I would rather be doing anything other than writing this right now, but as Susan Gerbic has put it, advocates of science and evidence-based medicine have “drawn a line in the sand” and I don’t feel that we can yield an inch.
For the last few weeks, skeptics have been following the heartbreaking plight of the family of Refael Elisha Cohen, a 6-year old with medulloblastoma, a devastating brain tumor. According to the family, every single medical option has been tried–chemotherapy, surgery and radiation–but the monster has come back. At this late stage, there are compassionate options, palliative care and the relief of pain.
The family, understandably, is still looking for something, ANYTHING that might conceivably help their son. This desperation has sadly driven them into the hands of Stanislaw Burzynski at just the time that he needs a little good PR. The family has embarked on a campaign, appealing to the White House for a compassionate use exemption so that Refael Elisha can receive antineoplastons, Burzynski’s “signature” drug, which have been pumped into generations of cancer patients but have never met the most basic requirements of the scientific community. (In fact, we have seen 3 high profile campaigns simultaneously, unprecedented in the two years I’ve been following the clinic.)
While there are no demonstrable benefits of antineoplaston therapy, there are known side effects, about 3 pages worth, according to the Clinic’s own patient consent form. Because nobody wants to see a child die, a hundred thousand people have signed the petition to allow a compassionate exemption for Rafael Elisha. I share the generous sentiment of these signers, that a child deserves a regular life. Yet, knowing what that Clinic is after two years of continual searching for evidence that the treatment might work, having read the stories of literally hundreds of former patients, I can say without fear of contradiction that to support this family’s quest for antineoplaston therapy puts cancer patients in harm’s way.
I’ve largely remained silent about this campaign, which has featured prominently in the global Jewish press. It seems clear in the numerous reports that have appeared in the press that the family has not received accurate information about Burzynski, the treatment, or the prospects for that drug’s approval. An article came to my attention this morning that I felt I needed to answer, “Houston Boy Battles Brain Tumor; Needs Community Support.” written by the Campaign to Save Refael Elisha Cohen and which appeared in the online Jewish Voice. I hope that the two sentences I submitted in the comments will appear, but just in case they don’t, I figured that I would use my own venue as an opportunity to critique some of the points that were made.
According to the press release published at the Jewish Voice (which is all I can call this appeal, even if it is touching and sincere), a group of volunteers are collecting and sorting leads from around the world for potential treatments. I would like to offer them an additional source, if they have not seen it, clinicaltrials.gov, which lists all active registered clinical trials. Currently, there are 57 trials currently open for treatments into the type of tumor Rafael Elisha has. Any one of these trials is a better option than any illusory trial that the clinic might dangle in front of this family, and I hope they look into these options. I’m certain that Rabbi and Mrs. Cohen will understand that all anyone wants is for their child to heal, and if there was any evidence that the Burzynski Clinic had anything promising, that I would go down to Houston and hold the front door open for them.
In fact, there is one medical option which several prominent cancer researchers continually taught as the most promising for brain tumors.
The latter is called antineoplaston therapy, developed by Doctor Stanislaw Burzynski. The therapy uses peptides and amino acids’ and is manufactured in a block long pharmaceutical laboratory which operates directly under FDA Supervision. The FDA recognizes that the trials show efficiency, but has pulled its approval in 2012 pending reapproval possibly within the year. Current negotiation process is over interpretation of argumentative technicalities.
I would suggest that the statement that antineoplastons are “taught as the most promising for brain tumors” could not possibly be less true, as evidenced by this USA Today article, “Experts Dismiss Doctor’s Cancer Claims.” There is no evidence that any cancer, much less the intractable ones that Burzynski has claimed success for, are caused by “antineoplaston deficit,” which is the entire premise of the therapies. Lack of antineoplastons are simply not recognized as a cause of cancer. Secondly, as far as I can tell, Burzynski has never identified a therapeutic target for these drugs. Instead, we get vague words that sound nice like, “it turns off the cancer cells,” but we are not given an explanation of how that is supposed to happen. I’m willing to bet that if you were to ask the Cohen family how this drug is supposed to work, they won’t be able to tell you either. And if they have the mechanism, I’d honestly be eager to hear it.
The statement that “the FDA recognizes that the trials show efficacy” is purest bunk. There are no clinical trials that would demonstrate this. None. Burzynski has never completed and published a single clinical trial. The tumors he treats, especially the brain tumors, have a pretty high turnover rate. You would think that in over 15 years he might have managed to publish a single clinical trial, but he hasn’t. As part of a deal with the FDA 16 years ago, Burzynski agreed to only treat patients with ANP under the auspices of a clinical trial. So he opened dozens and never published a single finished one. We should not be surprised, of course, when his lawyer says of the Clinic’s trials:
[W]e decided to hit the FDA with everything at the same time. All of his current patients would be covered in a single clinical trial which Burzynski called “CAN-1.” As far as clinical trials go, it was a joke. Clinical trials are supposed to be designed to test the safety or efficacy of a drug for a disease. It is almost always the case that clinical trials treat one disease.
