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Stanislaw Burzynski’s propaganda victory on antineoplastons: The FDA caves

Mark Crislip, founder of the Society for Science-Based Medicine, whose board of directors I’m proud to be serving on, an organization that you should join if you haven’t already, sometimes jokes that our logo should be an image of Sisyphus, the king of Ephyra whom Zeus punished by compelling him to roll an immense boulder up a hill. However, the boulder was enchanted and, as soon as Sisyphus reached the top, it would roll back down the hill. Sisyphus was thus forced to repeat this action throughout all eternity. The metaphor is obvious. Those of us who try to combat quackery and the infiltration of pseudoscience in medicine often feel a lot like Sisyphus. I always used to argue that, as amusing as it might be to have such a logo as an “in” joke, it’s far too much of a downer to inspire what SSBM wants to inspire: Action in the form of volunteers taking on projects, such as converting Quackwatch into a wiki and then continuously updating and adding to that wiki indefinitely. We have to believe that there is hope of someday succeeding. “Let’s push that boulder up a hill one more time!” does not exactly constitute an inspiring rally cry, although I can definitely understand the feeling at times the older I get and the longer I’ve been doing this. We can all appreciate gallows humor at times, and, besides, I’m not that pessimistic. I can’t afford to be.

Even so, I can understand the Sisyphus analogy right now with respect to an unfortunately frequent subject of this blog, the doctor in Houston who proclaims himself a cancer doctor, even though he has no formal training in medical oncology, isn’t even board-certified in internal medicine, the prerequisite for undertaking advanced training in medical oncology, and has no discernable training in clinical trials management. I’m referring, of course, to Stanislaw Burzynski, MD, PhD, the Polish doctor who since 1977 has been treating patients with substances that he has dubbed “antineoplastons” (ANPs). What are ANPs? Burzynski claimed to have discovered ANPs during his time at Baylor and described them as endogenous cancer-fighting chemicals in human blood and urine. 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. Meanwhile, the FDA has found numerous examples of Burzynski’s abuse of clinical trials, failure to keep necessary data, and failure to protect human subjects, while exposés by BBC Panorama and Liz Szabo at USA TODAY have been most unflattering, revealing at least one dead child as a result of the toxicity of Burzynski’s drug and a pattern of minimizing and hiding reports of adverse reactions.

I had at least two other ideas for what I thought would be informative, entertaining, and timely posts, but then late last week I found out about a new development in Burzynski’s latest battle to try to use patients as weapons again to bring pressure to bear on the FDA (as he did in the 1990s) to allow patients with brain tumors to be treated with ANPs under single patient INDs, also known as compassionate use exemptions or expanded access programs. Such exemptions allow some patients who have no good conventional options access to unapproved investigational agents. Ironically, one of the most damning findings about the Burzynski Research Institute and Burzynski Clinic from the FDA investigation last year was how Burzynski would play fast and loose with the rules regarding approval by the institutional review board (IRB) of such exemptions. IRBs are committees mandated by the Office for Human Research Protections (OHRP) to protect human research subjects by approving and monitoring clinical trials to make sure that patients are not placed at undue risk, that proper informed consent is given, and that clinical trials conform to all ethical and legal requirements. Let’s just put it this way. The FDA caved. You’ll see what I mean shortly. Worse, it caved in a way that basically abdicated its responsibility.

Those of you who read my not-so-super-secret other blog will realize that I’ve already begun the discussion over there, making part of this post repetitive. However, there have been new developments over the weekend, and I very much want a record of this abdication of duty by the FDA on this blog as well—in more detail, of course.

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

Sadly, the brief little quote from Michael Corleone (as played by Al Pacino) in The Godfather, Part III, basically sums up my feelings with respect to 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 Thursday evening, 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 some 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 naïve move, but it’s a huge mistake regardless, as you’ll see. I’m guessing that some will think of it as a very clever move, and so it is, but it’s a cleverness we could use less of.

