Stanislaw Burzynski and the cynical use of cancer patients as shields and weapons against the FDA: Has the FDA caved?

“Just when I thought I was out, they pull me back in.”

I know I’ve used that quote before several times over the 9+ years that I’ve been blogging. These days, I probably use it most frequently when it comes to the topic of Stanislaw Burzynski. Every time I think that I can give the topic a rest for a while (and, believe me, I do want to give it a rest), something invariably seems to happen to pull me back in. So it was yesterday when i was made aware of a new development so disappointing that I’m still wiping the dirt off my chin from my jaw dropping to the floor. Even more amazing is that my disappointment stems from my perception of cynicism that actually was not Burzynski’s, but rather by the FDA in response to Burzynski’s cynical use of cancer patients as shields and weapons against the FDA that has led those of us who support science-based medicine to be subjected to criticism from the families of patients with cancer, who have been erroneously convinced that Stanislaw Burzynski is their loved ones’ last chance to survive. Actually, I can’t tell if it’s a cynical move or a breathtakingly naive move, but it’s a huge mistake regardless, as you’ll see. I’m guessing that some of you will think of it as a very clever move, and so it is, but it’s a cleverness we could use less of.

For those of you not familiar (I know regular readers are and that my summaries in each post about Burzynski can get repetitive, but I live in hope that new readers will read this and be informed about what Stanislaw Burzynski is really all about), Stanislaw Burzynski is a self-proclaimed cancer doctor in Houston—he has no training in oncology and hasn’t even passed his boards in internal medicine—who in the 1970s claimed to have discovered endogenous cancer fighting chemicals in human blood and urine that he dubbed “antineoplastons” (ANPs) while working as a researcher at Baylor. Unfortunately, he soon became convinced that only he could develop them into an effective chemotherapy drug and left Baylor to administer ANPs to his own cancer patients. Patients flocked to him because he claimed to be able to cure cancers that conventional medicine can’t cure. This led to a series of battles between Burzynski and various authorities, including the Texas Medical Board, the FDA, and various attorneys general, because of his use of ANPs, which are not and never have been FDA approved, as well as for various—shall we say?—issues with insurance companies. Ultimately, in the 1990s Burzynski beat the rap and effectively neutered the FDA’s case against him by submitting dozens of clinical trials to the FDA for approval, which, given how much pressure the FDA was under from Burzynski’s friends in high places (like Texas Representative Joe Barton), the FDA ended up approving. However, as Burzynski’s lawyer himself bragged, these clinical trials were shams designed to allow Burzynski to keep treating cancer patients, not clinical trials designed to produce any real evidence of efficacy. Not surprisingly, although Burzynski has published the odd case report or tiny case series, he has not yet published the full results of even a single one of his many phase II trials. There is, quite simply, no convincing evidence that ANPs have significant antitumor activity in vivo in humans, even after 37 years.

Most recently, Burzynski was once again investigated by the FDA about a year ago. Because of the death of a patient due to hypernatremia (elevated sodium level) due to ANPs in 2012, the FDA put a partial clinical hold on his pediatric clinical trials and extended it to all of his clinical trials. What this means is that, although Burzynski can continue to treat patients who are already in his clinical trials, he can’t enroll any new patients in them. Of course, one of the big issues with Burzynski that contributed to his problems is that he abused the FDA process known as the single patient IND, which is more commonly known as the compassionate use exemption. These are special case exemptions in which a single patient is allowed access to an investigational agent outside the auspices of the clinical trial. Indeed, when the FDA slapped him down most recently, in December, one of its findings was just that: Burzynski abused the single patient IND process and his institutional review board (IRB), the committee that’s supposed to oversee clinical trials and safeguard the interests and safety of clinical trial subjects, played fast and loose with the rules.

Since the appearance of a USA Today story by Liz Szabo spelling out all of these issues, Burzynski has been frantically trying to save his empire. Whether “encouraged” by Burzynski or just by one of the patient groups that have become true believers, patients with deadly cancer have been starting petitions, pressuring legislators to intercede on their behalf to the FDA, and paint Burzynski’s ANPs as their only hope of survival. I have no doubt that these patients believe it, but they have the potential to inadvertently do great mischief to the law, either through their use of their tragic stories to promote misguided “right to try” laws or through their use of patient stories to pressure the FDA to loosen protections on human subjects in clinical trials. Perhaps the most tragic story is that of Rafael Elisha Cohen, a six-year-old child with medullablastoma who is not doing well and whose parents have been at the forefront of pressuring the FDA. As horrible as the Cohens’ situation is—and we all understand that it’s a horrible, horrible thing to have a child dying of brain cancer—unfortunately, the FDA appears to have decided to cave. The other day, on a Facebook page dedicated to raising money and pressuring the FDA, Devorah Teicher Cohen announced:

UPDATE-

Thanks to everyones hard work the FDA did end up approving the Antineoplastins for compassionate use – they do not let Dr B administer it.

The condition is, if an oncologist request this protocol FDA will issue compassionate use within 24 Hours.

FDA recognizes the data of those who survived aggressive brain tumors due to the ANP and is therefor after a long and cruel wasting TIME period allowing it. There are currently 8 pediatric patients that need this medicine NOW!!!!

Refael Elisha can not travel at this point…so we would need an oncologist in the NY area… we are in touch with other kids that are ready to get on a plain if they get the signal of a cooperating oncologist.

It is beyond words to express our sadness that FDA ended approving the drug but Elisha is in critical condition at the moment.

We are not holding our breath and are trying different alternatives , If HASHEM wills it we will get the medicine but if not, that is from Hashem for the good. in the meantime Please put your fillers out there and message me in privet if you have any leads , even if not in NY it might work out for the other desperate children.

im copying the announcement from ANP site

Wanted: One brave courageous oncologist or oncologists willing to examine and treat up to eight pediatric brain cancer patients who have run out of treatment options and now require a Compassionate Use IND Emergency Treatment protocol from the U.S. Food and Drug Administration. The ANP Coalition needs your help now. Please visit www.anpcoalition.org or call (925) 699-9116. Thank You

Can anyone figure out why I can’t make up my mind whether this decision, if what the Cohens say is true, is stunningly cynical or stunningly naive on the part of the FDA? Instead of standing firm for science and gently continuing to point out that it can’t allow any more single patient INDs for ANPs be approved based on science, Burzynski’s past violations, and the need to protect vulnerable cancer patients, instead the FDA has apparently dangled the false hope of a single-patient IND in front of Elisha’s family (and the eight other children petitioning for such INDs), likely knowing full well that it is incredibly unlikely that any reputable pediatric oncologist would agree to administer ANPs, given Burzynski’s reputation. In a way, what the FDA is doing inadvertently compounds the family’s agony. Now they have false hope again, except that this time it’s doubly false: False because, based on existing evidence and what we know now, ANPs almost certainly don’t work and false because of how incredibly unlikely it is that any pediatric oncologist will take on such a patient and administer ANPs, given Burzynski’s history, the utter lack of supporting science for the efficacy of ANPs, and the amount of work and expense that would be necessary to do so. The families will now be showing up in the offices of pediatric oncologists, begging them to agree to do a single patient IND and administer ANPs to these patients. Ditto some adult oncologists, who will have patients like Liza Covad pressuring them to do the same.

Here’s why the FDA’s action is doubly cynical (or naive). According to FDA regulations, there are a number of requirements for a single patient IND and several things any physician seeking to administer an investigational agent as a single patient IND must do. Here’s the most important one that makes it even more unlikely that any single patient IND will be granted:

5. Informed Consent Statement that states that informed consent and approval of the use by an appropriate Institutional Review Board (IRB) will be obtained prior to initiating treatment. In the case of an emergency, treatment may begin without prior IRB approval, provided the IRB is notified of the emergency treatment within 5 working days of treatment.

See what I mean? Even if a family were to find an oncologist willing to jump through all the hoops to get a patient on a single patient IND to be treated with ANPs, that single patient IND would still have to be approved by the IRB of the institution where that oncologist practices. Sure, the oncologist could declare the treatment an “emergency”—although in none of the cases that I’m aware of other than that of Elisha Cohen could be properly termed an emergency so urgent that the physician couldn’t wait a week or two to have the IRB formally approve the IND, even if ANPs actually did have any efficacy—and start treatment pending IRB approval, but as soon as the IRB convened to discuss the IND, very likely the plug would be pulled. No more ANPs. Remember, these would be real IRBs, not Stanislaw Burzynski’s sham IRB chaired by an old crony of his.

There’s another question to consider. Stanislaw Burzynski offered to treat some of these patients for free, supplying free ANPs and treating them for free. Will he still do so for them if another physician is treating the patient? My guess is that Burzynski will still send the drug free of charge, as he’s been trying very hard to use such offers for publicity and to “prove” that he isn’t such a greedy guy after all. However, that leaves all the other expenses of treatment, which will not be paid for by insurance because ANPs are not an FDA-approved drug. Who’s going to pay for all of that? Sure, some of the families have raised quite a bit of money. Elisha’s family, for instance, has raised nearly $150,000. Others aren’t so lucky.

Of course, one thing that needs to be emphasized is that this Facebook post is the only evidence out there that the FDA has caved and decided to allow single patient INDs. It is possible that one of the Senators or other legislators did indeed pressure the FDA, leading the relevant enforcement officials to come up with this decision in order to get them off their backs without actually doing anything. They could credibly say, “Hey, we offered compassionate use, but I’m sure you’ll agree that, given his history, we can’t let Burzynski be the one to administer the ANPs on compassionate use. Look at his history of playing fast and loose with IRBs and not obeying the rules with respect to INDs.” This might even sound convincing and seem clever, but, if the FDA really has done this, its consequences are likely to be disastrous. The long-suffering families will have false hope extended to them once again, only to find out that it’s a useless offer. In addition to continuing to scramble to raise money, they’ll scramble to find oncologists willing to do the not inconsiderable work to get a single patient IND approved and administer ANPs, something few, if any, of them are likely to succeed at doing. The families don’t know it yet, but what the FDA has done will only add to their misery. Meanwhile, Burzynski gets a propaganda victory.

If what the Cohens have announced is true, I don’t know what the hell the FDA was thinking.