I was originally going to write about an amazing article that appeared in the NEJM today, but then, as happens all too often, something more compelling caught my eye. Unfortunately, it’s compelling in exactly the wrong way. It’s infuriating and saddening, all at the same time. It’s also yet another example of how it’s so very, very difficult to blog about these cases. Yes, unfortunately, it’s Dr. Stanislaw Burzynski again in the news, and this time around he’s managed to snare yet another unfortunate family into believing the hype about his “antineoplastons,” his “personalized, gene-targeted therapy” that isn’t, and his rebranding of an orphan drug. This time, the unfortunate family is all the way in New Zealand, and their sad story popped up on my Google alerts. Sadly, the reporting is incredibly credulous, with the headline screaming out, Hope for toddler with rare tumour:
Parents must raise $375,000 to take Jesse to US for radical treatment
Shane and Michelle Bessant say Jesse, 2, has an inoperable tumour on his brain stem and a new cancer therapy trial in the US is his only hope of survival.
Auckland parents Michelle and Shane Bessant are pouring everything they have into getting their 2-year-old son to the United States for experimental brain tumour treatment they see as his only chance of survival.
That’s about $314,393 in U.S. dollars as of when I write this. In other words, it’s a huge heap of change, out of the reach of most people. Another family is being victimized halfway around the world by Dr. Burzynski. They have a child with a fatal brain cancer. As I said before three and a half months ago, when I wrote about a similar case, that of Chiane Cloete in England, not having a child myself I can only imagine what that must be like, what the Bessants must be feeling, how desperate they must be to save their precious child. They’re so desperate that they’d be willing to pay anything, to bear any burden, to do whatever it takes. In this, they’re just like the family of Chiane Cloete We don’t yet know what Cloete’s fate has been. At least I haven’t been able to find anything more recent than last December, which is when I first wrote about the case. I do not know whether Chiane is still alive or whether she’s died. I do know that, as he has done so many times before, profited from the desperation of parents who went so far as to hold major fundraisers to try to obtain the funds necessary to have Burzynski treat their children, or, as I described it a couple of years ago, harnessing the generosity of kind-hearted strangers to pay for woo. It’s a recurring pattern with Burzynski that appears to be playing itself out again with Jesse Bessant.
Unfortunately, so is the credulous variety of reporting that doesn’t actually look into Burzynski’s treatment and the lack of evidence for it. In the news story about Jesse, we’re treated to a story structured in the common form of a quack testimonial. The parents are told that their child has a horrible disease that will kill him. Conventional doctors regretfully tell them that there is nothing they can do that has a high chance of saving his life but that they do recommend chemotherapy. Then…the parents discover the Brave Maverick Doctor, who offers them hope where conventional physicians cannot. His patter is convincing because he sounds very science-y and presents a story that sounds compelling on the surface. But most of all, his patter is convincing because, infused with the love of their child, these parents want to save him at all costs. That credulity is understandable in the parents of a child dying of cancer. It’s not so understandable in a reporter:
Then a family member alerted them to a cancer treatment facility in Houston called the Burzynski Clinic which is carrying out trials of a new therapy.
The treatment is called antineoplaston therapy, which has nearly completed its second stage of Food and Drug Administration-approved trials.
It involves injecting patients with synthetic chemicals called antineoplastons, intended to protect the body from disease.
“We were just gobsmacked,” Mrs Bessant said.
“People with no hope, who had high-grade tumours and had been told to go to hospice were being treated there and were surviving.
Except that there isn’t good evidence that they were or are. As I’ve described before, the testimonials that Burzynski presents to support his therapy are not particularly convincing. For all his claims of such fantastic results in clinical trials, the fact remains that Burzynski’s therapy hasn’t passed phase II clinical trials, and the results of these trials that I’ve seen reported are not particularly impressive or convincing. It’s true that Burzynski has a phase III clinical trial registered, but, the last time I checked, Burzynski’s trial was not open to accrual. So what we’re left with is a series of patients giving testimonials on the Internet because they underwent Dr. Burzynski’s treatment and lived longer than their doctors told them to expect. As I’ve noted time and time again, dead patients don’t give testimonials, although occasionally we do see evidence of a more typical course. In fact, I wrote about just such a patient a couple of weeks ago, a child named Brynlin Sanders, who also had brainstem glioma. Brynlin had an inoperable brainstem glioma, underwent treatment at the Burzynski Clinic, and died about three months after diagnosis. Like the Bessants, the Sanders family tried to raise money to pay for the treatment, although their treatment, for whatever reason, “only” ran $50,000, probably because Brynlin didn’t live long enough for Burzynski to get more. Meanwhile, here’s what the Bessants are doing to try to raise the money:
Speedway-mad Jesse Bessant, 2, had a blast watching the cars while other fans dug deep into their pockets to help get the potentially life-saving treatment he needs.