About a year ago, Burzynski was once again investigated by the FDA. 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 a 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 misguidedright 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

When I first saw this message on the Prayers for Elisha Facebook page, I was cautious. I could find confirmation of this news nowhere else, not even on the ANP Coalition webpage. Then, on Friday afternoon, Liz Szabo, the USA TODAY reporter who previously published her epic expose of the Burzynski cancer machine back in November, basically confirmed the report with a followup story in USA TODAY, FDA agrees to let patients get controversial drug:

The Food and Drug Administration has agreed to allow a handful of cancer patients to receive unapproved drugs from a controversial Texas doctor, but only if they can find another physician to administer them.

The drugs are made by Houston doctor Stanislaw Burzynski, who was the subject of a USA TODAY investigation last year. While his supporters consider him a medical maverick, mainstream doctors describe him as a snake-oil salesman. Burzynski has claimed for more than 36 years to be able to cure certain hard-to-treat brain tumors with drugs he calls antineoplastons. The National Cancer Institute notes that Burzynski has never published definitive evidence that his drugs cure cancer or even help people live longer.

Burzynski has been unable to give these drugs since 2013, when the FDA placed his experiments on hold after the death of a 6-year-old New Jersey boy taking antineoplastons. In December, the FDA sent Burzynski a warning letter, noting that he inflated his success rates and failed to report side effects and to prevent patients from repeatedly overdosing. The Texas Medical Board also charged Burzynski last year with false advertising.

None of this stopped the FDA from caving:

The FDA acknowledged Friday that it has agreed to allow them to use the experimental drug, but only if they can find a qualified, independent physician to administer the drug. Beyond infusing the drug and overseeing their care, the doctor would have to formally apply for expanded access to an “investigational new drug,” as well as get approval from an institutional review board, an independent panel that reviews safety and ethical issues involved in clinical trials.

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 naïve 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 of FDA regulations, and, above all, the need to follow the FDA’s mandate and mission to protect vulnerable cancer patients, instead the FDA has dangled more false hope of a single-patient IND in front of Elisha’s family (and the eight other children petitioning for such INDs). It’s hard to believe that the FDA is not aware that it is incredibly unlikely that any reputable pediatric oncologist would agree to administer ANPs under these conditions, given Burzynski’s reputation.

In a way, what the FDA is doing inadvertently compounds the families’ agony. Now they have 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 not-inconsiderable amount of work and expense that would be necessary in order 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. Indeed, according to Szabo’s report, Liza Covad-Lauser, wife of rock star Sammy Hagar’s drummer David Lauser, claims that she has already found a doctor willing to administer the antineoplastons. One wonders if this doctor knows what he is getting himself into. As cynical as it might sound of me to say so, I can’t help but wonder whether being the wife of a member of a rock star’s band, who can garner signatures on petitions by other rock stars, has an effect.

Here’s why the FDA’s action is doubly cynical (or nave). As alluded to in Szabo’s article, 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, except for perhaps briefly as an “emergency exemption” in which IRB approval can be briefly deferred:

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 discuss 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 routinely used to treat some of these patients for free, supplying ANPs for no cost and waiving case management fees. No doubt this offer was primarily for publicity, to blunt the accusation that, based on the exorbitant amounts of money charged to previous patients, Burzynski is in it for the money. So, not surprisingly, according to Szabo’s report, Burzynski is still offering to provide the drug for free. That’s all well and good, but it 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? While it is true that 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.

A massive Burzynski propaganda victory?

Supporters of Stanislaw Burzynski wasted no time in using the FDA decision to defend Burzynski and, ironically, to attack the FDA. Many of you might remember McKenzie Lowe, the 12-year-old girl diagnosed with diffuse intrinsic pontine glioma (DIPG) in 2012. Her family has been trying to have her treated by Stanislaw Burzynski, making videos and lobbying Congress. Indeed, they succeeded in persuading their senator, Senator Kelly Ayotte (R-NH), to write to the FDA urging it to consider allowing McKenzie to be treated by Burzynski. On a Facebook page Friends of McKenzie Lowe, a message was posted on Sunday simultaneously rejoicing at the news as evidence that McKenzie has a chance to live and lambasting Liz Szabo:

As you all probably have read in the USA Today the FDA has agreed to let Mckenzie and the other people who want to use ANP CAN USE IT!!!! FANTASTIC!!!!! But as you can see the writer of the article is very bias on the negative side. As usual she one again took something good for these patients and in stead of focusing on the positive response from the FDA she took another swing at bashing Dr. Burzynski and his clinic. There was no mention of or comments from Anthony Stout, Rick Schiff, Jessica Ressel, Randy Hinton, Philip Norton and many many more..

