Two Stanislaw Burzynski's "success stories"

One of the strategies that Stanislaw Burzynski will undoubtedly use to "prove" in Eric Merola's new Stanislaw Burzynski movie that antineoplastons work in cancer will be to highlight "success stories." Last year, Burzynski apologists frequently pointed to a girl with an inoperable brain tumor named Amelia Saunders as a success story when the U.K. press widely featured her going to school in September but, very sadly, her family saw her tumor begin to progress again in December, ultimately resulting in her death about a month and a half ago. In the process, Burzynski did what he all too often does and misinterpreted her MRI, in which cystic structures commonly seen in Amelia's form of cancer as it progresses were seen, as evidence that her tumor was regressing. They were not.

So let's take a look at two cases frequently pointed out to me as Burzynski success stories: Laura Hymas (whose website is Hope for Laura) and the aforementioned Hannah Bradley (whose website is Team Hannah). On the surface to those not familiar with cancer they do look like success stories. If one digs deeper, the true story is a lot murkier. More importantly, as I will show, even if they really are success stories—which is not at all clear—they do not constitute convincing evidence of the efficacy of Burzynski's antineoplastons for cancer patients in general, nor do they justify what I consider to be Burzynski's highly unethical behavior.

Go Team Hannah!

I will start with Hannah Bradley's story because I've watched the entire 40 minute video Hannah's Anecdote. The documentary ends triumphantly several months after the events portrayed during the bulk of the film with Hannah apparently having had a complete response to Burzynski's antineoplaston therapy:

Let me just first say something before I begin my usual analysis. I love these two. I really do. They are the cutest couple, and their love for each other oozes from the video and envelopes the viewer, sucking him in like The Blob, but in a good way. The same is true of every video of them I've watched on their video blog. Yes, it's true that Hannah Bradley's partner Pete Cohen can be a bit cloying and annoying at times with his seeming desire to videotape everything, but I want Pete and Hannah to be able to live a long and full life together, growing old in each other's company. I really do. In fact, I'd love to hang with these two and maybe buy them a pint or two at their local pub (except that it's pointed out multiple times that Hannah can no longer drink alcohol). Sadly, this is unlikely ever to pass even if Hannah does completely beat her disease and is still around the next time I make it to London (which is likely to be at least a year or two), because I fear that if they see this (which is likely) they will misinterpret my analysis as trying to destroy their hope. Certainly the families of other cancer patients who have gone the "alternative" route, be it Burzynski's treatment or other "alternative" cancer therapy, have attacked me in that way. Such is not my intent, but what are skeptics supposed to do? Shy away from undertaking a dispassionate analysis of patient anecdotes used to promote dubious cancer therapies for fear of what patients will say?

After talking about how he needed to raise £200,000 in order to take Hannah Bradley to the Burzynski Clinic for what he characterizes as "life-saving" treatment, a campaign that produced media coverage in the form of articles with titles like 'I'll try anything to beat brain cancer', Pete Cohen describes Hannah's diagnosis, and elsewhere we find out that Hannah underwent awake brain surgery as the surgeons tried to remove all the tumor:

I first met Hannah in April 2010 and we fell in love and since then our relationship has gone from strength to strength. Hannah is 28, has a great personality and has a fantastic sense of humour. Our world took a dramatic turn in February 2011 when, out of the blue, Hannah had a major seizure in the middle of the night. She lost consciousness and was rushed to hospital.

Hannah does not remember much about the two months that followed as she had constant headaches and a number of seizures. She was diagnosed with a very serious brain tumour called Anaplastic Astrocytoma. Hannah decided to have surgery and the 1st of April 2011 and underwent a six and a half hour operation. She was awake for nearly three hours of this operation.

The operation was a success and they managed to remove nearly all of the tumour. We had to wait for the results of the biopsy for a few weeks and we remained positive. However, the news was not good and our world was rocked once more as the results showed a Grade III tumour. Hannah’s bravery and resolve once again rose up as shortly after this she started a six week course of radiotherapy. This went well for the first few weeks but was followed by Hannah’s hair falling out and bouts of tiredness and lethargy.

On top of all of this, Hannah has been dealing with losing her driving licence as she has had a number of seizures and now has epilepsy.

Six weeks after the radiotherapy finished, Hannah had another MRI to see what was going on with the tumour, Once again more bad news, as there were still remnants of this aggressive tumour.

Hannah’s treatment options are very limited and her life expectancy is for this type of tumour is normally around 18 months and this is why I started a mission to find people who had the same condition and are still alive today. I managed to track down a number of these people to speak to them.

In his movie, Pete points out that these people all led back to Burzynski. Of course, as I've said before, dead patients don't produce testimonials for alternative cancer cures. More importantly, Burzynski has a network of true believers, such as the Burzynski Patient Group and a filmmaker like Eric Merola, all of whom actively promote the Burzynski Clinic online. Add to that Suzanne Somers, who included Burzynski as one of her "doctors who are curing cancer" in her book Knockout: Interviews with Doctors Who Are Curing Cancer and How To Avoid Getting It in the First Place. Indeed, Chapter 7 is all about Burzynski, whom Somers describes as having invented "the most important and successful non-FDA-approved alternative cancer drug therapy ever in this country."

In any case, that was the end of July 2011. By November 2011, Pete had raised £35,000, which was enough to go to Houston, and that's where his documentary begins, as he and Hannah are preparing to fly to Houston in December 2011 to have a consultation at the Burzynski Clinic. These are interspersed with footage from earlier in which the couple document their quest, talk about how they were warned by their oncologists and other doctors not to waste their money and effort, and talk about their hopes. Not long after they appear at the Burzynski Clinic, they meet with doctors there who tell them that Hannah's most recent MRI scan showed progression of her tumor (around 8:30 in the movie). Now, I'm not a radiologist, much less a neuroradiologist, but I wondered at all the enhancement on the superficial area of the brain, just under where her neurosurgeon must have raised the bone flap to remove what he could of the tumor. One wonders if much of the remaining enhancement could be still post-surgical and post-radiation change. Certainly, the tumor is cystic-appearing, and after surgery such cysts would likely shrink and be reabsorbed even if the tumor were to keep growing.

Be that as it may, there were a number of things I found very interesting in this video. First, one notes that patients don't see the great Dr. Burzynski right away. Underlings see them first and commence treatment, assuring the patient that Dr. Burzynski is aware of what they are planning. Second, I'm not particularly impressed with the sterile technique used for putting her Hickman catheter in, nor am I particularly impressed with the sterile technique used to access it by the nurses, who appear to be rather inconsistent about wearing gloves and a mask when accessing the line.

As important as sterile technique is when inserting and accessing long-term indwelling catheters, these complaints are quibbles compared to what this video shows about antineoplaston therapy. First, I notice that nowhere was there anything mentioned about enrolling Hannah on a clinical trial. Remember, part of the consent agreement with the Texas Attorney General back in the late 1990s stated that Burzynski can distribute "antineoplastons" only to patients enrolled in FDA-approved clinical trials, unless or until the FDA approves his drugs for sale. Given what a thorough videographer Pete Cohen obviously is, I find this omission very curious. Certainly, given how much detail he's used in this video and in his vlogs I'd expect that if the subject of clinical trials was mentioned he would have included it.

The other thing that struck me was just how much Burzynski is full of it when he advertises antineoplastons as not being chemotherapy and, more importantly, as being nontoxic. At least a third of the video consisted of the difficulties that Hannah had with her treatment, including high fevers, a trip to the emergency room, and multiple times when the antineoplaston treatment was stopped. She routinely developed fevers to 102° F, and in one scene her fever reached 103.9° F. She felt miserable, nauseated and weak. I've seen chemotherapy patients suffer less. I was also very puzzled at how the Burzynski Clinic could allow a cancer patient to linger with a fever of 102° F and sometimes higher, accompanied by shaking chills, in a temporary lodging without admitting her to the hospital. It's not clear what sort of workup was done to evaluate Hannah either, what her white blood cell count was, or what her other labs were. Did they draw blood cultures? Did they get urinalyses and cultures? Did they do chest X-rays to rule out pneumonia? It's all very unclear, other than that she apparently was given some antibiotics at some point. Did she have the flu, given her flu-like symptoms, or was this due to her antineoplaston therapy? The reaction of the clinic staff (i.e., rather blasé, even though at one point Hannah clearly demonstrates a change in mental status, appearing "drunk" and complaining of double-vision) made me wonder if this sort of problem was a common occurrence. At another point, Pete and Hannah come to believe that the fevers might have been due to the tumor breaking down, which strikes me as implausible. Later, she develops an extensive rash. It's difficult to tell for sure what it is at the resolution of the video, but it looks like erythema multiforme, which is generally an allergic rash. What's the most likely cause of such a rash? Guess. Erythema multiforme is usually a drug reaction.

Near the end of the video, we see a series of MRIs. Hannah and Pete are told that the first MRI, done about a month after the antineoplaston therapy started, shows that the tumor has decreased in enhancement and size, with a decrease of about 10%. I don't know who this person is at the Houston Medical Imaging facility just up the road from the Burzynski Clinic is, but from what I could see it isn't clear at all that the tumor decreased in size by 10%. In fact, it's rather hard to tell if a tumor has decreased in size at all. With a tumor that size detecting a 10% decrease in size is almost within the margin of error of the test. Even Burzynski didn't try to sell this as a true decrease; he called it stable disease, which is what it was:

However, later in the video, there are more convincing MRIs. Indeed, the MRI dated July 29, 2012 looks quite good, with little or no enhancement left. The question, of course, is: Does this mean that Burzynski's antineoplaston treatment worked for Hannah? Sadly, the answer is: Not necessarily. It might have. It might not have. Why do I say this? First, she didn't have much residual disease after surgery and radiotherapy, and in fact it's hard to tell how much is tumor and how much is postop and radiation effect. Second, the median survival for anaplastic astrocytoma (which is a form of glioma) is around 2 to 3 years, and with different types of radiation therapy five year survival is around 15% or even higher. Thus, long term survival for patients with astrocytomas is not so rare that Hannah's survival is so unlikely that the most reasonable assumption has to be that it was Burzynski's treatment that saved her. More likely, Hannah is a fortunate outlier, although it's hard for me to say even that because, at only two years out from her initial diagnosis, she's only just reached the lower end of the range of reported median survival times for her disease.

I'm also very worried about Hannah. Her last couple of vlogs are the reason. For instance, in her vlog of December 2, 2012, in marked contrast to past vlogs, Pete is noticeably evasive when discussing her latest scan, and both Pete and Hannah appear uncomfortable:

Even more worrisome was a cryptic Facebook post from November 1, 2012:

It's been a long time coming but here is our new Team Hannah Blog. I am sorry we don't have the best of news but I am ok. http://www.teamhannah.com/2012/11/01/team-hannah-blog-011112/

The vlog entry referenced no longer exists. I have, however, managed to obtain a copy, thanks to a reader, and it's very, very worrisome. By all indications from that vlog, it sure sounds as though Hannah did have a recurrence. Hannah and Pete describe two scans after Hannah’s late August scan (the one that they posted on Hannah’s Facebook page), the first of which apparently showed a mass (although apparently Burzynski told them they couldn’t tell if it was scar tissue or tumor). Per Pete and Hannah, the Burzynski Clinic upped the dose of antineoplastons, which strongly implies to me that Burzynski thought it was tumor, but apparently by the second scan near the end of October the tumor hadn’t regressed. Hannah and Pete apologize for having taken more than two months between vlogs, saying that they didn’t want to give bad news in their first vlog after having released “Hannah’s Anecdote,” given that the first scan that showed probable tumor recurrence. They decided to wait until the second scan to do a vlog because they expected the second scan to show regression of disease again (i.e., good news to report). Depressingly, it appears not to have shown that.

Then there was Hannah and Pete's Christmas message. Now comes this latest vlog, dated March 2, 2013:

I'm more worried about Hannah than ever. Indeed, I almost fear to say what I feel obligated to say, because no matter how I do it, it's likely to be spun, as Merola's movie clearly has done to other skeptics with his ham-fisted shots of cancer patients crying about how skeptics are "attacking" them, as an "attack" on Hannah and Pete or as trying to rob them of hope. I also realize that there is a fine line between trying to use a case like Hannah Bradley's as a means of educating the public about ineffective cancer "cures" and coming off as attacking someone with a serious cancer. I try very hard not to cross that line, and I think I've been successful. However, if there's one thing I've learned during all these years, it's that if the person I'm discussing finds out about my post, that person always perceives it as an attack and lashes out. Similarly, Burzynski's groupies realize that it is very effective to appeal to emotions and cast Burzynski's critics as heartless villains so in the thrall of big pharma, ideology, or whatever that they will viciously rip into a patient whose life is threatened with a deadly cancer. Personally, I'd be very happy for Hannah and Pete if she were to survive to outlive me, whether it were due to conventional therapy, Burzynski's therapy, or a combination of the two. I hate seeing people die of cancer, which is one big reason I became a surgical oncologist in the first place. And if it were Burzynski's antineoplastons that did the trick, I'd be both happy at the discovery and furious at Burzynski for jerking people around for so many decades instead of doing the work it takes to prove the value of his treatment.

So why am I even more worried than ever about Hannah? It started right from the first shot of her latest vlog. Look at her face. It's subtle, but, I think real: an increased asymmetry in which her lower lip on the left part of her face is droopier than I had ever noticed before. I first noticed this in her Christmas vlog but it appears worse now, particularly in comparison to her in the 40 minute Hannah's Anecdote, which included lots of shots of her from December 2011 and January 2012. In those shots, she appeared to have had a bit of this going on then, but it was quite subtle. It's not so subtle anymore in her latest vlog and suggests that the tumor might be growing and impinging on nerves going to her face. She also struggles for words in a way that I don't recall ever having seen her do before. At one point, she has a hard time thinking of the word "risk" and has to be prompted by Pete. All of this makes me wonder if she's developed an anomic aphasia. I could be wrong about Hannah's facial asymmetry; I'm not a neurologist. I'm also less sure about the aphasia; it could well be that she simply had a bit of difficulty finding a word, no worse than any other person. In fact, I'd like to be wrong about this (and any neurologists and/or neurosurgeons out there reading tell me if I'm wrong about this—I'll tack on an addendum if you convince me). But I don't think I am, at least not about the facial asymmetry.

Next, Hannah admits that she has a "really cystic area in my head" but insists that there's no enhancing tumor and doesn't really say if what is there is increasing in size or stable. Given her affect and the expression on her face when she discusses these issues, I truly fear that it must be growing. There's also the issue of the marked change in how she and Pete discuss her scans. In Hannah's Anecdote and their vlog of July 27, 2012, Hannah and Pete exult (and understandably so!) that Hannah has had a "complete response." Now, "complete response" means just that: a complete response to therapy. No tumor detectable by MRI. That implies that anything seen on imaging now must have arisen between the scan of last August and now. Presumably, it arose between August and November, the time when she and Pete first noted that the news was "not the best of news." Shades of Amelia Saunders. Hers was a truly heartbreaking story, not the least of which because of the way that Stanislaw Burzynski appears to have strung the family along. I fear that he is doing the same with Pete and Hannah, who don't deserve that any more than Amelia and her family did.

