FDA issues warning to controversial Houston cancer doctor

Liz Szabo | USA TODAY

The Food and Drug Administration has issued a strongly worded warning letter to a controversial Houston physician, Stanislaw Burzynski, who was the subject of a USA TODAY investigation last month.

Burzynski – hailed as a maverick by his fans but a snake oil salesman by mainstream doctors -- has long claimed to have dramatic success in hard-to-treat cancers, especially brainstem tumors that are usually considered fatal. According to the National Cancer Institute, however, Burzynski has never produced definitive proof that his drugs save lives by publishing a randomized, controlled trial in a peer-reviewed journal.

In letters to Burzynski and his research institute posted online Wednesday, the FDA says that Burzynski inflated success rates for experimental drugs that he calls antineoplastons. The FDA also says Burzynski failed to report side effects and to prevent patients from repeatedly overdosing.

The FDA placed Burzynski's clinical trial on hold last year after the death of a 6-year-old boy, Josia Cotto, of Linden, N.J. The FDA also conducted several months of inspections of Burzynski's research.

But when the FDA asked to see the child's medical files, Burzynski sent the agency records that were different than those stored in his office, giving the appearance that the records had been altered, according to the warning.

Burzynski's failure to keep accurate patient records "raises concerns about subject safety and data integrity, as well as concerns about the adequacy of safeguards in place at your site to protect patients."

If Burzynski fails to adequately address the FDA's concerns, the agency could terminate his clinical trial; disqualify Burzynski from conducting future FDA research; issue a civil fine; or pursue criminal charges, according to FDA regulations. Burzynski has 15 days to respond to the FDA.

Burzynski's attorney, Richard Jaffe, said his client is preparing an official response to the FDA. "Until that is done, we have no further comment," Jaffe said.

The FDA found nine cases in which Burzynski misclassified patients as benefiting from his medications -- by claiming that their tumors shrank, disappeared or stayed the same size. In reality, the FDA said, these patients either didn't respond as Burzynski claims, or were taking anti-inflammatory drugs that could have made them appear to shrink. In one case cited by the FDA, Burzynski listed a patient as having a "partial response" to treatment – meaning that the patient's tumors shrank in half – even though the tumor actually got bigger.

In a written response to FDA inspections sent to the agency in April, Burzynski had argued that patients with brain tumors often need anti-inflammatory drugs, called corticosteroids, to restore normal levels in the body. The FDA did not accept this explanation, however, noting that Burzynski had patients on steroid doses that were five to 64 times higher than the levels that doctors would expect patients to need.

According to the FDA, these issues raise doubts about the "validity and integrity of the data collected at your site."

A handful of patients have petitioned the FDA, asking that the agency take Burzynski's trial off hold so that they can be treated with antineoplastons, which have never been FDA-approved.

Burzynski has been treating patients with antineoplastons since 1977, and has worked under the auspices of an FDA clinical trial since 1996.

Given the concerns about patient safety and the accuracy of Burzynski's data, the FDA should shut his trial down now, said neuro-oncologist Henry Friedman, deputy director of the Preston Robert Tisch Brain Tumor Center at Duke University.

"After all these years, with all these claims," Friedman said, "we still have no proof."