Let’s go back in history to all the nay Sayers that said Christopher Columbus was crazy. The world is flat, you will fall off the end. Hmmm. Einstein was a quack… Hmmm. Isaac Newton and Galileo were also shunned by their peers… Thank god they had the fortitude to push aside the nay sayers and continue to prove them ALL wrong. Imagine where we would ALL be today if they had listened to their critics and not persevered with what they believed.

Unfortunately, this is known as the Galileo Gambit, a nice, concise term used to argue that if you are vilified for your ideas it must mean that you are right. Unfortunately, what supporters of pseudoscientists and quacks frequently forget is that there are two requirements necessary to be able to credibly claim the mantle of Galileo. Not only must you be vilified, but you must be correct. Only one of these applies to Stanislaw Burzynski, and it isn’t the second one.

As has been the case whenever I’ve discussed patients like McKenzie Lowe, Liza Covad-Hauser, Rafael Elisha Cohen, and others, I understand, at least as much as it is possible to understand without actually being in their shoes. As I’ve pointed out before, it was only five years ago that my mother-in-law died of widely-metastatic breast cancer. Burzynski couldn’t have saved her. ANPs couldn’t have saved her. And I know I’m going to take some flak for saying this, but ANPs cannot save McKenzie Lowe. At least, there is no convincing evidence that they can, and, quite frankly, the only blame for why this is true should be placed at the feet of Stanislaw Burzynski himself. He’s the one who, thinking himself the “brave maverick doctor” to whom the rules of science don’t apply, has failed to produce such evidence. While I understand the desperation these families are feeling, I keep hoping that they will realize that it is not doing McKenzie, Elisha, or any of the others any favors to subject them to toxic chemotherapy—and, make no mistake, ANPs are toxic chemotherapy, efforts of advocates to paint them as “natural” and “nontoxic” notwithstanding. As I’ve pointed out time and time again: If there’s anything worse than dying of a terminal illness, it’s dying of a terminal illness and suffering unnecessary complications or pain for no benefit in the process.

I can totally understand why families like the Lowes might become very excited about the possibility of being able to use ANPs. As misled as they have been and mistaken as their belief is, they do, after all, genuinely believe that ANPs represent the last chance their loved ones have to be able to survive their cancer. Unfortunately, our old “friend” and Burzynski’s very own propagandist, Eric Merola, who has produced two fawning “documentaries” about the Great Man chock full of misinformation and exaggerations, coupled with attacks on Burzynski critics and skeptics, has no such excuse and deserves none of the compassion that we should all have for patients dying of brain tumors and the families who love them. Not surprisingly, he, too, has leapt into the fray with a typically frothing-at-the-mouth conspiracy-laden pile of fetid dingos’ kidneys of a response to Liz Szabo’s story, in which he examines it line-by-line and basically responds with typical Burzynski propaganda, misinformation and lies.

There’s too much there for a full response in this post. Besides, most of the misinformation that’s there I’ve covered before, often multiple times. Perhaps I’ll respond a bit more on my not-so-super-secret other blog. Here, I simply want to point out a few of the most egregious examples. For example, here is probably the most egregious lie on Merola’s part:

A DIPG in a child has never been cured in medical history — why would the FDA make such a claim in this case? It’s quite simple, the science proved it works and cures in many cases, and therefore the science was allowed to progress. There are no “claims” other than what can be proven. Maybe it’s because the cancer industry doesn’t like anyone else “claiming” anything as they own the luxury of claiming what cancer medications work or not work — and we know how well that has worked out for us.

As I’ve pointed out before, this is, quite simply, untrue. It’s also been pointed out time and time again to Merola that this is untrue. Long-term survival for patients with DIPG is rare, definitely, but it’s always dangerous to make an absolute statement like the one that Merola has done. Contrary to Burzynski’s claim, there are occasional long-term survivors, some of whom received little or no therapy, such Connor Frankenberg, a child from Germany, and two patients who underwent spontaneous remission. There is even a site, the DIPG Registry, whose purpose is reporting on how patients with DIPG do and promoting Just One More Day, a registered non-profit 501(c)(3) organization dedicated to helping families affected by a diffuse intrinsic pontine glioma, established by the families of DIPG victims. The DIPG Registry concludes that, although the vast majority of children with DIPGs do poorly:

The bottom line is: 1) we really don’t understand DIPGs, and 2) there are children who have been diagnosed with DIPGs-based on the incomplete knowledge that we currently have- who truly have had spontaneous regression of their tumors and 3) most of the children with reports of spontaneous regression have been very young.