Is there Hope for Laura?

The other Burzynski success story that Burzynski supporters dare us to take on is that of Laura Hymas, whose website Hope for Laura. Her story is described on her website thusly:

Laura was diagnosed with an Oligodendroglioma, which is a rare brain cancer and in the UK there is no cure. The tumour goes deep into the brain and is in a location that is inoperable so our doctors applied a "watch and wait" approach which is standard for this type of cancer where you have a scan every three months to see if anything changes rather than applying treatment. However we recently had worse news in April 2011 Lauras MRI scan revealed growth and changes, she had a biopsy and we learnt that the tumour had progressed into a Glioblastoma Multiforme which is the most agressive type of brain cancer with a much poorer prognosis.

Many aspects of Laura's story are similar to Hannah's story, but one thing is different in that she started out with a less aggressive tumor, which was managed by "watchful waiting" for a time, until the tumor progressed. More details can be found at her page on the Burzynski Patient Group website. Laura started having more seizures; she was unable to care for her son; she felt as though her "life was slipping away," and so it appeared to be. Then, like Hannah, she decided to go to Houston to the Burzynski Clinic, and like Hannah and Pete Laura and her fiance started raising money, succeeding in raising £100,000 in just a few weeks from donations. What happened next is described here:

I have now been on antineoplaston therapy since the 8th August 2011. The side effects I have suffered are tiredness, a skin rash which subsided after a few days when I began treatment and a severe thirst! The medicine is rich in sodium and I have to infuse 2 litres of it daily (a dose which lasts 90 minutes every 4 hours 24/7) so I drink approx 5 litres of water daily. This is a very full on treatment; it isn't making me feel ill but while the pump is running it does affect my day to day decisions like for example going shopping or Ben is driving us to see family far away I need water to drink and a toilet close by! I am carrying around an infusion pump all day connected to my Hickman line in my chest. It's like having another baby!

To be honest though, for me it's really worth it. I won't be connected to the medicine pump forever! I have an MRI scan at a private hospital every 6 weeks. When we came home in August it took me until the middle of October to slowly increase my antineoplaston dose up to "maintenance dose" - this is the dose that Dr Burzynski deems is most effective for my body weight.

Then six weeks later on the 29th November 2011 scan my tumour started shrinking, by 36%. On the 10th January 2012 I had another scan - 56% tumour decrease!

I just had a scan on the 21st February and it was even better news - 77% tumour decrease!

As of August 2012 there is now no trace of my Tumor at all. I always get a second opinion from a UK radiologist who confirms there is just a cavity left which should resolve over time.

So is Laura's case proof that there's something to Burzynski's treatments? Is the fact that Burzynski has apparently discharged Laura as a patient slam-dunk evidence that Burzynski has cured her of her incurable tumor? Sadly, no.

For one thing, late complete responses to radiation are not as rare as Burzynski supporters imply, as described in this Medscape article on glioblastoma:

The responsiveness of glioblastoma multiformes to radiotherapy varies. In many instances, radiotherapy can induce a phase of remission, often marked with stability or regression of neurologic deficits as well as diminution in the size of the contrast-enhancing mass. Unfortunately, any period of response is short-lived because the tumor typically recurs within 1 year, resulting in further clinical deterioration and the appearance of an expansile region of contrast enhancement

Moreover, complete remissions in glioblastoma do occur. They're very uncommon—rare, even—but they do occur. There are case reports in the literature, such as this one, and a recent series describes the outcomes of long-term glioblastoma survivors as rare, but increasingly common. The bottom line is that we don't know why Laura is still alive. It's possible that it was the antineoplastons; it's possible that it was the radiation therapy alone.