At 22 months old the Browns Bay resident was diagnosed with a rare brain stem tumour.
He is the first known case in New Zealand.
There is nothing that can be done for him here and Jesse’s family needs to raise $375,000 to get him treatment in Texas.
A fundraising event was held at the Rosebank Speedway on January 29.
Jesse’s mum Michelle says they raised $953 by walking around the crowds with buckets and asking for donations.
“Almost everyone donated,” she says.
Like the parents of Chiane Cloete, Brynlin Sanders, Billie Bainbridge, and a host of other highly unfortunate children suffering from inoperable brain tumors, Jesse’s parents are trying their best to do what they think is right for their child. Their love for their son has been channeled into a single-minded pursuit of the means to reach what they think to be a cure, and they’re doing whatever is necessary to get access to that cure, whatever the cost. What parents wouldn’t do that? According to the article, they need $100,000 to get Jesse to Texas and begin the first stage of his treatments at the Burzynski Clinic, of which they appear to have raised approximately $25,000, if their fundraiser page is any indication. They’ve done all that work, and they’re still only one-quarter of the way to the first milestone. It’s maddening because Dr. Burzynski’s therapy, such as it is, is certainly not better than anything offered in New Zealand, but his hype has convinced the Bessants that it is.
For all the similarities between Jesse and the other children I’ve mentioned, it should be noted that there is at least one big difference between Jesse and Brynlin, their both having brainstem tumors notwithstanding. Unlike Brynlin, Jesse is reported to have a tumor known as a ganglioglioma. I was unfamiliar with this tumor type, but its name suggested that it was probably not malignant. So I looked it up. According to the St. Jude’s website, gangliogliomas are “rare, benign tumors” that arise from ganglia-type cells and are also known as gangliocytomas and ganglioneuromas. The prognosis is described thusly:
Following a complete resection of the tumor, chances of long-term survival are near 100 percent. If the surgeon could not remove the entire tumor, sometimes radiation therapy is necessary. The prognosis still remains very high, 80-90 percent.
Malignant progression of ganglioglioma can happen, but it is rare, and even benign tumors like gangliogliomas can regrow after being surgically resected if they are not completely resected. In any case, this is how the Bessants describe their child’s plight immediately after they finally had a diagnosis:
Everything moved on very quicky from here, we felt like we were being shuffled out of the room, sorry Bessant family we can’t do anything for your son, thank you for your time, good luck and good bye. We hadn’t even had time to digest Jesse’s diagnosis when we found ourselves meeting Jesse’s Oncologist and discussing the merits of chemotherapy, brain shunts, hydrocephalus. The Oncologist did his best to explain the tumour to us, working in Jesse’s favour was that Gangliogliomas can be very slow growing low grade tumours, what was not working in Jesse’s favour was the location of the tumour in the brain stem, he made it very clear that chemotherpay and radiation treatment had shown no benefit in treating these Ganglioglioma’s overseas. Our best option would be to adopt a “wait and watch” approach, to monitor the tumour growth and only intervene if absolutely necessary or when Jesse’s condition deteriorates. Surgery was still out of the question due to the high risk of neurological damage, disability and fatality.
One notes that Jesse’s case had puzzled his doctors for a long time before the diagnosis was finally made. There is a long description of his symptoms, which apparently began shortly after birth but got worse a few months ago, the long and winding workup, and how even after the initial biopsy the doctors weren’t sure if this was a neoplastic process or an infectious process. Finally, as is often the case with rare tumors, it took consultations with experts overseas examining the biopsy to come to a definitive diagnosis. Most hospitals just don’t have the expertise to diagnose such rare tumors.