The CAN-1 protocol had almost two hundred patients in it and there were at least a dozen different types of cancers being treated. And since all the patients were already on treatment, there could not be any possibility of meaningful data coming out of the so-called clinical trial. It was all an artifice, a vehicle we and the FDA created to legally give the patients Burzynski’s treatment. The FDA wanted all of Burzynski’s patients to be on an IND, so that’s what we did.
Burzynski personally put together seventy-two protocols to treat every type of cancer the clinic had treated and everything Burzynski wanted to treat in the future. […]
This was in the spring, which means that it’s been about a year since Burzynski “completed” his clinical trials. Now it is his obligation to publish. If they work, any delay can be attributed directly to his not publishing his results.
Because the clinical trials are supposedly already “completed.” He just told you that himself. As of last month, at least, Liz Szabo at USA Today could report:
Even his staunchest supporters wonder why Burzynski’s drugs are nowhere close to receiving FDA approval. […]
In fact, the FDA hasn’t had a chance to approve Burzynski’s drugs. He has never officially asked.
Although Burzynski said he has completed 14 intermediate-phase studies, he has yet to file a new drug application, the final step toward getting a drug approved.
So, why the end run around regulation if his drugs work? Again, his treatment is “nowhere close to receiving FDA approval.” Why does this family believe otherwise? If nothing else, I would be keenly interested in knowing that.
The statement that the process of restarting trials is due to “current negotiation process […] over interpretation of argumentative technicalities,” is also tragically inaccurate. According to a warning letter issued 2 days after the Cohen family launched their petition to the White House, the issues are not mere technicalities. I quote Liz Szabo of USA Today again:
In letters to Burzynski and his research institute posted online Wednesday, the FDA says that Burzynski inflated success rates for experimental drugs that he calls antineoplastons. The FDA also says Burzynski failed to report side effects and to prevent patients from repeatedly overdosing.
The FDA placed Burzynski’s clinical trial on hold last year after the death of a 6-year-old boy, Josia Cotto, of Linden, N.J. The FDA also conducted several months of inspections of Burzynski’s research.
But when the FDA asked to see the child’s medical files, Burzynski sent the agency records that were different than those stored in his office, giving the appearance that the records had been altered, according to the warning.
Burzynski’s failure to keep accurate patient records “raises concerns about subject safety and data integrity, as well as concerns about the adequacy of safeguards in place at your site to protect patients.”
Ironically, the Cohens reside 10 minutes away from the Burzinsky clinic. They can see the meds that can potentially save their son even touch the medicine but cannot administer the antineoplaston due to the FDA clinical hold.
Firstly let us state in crystal clear turn the Cohens are rational, intelligent people. They have researched the Burzynski option on many levels and encourage people to watch the eponymous film “The Burzynski movie part 1 & 2”. In doing so one can readily comprehend why the Cohens are doing their utmost to obtain this treatment. Who within reason could blame them?
Nobody doubts this, but these poor people are also under duress and running out of time. The two Burzynski two movies are veridically worthless. Again, you have a handful of anecdotes from a few people who happen to have survived and no opposing views. This has been an effective recruiting tool for the desperate, but as this oncologist’s analysis reveals, the director clearly did not understand the patient files that were given to him. The second movie is simply dishonest by omission. Furthermore, the director is clearly a true believer and given to irresponsibly demonizing critics instead of taking into account contradictory evidence. Take for instance his comments about a prominent Burzynski critic who started his online skeptical career debunking Holocaust deniers:
Nobody blames the Cohen family for their petition or their desire that their son survive. However, the whole reason that decade-long record of overdoses, inaccurate outcomes, and make no mistake, untold millions of dollars raised and clearly wasted on apparently unpublishable clinical trials, were allowed to happen is because Burzynski’s desperate patients campaigned for him the last time he faced regulatory sanctions. So when those lobbying for Burzynski charge that skeptics:
actively speaking against the petition while strong arming others to follow suit are trying to directly hurt the compassionate work of over 80 strong volunteers who are working around the clock to aid the Cohen family
…they are correct, because it has happened before and untold hundreds of cancer patients bore the consequences of that kindness. Uninformed compassion can and has done immense damage in the past. It is my sincere hope that Refael Elisha Meir ben Devorah is healed entirely and he that does not suffer.
For people who want to understand why I and dozens of other skeptics are fighting, this video puts our campaign in context. Please watch it before accusing skeptics of being heartless: