I'm depressed as I write this, because I have some very sad news to relate to you. It appears that Laura Hymas died on Sunday morning. I learned this on Twitter yesterday from a source I trust, not to mention from mentions on Ben Hymas's Facebook page, in which friends are commenting on how they remember her. Apparently, her brain tumor recurred, and ultimately she entered hospice and was reported to be in her "final stages" a week ago. My sympathy goes out to her family. She died at far too young an age. No one deserves to have her life cut so short by such a deadly brain tumor, particularly a young mother recently married.
Hymas, as you might recall, has been held up as a success story by a doctor about whom I've blogged on numerous occasions over the last two or three years. I'm referring, of course to Stanislaw Burzynski, a doctor who is not an oncologist—and not even board certified in internal medicine—but claims to have discovered a treatment for the deadliest cancers that is far superior to the standard of care, even going so far as to claim to be able to cure a cancer that's never been cured before, namely diffuse intrinsic pontine glioma (DIPG). As I've pointed out before, this claim is false, at least the part about there being no record in the medical literature of anyone having ever survived DIPG. That's not the only—shall we say?—liberty with the truth that Burzynski and his supporters have used, but I'm not really in the mood to go too much into the detail of why I consider Burzynski to be a despicable human being, other than to mention how his supporters have used the story of Laura Hymas to promote his treatment with antineoplastons. Indeed, she is prominently featured in Eric Merola's latest paean to the brave maverick doctor in a segment in which a recording she took of a conversation between her and her NHS oncologist in which she and her family tried to persuade him to be the U.K. doctor monitoring her as she takes antineoplastons. It's a harrowing scene in that I saw a doctor trying desperately to do the right thing, despite extreme pressure from the patient.
As recently as November, Ms. Hymas was still being touted as a success story, cured by Stanislaw Burzynski when no one else could cure her, leading to credulous stories in the British press like this one, entitled Young mother with brain cancer given just a year to live BEATS the disease and gets married after having controversial treatment in the US. And, I must admit, that of all the Burzynski "success stories," that of Laura Hymas puzzled me more than most. Of all the many patient anecdotes presented by Burzynski and his propagandist as "success stories," Hymas's story was the one the came the closest to actually sounding as though there might be something to it, although I still couldn't rule out that her good fortune was a result of the conventional therapy she had before she discovered Burzynski. Then she dropped out of site not long after her wedding, and I got worried. I had a bad feeling about her, and I really hate it when my bad feelings turn out to be right.
As I said, I'm saddened to learn of her death. It doesn't matter that she was used by Burzynski. She really believed that Burzynski had saved her, making it difficult, if not impossible, for me to be too hard on her, just as I find it difficult to be too hard on any of Burzynski's patients, preferring to put the blame squarely where it belongs: On Burzynski. I also feel obligated to discuss her death. Given how many of Burzynski's previous "success stories" who ultimately turned out not to be anything of the sort were forgotten by Burzynski after their deaths, never to be mentioned again, instead to have all memory of them other than by their families wiped from the Internet and no mention of their deaths, we skeptics honor them by remembering them and, to the extent we can learn it, how they died, because it's certain that Burzynski and his supporters won't. In fact, they'll do their best to bury the memories of former "success stories" who died.
Just to show you how despicable the Burzynski propaganda machine is, though, I can't help but point out that it is still going full tilt. Just yesterday, as the news of Laura Hymas's death was hitting Twitter and Facebook, the pro-quackery organization known as the Alliance for Natural Health (ANH) used Burzynski again to promote yet another bad, bad law known as the Compassionate Freedom of Choice Act:
Recently, by working together, we made great strides in helping dying patients win access to potentially lifesaving treatments. It was your messages, together with those of other grassroots activists, that convinced the FDA to grant 12-year-old McKenzie Lowe a right of access to Dr. Stanislaw Burzynski’s innovative antineoplaston treatment.
As you may recall, the FDA had insisted that the treatment go though the agency approval process. Although the treatment had proven effective in earlier years and had passed the agency safety tests, it had not completed the whole process. The FDA had the authority to grant “compassionate use” to patients who asked for it and would clearly die without it, but chose not to grant it.
Despite our recent victories, the fact remains that the compassionate exemption rule is broken beyond repair—after all, these patients needed government permission to try to save their own lives, and the government couldn’t care less.