One thing that I need to emphasize here is that just because a tumor is “benign” doesn’t mean it can’t cause serious problems or even kill. As with real estate, location is key. If a “benign” tumor is growing in a location where its growth can impinge on vital structures (like the brainstem), erode into huge blood vessels (like the aorta), or obstruct vital structures (such as the trachea), its “malignant” or “benign” nature becomes almost a secondary consideration. In other words, in the wrong location a benign tumor can kill just as readily as a malignant tumor if it’s inoperable. In any case, while I was initially baffled by the recommendations of the oncologists in New Zealand, their reasoning became more clear when I looked up old medical school information about this tumor type and realized it is a benign tumor that’s generally slow-growing. Such tumors rarely respond to chemotherapy, although, depending upon the tumor type and how rapidly it’s growing, sometimes radiation will shrink them. The problem with such slow-growing benign tumors, of course, is that the efficacy of chemotherapy and radiation depends up the tumor having a higher growth rate that the cells in the surrounding tissue. The greater the difference in proliferation, the more toxic such treatments are to the tumor compared to the surrounding tissue. Benign tumors like this tend not to be growing much faster than the cells in the surrounding tissue. Also, because they are not fully malignant, they also tend not to lack as much of the DNA repair machinery that malignant tumors do, and it is defects in that machinery that contribute to the sensitivity of malignant cells to DNA-damaging agents, such as radiation and some forms of chemotherapy.
Once again, as I’ve pointed out before, writing a post like this is very difficult. It’s quite possible that the Bessants might see it, given that I’ve linked to their fundraiser page and given that it’s not uncommon for Burzynski parents to Google their child’s name in order to see what sort of news coverage they’re garnishing. I almost didn’t write this, given that I’ve written about Burzynski’s luring in desperate parents of suffering children facing a terminal illness on multiple occasions and sometimes received criticism that I’m somehow “kicking them when they’re down.” Such is not my intent. Such has never been my intent. However, it is my intent to warn others.
Burzynski’s treatments are unproven and most likely do not work. At the very best, his antineoplastons might have modest antitumor effects, but only at incredibly high doses. Moreover, as I’ve pointed out time and time again, Burzynski does use chemotherapy. He uses a lot of it. Moreover, his “personalized gene-targeted cancer therapy” is nothing more than using a commercial gene test to profile the tumor, then incompetently guessing at what targeted therapies might work based on that profile. It’s nothing more than throwing everything but the kitchen sink at the tumor, using cocktails of extremely expensive targeted therapies in combinations that have never been tested and might well result in synergistic toxicity. Meanwhile, Burzynski’s antineoplaston therapy has “evolved” into nothing more than the repackaging of an orphan drug as a cancer therapy. Meanwhile, because he is not allowed to use antineoplastons outside of the context of a clinical trial, he has a bunch of useless clinical trials that never end and rarely see print in a peer-reviewed journal indexed on Medline to which he signs up patients and charges them for the drug. This is, as I’ve pointed out before, generally frowned upon. Patients on clinical trials are not supposed to pay for the experimental drug or any treatments that are not routine standard-of-care. Finally, when bloggers start criticizing Burzynski for his activities, he hires hamfisted and incompetent reputation enforcers to threaten them with legal action, while on various discussion forums Burzynski disciples will swarm in like flying monkeys to complain to admins that criticism of Burzynski’s activities constitutes “abuse.” In addition, recently Burzynski has been sued for misrepresenting his clinical trials. That’s all on top of the hearing in front of the Texas Medical Board scheduled for April, in which he could very well lose his medical license.
I know the Bessants are suffering. We all hate to see a child suffer, and the parents among us can’t even imagine what they’re going through. The death of a child is generally one of the most painful events any parent can go through. Our children are supposed to outlive us. Certainly, they are not supposed to die after only two or three years on this earth. However, as hard as it is to say, I hope that the Bessants will realize that Burzynski can’t save Jesse any more than the doctors in New Zealand can, but he can extract huge sums of money from them and put Jesse through hell before he finally dies. As hard as it is to accept, it is likely that the best option for Jesse is exactly what his doctors have said, as unacceptable as that might be. The only other option I can think of that might hold out some hope would be to seek out Dr. Charles Teo in Australia. Complete resection of Jesse’s tumor is the only hope he has to survive, and the surgeons in New Zealand consider it unresectable. Dr. Teo might–might–be able to resect this tumor where other surgeons cannot. Certainly, if the Bessants are going to raise hundreds of thousands of dollars to try to save Jesse, that money would be much better spent on a treatment that might actually save Jesse. Burzynski’s antineplastons are not that treatment.