Moreover, discharging a patient after her treatment for cancer is complete is something real oncologists almost never do right away. They usually follow their patients, often for five years, sometimes even longer. At this point, Laura is less than six months out from the first MRI scan showing no residual tumor, and she only just finished her antineoplaston therapy. Next, keeping an open mind I will admit that Laura's case is more suggestive of an antitumor effect due to antineoplaston therapy than Hannah's case is. What I find curious is the delayed effect. Laura started her therapy on August 8, 2011 but it was not until nearly four months later that the tumor showed evidence of shrinking. That's a long time for an active therapy to start to work, particularly for a tumor that is as aggressive as a glioblastoma. It's possible, but in general, even if antineoplastons really saved Laura, a treatment that takes three or four months to kick in is generally not that enthusiastically embraced by oncologists. Basically, Laura Hymas' case seems a bit more consistent with an antitumor effect due to antineoplastons than Hannah Bradley's case, but it is only a single case.

So what might be going on here?

Antineoplastons versus sodium phenylbutyrate

Back in late 2011, when I first took a serious interest in Stanislaw Burzynski and what he's been doing, I wrote a three-part series in which I (1) analyzed Burzynski The Movie, its claims, and whether there was any evidence that antineoplastons do anything for cancer; (2) discussed why Burzynski's claims that his "personalized gene-target cancer therapy" are overblown and nonsensical; and (3) how there might be a way to understand how antineoplastons might actually be real drugs, with the problem, of course, being that, when it comes to demonstrating efficacy, Burzynski is doing it wrong—very wrong indeed. They say that even a blind squirrel occasionally finds an acorn. Is it possible that antineoplastons are the acorn that Burzynski found?

I pointed out originally that antineoplastons A-2.1 and A-10 are nothing more than simple chemicals based on amino acids. Saul Green explained years ago, A-2.1 is nothing more than phenylacetic acid (PA), and AS-10 is phenylacetyl glutamine (PAG). According to a recent review article on a drug called sodium phenylbutyrate (PB):

Sodium phenylbutyrate (figure 1), a HDACI, is an aromatic fatty acid that is converted/oxidized in vivo into phenylacetate (PAA) by β-oxidation.[11] In humans the so formed PAA is eliminated by conjugation with glutamine to form phenacetylglutamine, which is excreted in the urine. This metabolic pathway is the mechanism by which phenylbutyrate acts as an ammonia scavenger in patients with urea cycle disorders (UCDs) and hyperammonemia.

If you peruse ClinicalTrials.gov for Burzynski’s current clinical trials, you’ll find that pretty much all of them use antineoplastons AS-2.1 and A-10; i.e., phenylacetic acid (PA) and phenyl acetyl glutamine (PAG). It turns out that PB is a prodrug for PA and PAG, which means that the drug is converted into an active metabolite to work. What Burzynski calls antineoplastons are nothing more than the byproducts of the body’s metabolism of the orphan drug sodium phenylbutyrate. In fact, according to this report, the “conversion of phenylbutyrate to phenylacetate was extensive (80 ± 12.6%), but serum concentrations of phenylacetate were low owing to rapid, subsequent conversion to phenylacetylglutamine.” In other words, phenylbutyrate is nearly completely converted to PA, which is then rapidly converted to PAG.

Remember my post about the use and abuse of the term "epigenetics" by various questionable practitioners, in which epigenetic effects and changes are invoked not unlike magic (or like another favorite buzzword "quantum") to "explain" why various woo works? In that post, I explained a bit about epigenetics and why it's a hot area in cancer research, as well as why histone deacetylase (HDAC) inhibitors are a promising new avenue for cancer therapy. I also puzzled about why HDAC inhibitors are considered "targeted," given that they have the potential to affect huge swaths of chromatin and the expression of the genes in the DNA therein, but that's just me. I guess I can't fault Burzynski too much for "talking the talk," even though it's quite clear that when it comes to targeted therapy he doesn't know what he is talking about, as has been amply documented. Indeed, Burzynski is so arrogant that he recently gave an interview in which he claimed to have been a pioneer — perhaps even the originator — of the concept of gene-targeted cancer therapy back in the 1990s. It's utter rot, of course, but Burzynski really did make that claim.

