as having withdrawn on September 30 because that was the last day she was treated.
The FDA's analysis of Burzynski's records is at odds with Burzynski's interpretations. In a preliminary summary of Burzynski's results released this spring, the agency calculated that the tumors of 36 of Burzynski's 828 patients showed a positive response while on his drugs. The FDA found no apparent effect on breast, prostate, and lung cancer. In brain tumors, the FDA found a 13.5 percent response to antineoplastons--not a 30 percent complete response rate, as Burzynski allegedly told the Walshes and others. (The FDA says that a response rate of 10 percent or less usually means that the drug is not effective.) Burzynski and his lawyer, Richard Jaffe, denounced the FDA for releasing the data and accused the agency of misrepresenting the true figures. In a letter to the Cancer Letter, Burzynski complains that the FDA did not count 72 patients who had "stable disease." That, however, is a measure that other researchers say does not necessarily indicate that the cancer has stopped growing. Burzynski told U.S. News his latest results are even better: Only 5 percent of brain tumors got bigger, while 60 percent disappeared completely or shrank by more than half.
If true, his data should bear that out. Says Thomas Garvey, a physician and consultant hired by Burzynski to help him present his data to the FDA: "The protocols [Burzynski's] are weird. They are naive, they are flawed, and there are a lot of them. But even a ball of worms can be disentangled. That's what we're trying to do."
Such debate over the meaning of his trials signals that cancer patients will remain in the dark as to whether Burzynski's antineoplastons will be judged more effective than other medicines. Some patient advocates are disappointed by what the oncologists found when they reviewed Burzynski's clinical trials for the Cancer Letter. "I am truly aghast because supposedly we have laws and regulations and a legal system in place to stop this from happening," says Fran Visco, president of the National Breast Cancer Coalition. "Resources should be allocated to an appropriate trial run in the right way. While we waste time in these trials, people die."
This story appears in the October 5, 1998 print edition of U.S. News & World Report.