Young like Tori Moreno, I might add.

The big problem is that Burzynski appears not to keep good enough records to demonstrate one way or the other whether he can cure DIPG, and we already know from recent FDA findings and Liz Szabo’s USA TODAY report that he has misclassified partial and complete responses. Yes, DIPG is a horrible disease, with only 10% surviving beyond two years. Unfortunately, there is no convincing evidence that Burzynski can do any better than conventional oncology, much less cure DIPG, as much as his propagandist Eric Merola repeats the claim that he can. Worse, Burzynski hasn’t published a single one of his completed phase II clinical trials, even though he’s had well over 15 years to complete them. With tumors this lethal, most such trials should be completed within five years, which brings us to more of Merola’s misinformation:

The National Cancer Institute notes that Burzynski has never published definitive evidence that his drugs cure cancer or even help people live longer.

The above links are examples definitive published evidence. Notice how this writer links to a PDF highlighting her point, vs. the National Cancer Institute (NCI) website itself. Is it perhaps because on the NCI website it clearly says:

“A phase II study also conducted by the developer [Burzynski] and his associates at his clinic reported on 12 patients with recurrent and diffuse intrinsic brain stem glioma. Of the ten patients who were evaluable, two achieved complete tumor response, three had partial tumor response, three had stable disease, and two had progressive disease. Patients ranged in age from 4 to 29 years.”

The NCI felt its own source to be “definitive enough”, why not Liz?

*Source:
http://www.cancer.gov/cancertopics/pdq/cam/antineoplastons/healthprofessional/page5#Reference5.12

Merola clearly does not understand the difference between “responses” and cures. Similarly, he does not understand what constitutes “definitive evidence.” (Hint: It isn’t case reports, tiny case series published in bottom-feeding journals, or incomplete phase II trials. Moreover, just because the NCI cited a crappy review article by Burzynski that reported partial results phase II trials, a ten year old partial report of a phase II trial, a dubious paper published in an integrative medicine journal, and another review article published by Burzynski in a journal that appears not to be even indexed by PubMed does not constitute “definitive evidence” that ANPs work. All it means is that the NCI was examining the existing published evidence on its way to writing this:

To date, no randomized controlled trials examining the use of antineoplastons in patients with cancer have been reported in the literature. Existing published data have taken the form of case reports or series, phase I clinical trials, and phase II clinical trials, conducted mainly by the developer of the therapy and his associates. While these publications have reported successful remissions with the use of antineoplastons, other investigators have been unable to duplicate these results [10] and suggest that interpreting effects of antineoplaston treatment in patients with recurrent gliomas may be confounded by pre-antineoplaston treatment and imaging artifacts.[11,14,16] Reports originating from Japan on the effect of antineoplaston treatment on brain and other types of tumors have been mixed, and in some Japanese studies the specific antineoplastons used are not named.[9] In many of the reported studies, several or all patients received concurrent or recent radiation therapy, chemotherapy, or both, confounding interpretability.

Mr. Merola, that’s hardly “definitive”! Nor is the “definitive” study by Hidaeki Tsuda featured in the second Burzynski movie, given that nothing has been published to allow us to evaluate the methods. Nor is the fact that The Lancet Oncology apparently editorially rejected a manuscript submitted by Burzynski evidence of any sort of “conspiracy” against him. Indeed, I now probably know why Burzynski’s manuscript was rejected. Look at the title: “Glioblastoma multiforme: A report of long-term progression-free and overall survival of 8 to over 16 years after antineoplaston therapy and a review of the literature”. Yes, the title seems to indicate that this is just another small case series. No wonder The Lancet Oncology didn’t even bother to send it out for review!