That’s why we’re thrilled that last Thursday, Rep. Morgan Griffith (R-VA) introduced the Compassionate Freedom of Choice Act of 2014. The bill eliminates the requirement for an FDA okay, and streamlines physician and patient access to experimental drugs. It would ensure that dying patients and their families—who don’t have the time or the strength to do so—won’t have to wage fruitless bureaucratic wars.
The ANH is describing, first, how the FDA caved under pressure, in essence wiping its hands of the issue of whether to allow compassionate use exemptions (a.k.a. single patient INDs) for Burzynski's antineoplastons. Basically, the FDA ruled that, even though Burzynski has been cited numerous times for serious FDA violations and his antineoplaston trials are on partial clinical hold, patients can still receive antineoplastons on a single patient IND, just as long as the physician administering them is not Stanislaw Burzynski or someone who works for him. As I pointed out at the time, it was an astoundingly cynical decision and a massive propaganda victory for Burzynski.
But what about this H.R. 4475, this "Compassionate Freedom of Choice Act of 2014"? Unfortunately, the text of the bill is not yet published. That means we can only take the word of Rep. Griffith and speculate. To me, this bill sounds as though it could be a lot more problematic than the "right to try" bills I discussed not too long ago. Those bills, as I mentioned, are state bills. They don't change federal law, and the FDA would still have jurisdiction over anything that had any interstate commerce component whatsoever. Unless a pharmaceutical company is in the same state as the patient, the FDA has jurisdiction. Actually, the FDA has jurisdiction anyway, because it has jurisdiction over drug approval in the US, and no drug company is likely to grant access to unapproved pharmaceuticals if it thinks that it will harm its chances of approval for its drug. So changing the law for a state is unlikely to have much of an effect in terms of opening up access to experimental drugs. Changing federal law, on the other hand, could make a difference.
The law, as described, is designed to "allow the manufacture, importation, distribution, and sale of investigational drugs and devices intended for use by terminally ill patients who execute an informed consent document, and for other purposes." According to Griffith:
The Compassionate Freedom of Choice Act removes disincentives for drug companies to grant exemptions. For example, the current application for expanded access requires a drug company to share information—which we all know drug companies don’t do very well. It also requires, among other things, a detailed description of how and where a drug will be produced; complex pharmacological and toxicological information; and, if enough patients ask for the drug, a whole new Investigational New Drug (IND) application by the drug company.
Under the new bill, drug companies can still deny dying patients access to experimental treatments. But if they don’t have the FDA in opposition, drug companies may be much more willing to say yes. For example, drug companies are often afraid that if a patient dies under expanded access treatment, it will affect the eventual approval of the drug. With less risk and less paperwork, drug companies are more likely to increase patient access to experimental drugs.
It's hard to tell exactly how this bill would affect access to experimental drugs without knowing what "disincentives" it removes and its specific language. I can say, however, as I've pointed out before that in the case of most experimental drugs that have only passed early stage testing (as in phase I clinical trials), the likelihood of doing no good or even causing harm is likely to be much higher than the likelihood of doing any good. Such a bill, depending on its wording, has the potential to do great harm. Moreover, such bills are custom-made to allow someone like Stanislaw Burzynski, who has long been supported by the ANH, continue to ply his trade.
Another way that we can honor Burzynski patients like Laura Hymas is to do our utmost to make sure that doesn't happen.
And at the same time, Burzynski appears to have published
Based on the description of the bill, this seems like something that pharmaceutical companies and quacks of all stripes would love to see passed. The FDA already lacks teeth to do what they're charged with doing. Why try to rip out their gums, too?
Hmm...40 patients enrolled, but the paper is only focusing on 17 of them. Non-blinded study. No control group. Can't wait for Orac to sink his teeth...err...blinky lights into this one.
@Todd - I see that he's still using the whole "stable disease" category, which I believe, based on what Orac has written in the past, isn't even a designation normally used to classify patients in Cancer studies.
And this would be overseen by the same IRB that is packed with shareholders of the Burzynski Clinic, right?
From the link:
The study was designed as a single-arm, two-stage, phase II trial of ANP as monotherapy. The study was listed by the National Cancer Institute (NCT00003459). It was supervised by an independent Institutional Review Board (BRI-IRB, BC-BT-11).
Yep. Same IRB packed with Burzynski's COI-riddled cronies.
Both researchgate and pubmed (and probably others) now permit to comment and evaluate the quality of an article... I wonder what would be the effect of commenting an article such as Burzynski's there by pointing out methodological, ethical, or other scientific issues.