Perusing the article, I find there is a fair amount of preclinical evidence that PB has antitumor effects in certain tumor types, specifically colon carcinoma, Burkitt lymphoma, primary acute myeloid leukemia, retinoblastoma, prostate cancer, U138 MG, T98G, U373 MG and A-172 glioma cells, medulloblastoma, and hepatocellular carcinoma. Ironically, however, its ability to pass the blood-brain barrier is a problem, which makes it odd that Burzynski keeps using it for brain tumors. In these preclinical models, PB shows evidence of being an HDAC inhibitor, chemical chaperone, and pro-differentiation agent. The clinical evidence is much less impressive, however. Indeed, I reviewed the clinical evidence for PB as an anticancer therapy the last time I discussed this, and all I found was a bunch of phase I studies showing safety but no real efficacy. Of course, phase I studies aren't designed to show efficacy, but often investigators can get a hint of whether a drug is likely to have activity from phase I results. There was also a case report from 2002 showing a durable remission for four years in a malignant glioma treated with PB.

Given the impending release of Burzynski II, I decided to update my search, to see if there was anything more recent about PB and cancer. Of the references I found on PubMed, there were no new clinical trials published over the last 14 months since my last review. There were a few preclinical studies using cell culture and mouse models, but nothing new in human subjects. The state of our knowledge regarding PB and cancer can thus be correctly said to be more or less unchanged since late 2011. If Dr. Hideaki Tsuda, the Japanese anesthesiologist at Kurume University Hospital is correct when he states in the second trailer for the Burzynski sequel that he has done a randomized controlled clinical trial demonstrating the efficacy of antineoplastons in metastatic colorectal cancer, that would be the first convincing clinical evidence that antineoplastons (excuse me, PB) have significant efficacy in any cancer.

Of course, Tsuda hasn't published the results of his clinical trial, which makes me wonder why he's appearing in Burzynski II touting the results of his study when it hasn't been published yet. The last study I see from his group is a study from 2005 examining breast cancer cell lines, although there is a case study from 2003 looking at colon cancer that shows mildly promising results. As Elton John would sing, I've seen that movie too. Burzynski's been claiming he'll publish the results of his clinical trials for decades now. Tsuda has been collaborating with Burzynski for over 25 years. Am I going to hold my breath waiting for him to publish the results of this randomized clinical trial he's touting in the trailer? Not really. It's time for Burzynski and Tsuda to put up or shut up.

The bottom line

So we come full circle, back to the question of whether the cases of Hannah Bradley and Laura Hymas are convincing evidence that Burzynski's antineoplaston treatment works. Contrary to what Burzynski defenders claim, I started out agnostic regarding the question of whether antineoplastons have any value in cancer therapy. What I objected to was how Burzynski has continued to use them in patients and, in my opinion, abused the clinical trial process as a means of continuing to use them. Burzynski's motivations and ethics aside, considering these cases and their (so far) good outcomes from from a purely scientific and medical standpoint, we have three possibilities:

1. Spontaneous remission
2. Treatment effect due to conventional therapy
3. Treatment effect due to antineoplastons

Note that the second and third possibilities are not mutually exclusive. Both could be operative. Combination therapy is the rule these days.