Of course, Merola can’t resist a swipe at yours truly, who was quoted in the article:

A guy who is paid to write blogs attacking this innovation and other innovations that compete with the status quo, a guy who has never once met a Burzynski patient, never once visited the Burzynski clinic, a guy who supposedly finished his residency and now writes “thought police” blogs for a living — yeah, let’s hear more about what this guy has to say, that’s productive. He does fit the angle for Liz’s one-sided propaganda, so I understand why she included his irrelevant opinion.

I don’t know whether to laugh or be annoyed at Merola’s continued lies about me. Once again, I do not need to visit the Burzynski Clinic to know that ANPs almost certainly don’t work. I did finish my residency and am board certified in surgery. If Mr. Merola doesn’t believe that, he can go to the American Board of Surgery website and search for my name in the section that lets the public verify the board certification of surgeons, realizing that you can’t become board certified without finishing an accredited residency. Finally, I don’t actually blog for a living. It’s my hobby. I’m not paid at all for my work at SBM, and at my not-so-super-secret other blog I make a small amount of money that I could completely do without if that gig ever went south or I decided to bolt. As for having something worthwhile to say, well, let’s just say that I’d put my record for science-based analysis against Mr. Merola’s record for pseudoscience-based propaganda any day, particularly in light of his latest paean to another brave maverick, this time promoting the long-discredited cancer quackery known as laetrile.

At least Merola included a link to my page on SBM. He seems to think it’s something nefarious, while I’m more than happy to include a link to it here. Please, read.

Why did the FDA cave?

What is puzzling to me is why the FDA caved. While it is true that the ANP Coalition managed to get a few misguided legislators, the most prominent of whom was Senator Kelly Ayotte (R-NH), to write letters to the FDA urging that it consider allowing ANPs to be made available through single patient IND/expanded access to their constituents, the letters were fairly wishy-washy. Moreover, the Change.gov petition to the White House response to a We The People online petition to have the FDA allow Elisha Cohen access to ANPs through compassionate use exemption resulted in a response that, boiled down to it essence, said that it’s the FDA’s job to make such decisions, and let the FDA do its job, along with links to various FDA web pages with information about investigational drugs and information about how the FDA could be contacted. It was a shockingly reasonable response.

One rationale reported in Szabo’s article seems to be that by allowing ANPs to be used but excluding Burzynski from having anything to do with administering them other than producing them, in essence, relegating him to the role of a drug manufacturer, useful data might be obtained and patients protected while obtaining that useful data. This is utter nonsense. As Howard Ozer is quoted in Szabo’s article:

“This is a cop-out,” says Ozer, director of the University of Illinois Cancer Center, who studied Burzynski’s research results in the 1990s.

“Because it can be toxic and cause life-threatening sodium problems, patients (who take antineoplastons) are at risk and could die,” Ozer says.

The scientific community will learn nothing by giving these patients antineoplastons, because there will be no comparison group, Ozer says. Any doctor who agrees to give antineoplastons could be biased in favor of them, which could skew the results.

Exactly. As Dr. Peter Adamson points out in the article, after 37 years, if antineoplastons were the wonder drugs that Burzynski claims them to be, scientists would know it by now. ANPs aren’t wonder drugs. There isn’t even any evidence that they’re better than standard-of-care, as dismal as that is for DIPG.

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, which is why I rather suspect that Dr. Henry Friedman is right when he says:

“It will deflect criticism from anybody who says the FDA is unilaterally denying dying patients something that could help them,” says Friedman, deputy director of the Preston Robert Tisch Brain Tumor Center at Duke University in North Carolina, who also reviewed Burzynski’s studies in the 1990s. “It will get FDA off the hook.”

That, I believe, is exactly why the FDA did it, not to help dying children. Unfortunately, the consequences of the FDA’s decision are likely to be disastrous. 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. The families don’t know it yet, but what the FDA has done will only add to their misery. Indeed, I’ve already seen reports that patients have contacted “dozens” of pediatric oncologists looking for one who will administer ANPs, and, given that the number of pediatric oncologists in the US is small, a significant proportion of them are likely to be contacted.

Meanwhile, Burzynski gets a propaganda victory, thanks to the FDA’s failure to enforce its own standards, and alt-med proponents get another conspiracy theory to add to the list of conspiracy theories surrounding Burzynski already.

Posted in: Cancer, Clinical Trials, Politics and Regulation

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