Hmm…40 patients enrolled, but the paper is only focusing on 17 of them.
Yet, at least in the abstract, most percentages are given to the nearest 0.1%. Note that with a sample size of 17, one patient would be about 6%. Do they not teach clinical researchers (two or three of whom presumably reviewed this paper) the concept of significant figures? I'm not qualified to evaluate the medical content (I'm a physicist, not a physician), but that alone would have been enough for me to raise a warning flag.
Eric-I'm a physicist (well BS level) now a physician. I was underwhelmed with how poor statistics were taught in medical school (unless you went to grad school for mph/phd).
Any indication whether the subjects involved in this study are among those whose MRI records and tumor measurement worksheets the FDA discovered had been destroyed? If their records aren't examinable--if all Burzynski can offer is his personal assurance that he complete and partial responses, or 'stable disease' however he defines it occurred--he might as well have saved time and effort by not publishing.
From newspaper reports, Colorado is also considering a Compassionate Freedom of Choice Act. So far it has been amended to require that the medications be provided free or at manufacturer's cost. Loopholes may still exist, however.
This appears to be the text of the bill.
I see no evidence that Child's Nervous System is peer-reviewed, just open access. Their acceptance criteria is pretty slim, only asking for limited IRB approval for subject testing.
But perhaps I'm missing something and I'm incorrect. Anyone else know more about that Journal?
When I Googled the impact factor, here is what I got.
YearImpact Factor (IF)
I am not a scientist, but I know that is low.
I wonder if Laura's sad story is the reason why Hannah Bradley's latest blog from February is such a tear-filled appeal to support Burzynski, as they wonder what would happen if her tumor came back. My heartache for these people is commensurate with my disgust for Burzynski's deceit.
I'm so sorry to hear about Laura. Such a tragedy.
As for the paper, it says that 5 of the 17 patient sample survived longer than 1 year after start of ANP and only two of those patients survived longer than two years after start of ANP. One of the two passed away around 47 months after start of treatment, and the other is still alive 15+ years later.
I sincerely hope that their disclosure of her identity was with her consent. If Burzynski let slip her identity as one of the study subjects, that violates subject confidentiality. But then, we already know that ethics is just a jumbled of letters to Burzynski.
"I am not a scientist, but I know that is low."
I am a scientist, and I can say that that isn't necessarily low. Especially a 5 year average. Specialty journals never get really high impact, because they don't get cites outside of their specialties very often. That doesn't mean that the content is sub-par automatically, though. It appears that the Child's Nervous System publishes a lot of case studies, things that wouldn't really require peer-review, as case studies aren't widely recognized as something worth citing.
I still can't find evidence that bigger research articles do get peer-reviewed, though. I think this is starting to look like a pay-to-play journal.
@Todd W and Mike,
Since they've referred to her in the movie and multiple web sites, it seems that cat is already out of the bag.
But, this site
Jessica Ressel's medical records are published by written authorization by Jessica Ressel-Doeden.
But then, it also claims
Of the patients treated with antineoplastons with this type of tumor: 27.5% of them were cancer-free at the end of treatment, with 27.5% of the patients living at least 5 years after diagnosis.
From Orac's analysis, she was the one with no biopsy and tumor sizes that bounced up and down.
And, why did they leave out the other 10 who survived longer than 5 years???
No comment. The story and the evidence should speak loudly to all rationally-minded people.
I think this is starting to look like a pay-to-play journal.
Which would be distressing news if true, because Springer is supposed to be one of the more reputable publishers. (My name is on a paper published in one of their journals--they acquired that title in a merger sometime after that paper was published.) I wasn't aware that medical case studies are usually not peer-reviewed (comparable papers in my field usually are), but even so, this study involves more than a few patients. I can understand the claim about approval by Burzynski's packed IRB sliding through (most reviewers who aren't themselves familiar with Burzynski would presume that the IRB is genuine, just as they would presume that the described experiment was actually performed), but there should have been some form of peer review on this paper. OTOH, I have read more than one paper where my reaction was, "How the $&@% did this get past the referees?" so I shouldn't be surprised if large amounts of that occur in the biomedical world.
Mark - that's from 2012, though it appears to be identical to the 2014 version.
The text of HR4475 is up now, though, and I found <a href="http://www.gpo.gov/fdsys/pkg/BILLS-113hr4475ih/pdf/BILLS-113hr4475ih.pd… link via THOMAS.
... and evidently I can't do HTML today.
Sadly, not in time to help...