Given that spontaneous remission is rare in malignant brain tumors, possibility #1 is highly unlikely, albeit not completely impossible. In Hannah's case, I tend to conclude that most likely possibility #2 is primarily at work here, although it's not possible to exclude a contribution from possibility #3. The reason I conclude this is that the shrinkage of the enhancing part of her tumor is not outside the range of range of effects that can be observed due to her radiation therapy in mid-2011 in that the enhancing mass of her tumor continued to decrease in size. Given the biology of her tumor, a year and a half since her radiation therapy was completed is too short a time to conclude that Hannah is an antineoplaston success story, particularly in light of her more recent reports that make me suspect that her tumor might have recurred. How else can I explain their rather evasive comments in their last two vlogs, coupled with their cryptic Facebook comment, a vlog that is apparently no longer on her website, and the fact that they have not shown any of Hannah's more recent scans, which is in marked contrast to what they did when her tumor was shrinking? I sincerely hope I'm mistaken. I don't want either Hannah or Laura to recur and sincerely hope that they are, indeed, disease free and remain so for decades to come. But just because either of them might be disturbed by my frank discussion is no reason for me not to discuss their cases, particularly given that they have both apparently agreed to assist Eric Merola in producing his latest propaganda-fest.

Laura Hymas is different in that she provides somewhat more suggestive evidence for a possible antitumor effect from antineoplastons, given the longer period of time since she finished her radiation therapy and since her still being in complete remission five and a half months after her first scan showing no residual tumor. However, her case is by no means the slam-dunk evidence that Burzynski supporters claim it to be (or, for that matter, that Merola touts it as in his upcoming movie), given that it has been less than six months since confirmation of a complete response. Moreover, given that HDAC inhibitors do seem to have some efficacy against glioblastoma, it is not unreasonable to expect that antineoplastons might actually have had activity in Laura's case. Making claims, as Burzynski does, however, that his antineoplaston therapy is more efficacious than conventional therapy is unwarranted based on a single patient. Conventional therapy can produce durable remissions and complete responses, too, and, although they are still rare, they are becoming more common. That's why legitimate randomized clinical trials are needed to determine if PB/antineoplastons have antitumor effects in humans; which tumors are sensitive; if there are any biomarkers of sensitivity; and to separate the signal from the noise. Anecdotes like those of Hannah Bradley and Laura Hymas can be suggestive, but in and of themselves prove nothing.

It's also why what Burzynski appears to be doing is an incredible disservice to cancer patients. If, in fact, he has a treatment that is so much more effective against "untreatable" cancers than anything we currently have, then he should have been able to demonstrate it by now. His excuses, in fact, are pathetic. He trots out the old alternative cancer treatment excuse that big pharma, the FDA, and the "cancer industry" are out to get him because they don't want a cure for cancer. You can see in Merola's second trailer that parroting this hoary old chestnut beloved of cancer quacks is going to play a central role in Burzynski II. Burzynski claims he has no pharma funding and no resources to do proper clinical trials, despite registering over 60 clinical trials since the 1990s and publishing none of them. This is in marked contrast to startup companies with fewer resources than Burzynski managing to take a drug concept through clinical trials within a few years. For example, contrast Burzynski's story to a story like that of PLX4032, which was taken through phase I and II trials rapidly and is now in phase III trials.

No, I don't buy Burzynski's excuses at all.

Burzynski is not doing what real scientists and clinical investigators do, and neither is Hideaki Tsuda. Publicizing their alleged research in a movie made by a Burzynski sycophant, toady, and lackey is just not the way science is supposed to be done, and his record, as demonstrated by the public record, appears dismal. Barring the publication of truly convincing clinical trial evidence by more sources than just Burzynski (given that Burzynski has already shown his methodology for conducting clinical trials to be questionable at best and that his preclinical data supporting his methods are at best weak and usually appear in publications that are not peer-reviewed), if we take in totality the evidence for the anti-cancer efficacy of PB along with all the evidence from the past 35 years for the anti-cancer efficacy (more properly, the lack of efficacy) of antineoplastons, the inescapable conclusion is that PB/antineoplastons might—I repeat, might—have very modest efficacy against some tumors through the HDAC inhibitor activity of PB. Whether that activity seen in some preclinical models truly exists and translates into human use and, more importantly, whether it is worth the not inconsiderable toxicity of antineoplastons as prescribed by Burzynski, are questions that Burzynski has, in my opinion, already shown that he will almost certainly never answer.