This DCA-based molecule is small enough to get past the blood brain barrier.
Seven years and counting since its discovery as an anti-cancer agent. Seven years it has been known it reactivates the mitochondrial apoptosis mechanism. At this rate, because there's no big money in big pharma for this compound, it will be seventy and seven years before there's any approved drug available.
The text of HR4475 is up now, though
That's really poorly drafted:
"In order for an investigational drug or device to be intended for use in accordance with this subsection, such drug or device must be intended for use...."
Section 561A(c)(1) would seem to tank it, because there's no apparent way to enforce § 561A(b).
Moreover, considering § 561A(d), this would represent blanket permission to import psilocybin.
^ Ah, scratch that; it's only the FDA.
However, the field preemption of § 561B is quite unlikely to fly. In fact, it might be straight-up unconstitutional.
I am really concerned with the move in American politics today to brand everything related to government 'bad' while 'freedom' dictates that whatever anyone wants to do is fine. I really hate the thought of these bills putting false hope in the hands of desperate people willing to try literally anything to cure themselves. We are rapidly deregulating ourselves back to the day when anyone could slap a label on a bottle of well anything and claim their formula was a miracle cure for everything! Granted, DSHEA has given the quacks plenty of leeway in this area but allowing everyone else in on the fun is a really bad idea. How many people are going to have to die from these treatments before the public gets outraged? I am saddened to think that no one will care if they die, after all weren't they going to die anyway? Who cares if they poured out their life savings, borrowed from friends and family, and spent their last weeks in pain with unanticipated side effects? This makes me really sad. I also offer virtual condolences to the family. It is tragic to mourn any death but the death of a young person in prime of life is heart breaking.
I commented on Alliance for Natural Health's website about the young lady's case, but, of course, it is awaiting moderation. Though this is their claim:
"ANH-USA provides a comment forum for our readers to share their constructive thoughts and criticisms about our newsletter articles and engage in civil debate with other readers. All comments are pre-moderated regardless of author. We never censor comments based on political or ideological point of view."
I'm willing to bet my comment never makes it through. This is what I posted over there:
"Yes! Let’s give him more victims! You know, like Laura Hymas. You should remember her. She’s the one these people said Burzynski cured in November. She died 13 Apr 2014. Of the very thing he claimed to cure her of. https://twitter.com/Roochella/status/456050899950243841
Remember her. None of these folks will."
Any takers on this wager? ;)
I see no evidence that Child’s Nervous System is peer-reviewed, just open access.
It's not OA; it has an OA option, which is pretty common these days.
Of course, the received date indicates precisely what it takes to get Scamislaw to disgorge 20-year-old data.
Groan - the comparative mris are of different transverse sections of the brain. Come on - that's just sloppy, SB!
My profound condolences to the family and friends of Laura Hymas, who are grieving her loss.
That's all I feel I should say at this time.
R. W. Foster, I would take the other side of that bet. As long as you're civil and stick to the facts, they will post it. I've submitted several postings critical of some aspects of the supplement business, and none were censored. In earlier threads, ANH did indeed post submissions that called out Burzynski for his quackery. You should not assume one-sided censorship in the absence of data.
The moderation time is somewhat long and unpredictable. Your posting might show up tomorrow or next week. I believe it will show up, sooner or later.
At this rate, because there’s no big money in big pharma for this compound [DCA]
It's nearly three years since Orac got bored with posting about DCA. Executive summary: it would be highly profitable for Big Pharma if it worked, but it doesn't, and only has massively toxic side-effects.
David Springer repeats the old claim about absence of research into DCA (due to non-profitability) while linking to an article about a new, patentable, potentially-profitable variant of DCA invented by researchers.
I contacted the editor to inquire about, you know, what the hell. His response:
From S Petersburg
I have invited eminent neurooncologists to write a comment to be added to the articles. Unfortunately the reviewers of the journal failed to provide their promised evaluation so that after several months there were 'no negative comments" to justify rejection. Now I look for receiving comments from Canada and UK to be published in the journal. Should you be willing to contribute you are welcome.
Not. Peer. Reviewed.
Interesting. B. finally published a paper. Well that's something, isn't it? Phase II is only a single arm, so is not expected to be definitive, but if promising could support the idea of phase III RCT. If survival results look promising, it is always a question of whether you had selected patients with better outcomes or whether the drugs caused better outcomes. But response is somewhat immune to this. How do the data look? Well, not as good as reported, actually.
A few oddities:
(1) The one "Complete response" (CR) isn't, in fact, a CR. The authors have an abnormal MRI for patient 8 and they inexplicably and post-hoc decide to use a "negative" PET to declare CR in just this one patient. No other PETs are done, apparently. Well the whole brain takes up glucose if you are alive, so the PET is not really a very useful way to assess response. In fact, PET uptake can't be used to measure tumor response by standard criteria because it's a measure of glucose uptake not tumor size. Moreover, the tumor is clearly present in patient 8 at the bottom of Figure 2... and is listed as present but "decreased" in table 2. This is not a CR.
(2) Illustrated responses don't actually illustrate response. Figure 3 compares before-and-after images of completely different sections of the brain (notice eyes in bottom; sections above eyes at top), so we cannot see if the tumor changed size. In figure 2, ignoring the claimed CR, partial response even is suspect. Most of the changes appear to be decrease of swelling (shows up white on T2 and dark on T1) which could be attributed to steroids.
(3) Still using steroids. The FDA recently cited B for not following protocol and assessing response for patients on steroids. This is because reduced swelling from steroids can be mistaken for smaller tumors. The authors only specify that steroids above a certain unspecified threshold cannot be used for CR assessment. It is apparently used for PR, which makes one wonder whether observed PRs--decreased swelling-- were from steroids or drug.
(4) Incomplete study This study is not complete. If it was a 2-stage design as reported, why wasn't it designated as such on its CT.gov record, which lists enrollment plan of 20-40 subjects? Either way the study is incomplete-- didn't enroll 20 and, if 2-stage design, the authors need to specify the criteria for going on to stage 2. Were the criteria met? If so, then the study is not actually complete. If not, then the study is terminated due to lack of efficacy.
Some other loose oddities are the many changes in the protocol, >10 years to accrue, reporting about 8 years after collecting data, and that some patients were not evaluated at 12 weeks as specified by the primary endpointaccording to their CT.gov registration. The reported grade 4 toxicities contradict the idea that ANP is non-toxic, and given history, may be underreported.
Finally the one long-term survivor (apparently case 8) had survived 5 years prior to starting the therapy, suggesting the nature of this unusual disease could account in some part for the long survival compared with typical DIPG.
Nice that B. finally published a clinical trial. But it actually a partial trial again... and results are underwhelming.
Unfortunately the reviewers of the journal failed to provide their promised evaluation so that after several months there were ‘no negative comments” to justify rejection.
That's a curious way to run a journal.
I am guessing Elsevier might need to know about the quality control of this journal.
Please direct comments regarding the recently published Burzynski clinical trial (or, as I like to describe it, the 42.5% of a clinical trial) to the post in the link above and do not continue discussing it in this comment thread.
Of course it's a success story.
Burzynski got paid.
btw, if someone has spare time, it would be very useful to assemble a scrapbook of statements and web clippings for each of the "success" stories before they disappear from both the web and cache (they can ask to be removed from wayback, etc). It would have much more impact if one can see the Bz campaign for each victim before its sudden end (along with the mark and their money).
@ Mike -- that's good to see!
Would you have been less saddened had she died from FDA approved Toxic Chemo??
Oh look. Another one who doesn't realize what Burzynski offers *is* "toxic chemo".
We mourn her death however it happened.
But, it is especially sad because her case has been used as a "success" to convince other patients facing cancer with a bad prognosis to raise tens of thousands of dollars to pay Burzinski up front to experiment on them.
Apparently Burzynski offered "gene therapy". Hmmm...
Her husband's comments are pretty depressing. It seems he still thinks the treatment worked and they wanted more of it.
Of course the treatment worked. The transfer of funds from his wallet to Burzynski's bank account was completely successful.
Ok, snark aside -- I'm not sure one can humanly expect the husband to act otherwise. It would be tantamount to admitting that he knew the two of them had invested so much time, money, etc in something that turned out to be junk.
Eric Merola finally acknowledges Laura's death on his movie's website here:
but then he very quickly moves on to the news of the newly-published "peer-reviewed" (NOT) Burzynski study:
The majority of the blind opposition to Antineoplastons comes from the prejudicial prerequisite of having the Phase 2 trials published in the peer-reviewed literature. Well, that argument is slowly becoming less and less valid.
A diffuse intrinsic brainstem glioma in a child (DIPG) has never been cured in a controlled clinical setting in medical history. Antineoplastons are the first in world history to have ever cured it.