Tagged: juvenile pilocystic astrocytoma

Burzynski Patient Briannah O.’s Story

At four months of age, after seizures and becoming rigid and immobile, Briannah O. was diagnosed with a exceedingly rare brain tumor, a juvenile pilocytic astrocytoma. The tumor was inoperable and eventually the size of a grapefruit. It’s typically slow growing and while usually benign, it occupies valuable real estate. In the first two months after diagnosis, Briannah had two shunts put in to relieve pressure from fluid accumulating in the brain and had a very, very brief trial with chemotherapy. According to one of Briannah’s websites, in early 2007, the family was interested in going to the Burzynski Clinic.

Almost immediately, the family started to run into troubles with the Clinic. Brianna’s mother left a post on a message board on May 10, saying that she was unable to get any of her local doctors to agree to work with the Burzynski Clinic, whereas the “B. Clinic people are making me feel very pressured like it’s my problem.”

The antineoplaston treatment can only be administered, per an agreement with the FDA in the mid-1990s, under the auspices of a clinical trial. Burzynski opened over 60 clinical trials and in the nearly two intervening decades has only finished one and published zero.

One of the problems is that after the training period of a few weeks in Houston, Burzynski shifts the burden of administering the treatments for months onto the caregivers. The fact that the administration of the treatment is in the hands of well-meaning amateurs may have contributed to a finding the FDA made earlier this year:

You failed to protect the rights, safety, and welfare of subjects under your care. Forty-eight (48) subjects experienced 102 investigational overdoses between January 1, 2005 and February 22, 2013, according to the [trial number redacted] List of Hospitalizations/SAE (serious adverse events) [redacted] Overdose [redacted]/Catheter Infection report. Overdose incidents have been reported to you [….] There is no documentation to show that you have implemented corrective actions during this time period to ensure the safety and welfare of subjects.” [emphasis added]

Because people are administering this treatment at home, they need local physicians to take over the routine tasks of blood work and other local monitoring. It is not at all surprising to see that a patient would have a difficult time finding a knowledgeable physician refusing to work with Burzynski. When she says no doctor is going to “sign any documents that legally bind them to the clinic and that legally links them to be a  FDA co-investigator,” it is ironic, since according to an argument by which Burzynski managed to cling to his medical license last year:

“Respondent’s ownership of the clinic and his self-designation as the clinic’s chief physician on some forms, his ability to hire and fire everyone, and even that the forms which state that he is in “charge of treatment” (as stated in the informed consent forms for patient A) is only evidence of responsibility under vicarious liability theory, given the fact that the medical records detail exactly what doctors provided services to these two patients and who was involved in the delivery of medical care to these patients.”

It sure sounds like someone knows it’s a bad idea to take responsibility for patients at that clinic.

The same day as that previous post, Briannah’s mother posted that the “Barbara T.” at the Clinic explained that the family needed a FDA form 1572 to proceed. “Barbara T” was likely Barbara Tomaszewski, who was Burzynski’s business manager. According to Burzynski’s lawyer, during the period when Burzynski was indicted, she kept the clinic afloat:

“As the patient numbers started to decline, she came up with dozens of ways to cut costs and increase income. Some of the services that had been farmed out, like pharmacy supplies, were brought inside the clinic, and buildings were refinanced. She did whatever she had to do to squeeze or save every dollar she could.” (108)

I wonder how much of the current “everything but the squeal” business model of the clinic is a legacy of this woman?

Later that same day still, she heard the same message from Dr. Weaver. Dr. Weaver, by the way, was sanctioned in May 2014 by the Texas Medical Board for his behavior at the Burzynski Clinic, including:

Weaver1

Weaver2

Sure I could go into the problems that the FDA reported about how Burzynski’s institutional review board works, but who has the time? Luckily, there’s an entire Form 483 covered with numerous observations that suggest the board is unable to oversee a trial.

On the 17th of February, Briannah’s story hits the papers, as the family desperately scrambles for funds for Burzynski:

The [O family] and their friends and family are seeking financial help to get Briannah to Houston for the experimental therapy, which uses synthetic organic compounds called antineoplastons.

Dr. Stanislaw Burzynski originally developed the treatment more than 30 years ago, and has treated more than 3,000 patients with the intravenously injected compound, which essentially reprograms the brain tumor cells to die off and stop growing.

The procedure is considered experimental because Food and Drug Administration trials that have been ongoing since the 1990s are still under way. That also means it is a private treatment that insurance won’t reimburse. Just starting the treatment costs $20,000, and it can add up to as much as $200,000 over time.

Preliminary FDA trials show that of 226 brain tumor patients treated, 29.2 percent showed complete disappearance or more than 50 percent shrinkage of the tumor, 35.8 percent showed stabilization, while 35 percent had continued tumor growth, according to the clinic.

One wonders where these numbers came from and if they can be said to represent anything real. It’s hard to know since the FDA observed that 2/3 of the outcomes that they examined during an inspection were inaccurate, finding:

Burzynski (as investigator, the subject of the inspection) “failed to comply with protocol requirements related to the primary outcome, therapeutic response […] for 67% of study subjects reviewed during the inspection.”

This means that several patients who were reported as “complete responses” did not meet the criteria defined in the investigational plan, as were patients who were reported as having a “partial response” and “stable disease.”

At the end of April, Briannah has a shunt revision. The next week, she is up and bubbly, apparently healthy enough to travel to Houston:

The gift of little Briannah’s health comes at the perfect time, following a $22,000 anonymous donation to help start an experimental treatment at the Burzynski Clinic in Houston. Another $2,200 has been donated into an account established for Briannah.

But the struggle the [O family] began almost a year and a half ago, when their daughter was diagnosed with an inoperable and life-threatening brain tumor, is not over.

They need a local doctor to agree to be the “co-investigator” for Briannah while she is home from her treatments in Houston. And, in future months, they will need an estimated $7,600 per month to maintain the treatments.

Tammy said she is not a desperate mother grasping at straws, but a mother who has no other options for treating her baby’s condition.

The treatment is considered controversial in some medical circles.

This passage is simultaneously heartening and infuriating. The generosity of strangers is often amazing, and this is one of the biggest anonymous donations that we have seen since we started this website. Nonetheless, I am baffled that hundreds of patients have been looking for media attention to raise money for decades, and not one reporter has gotten off their backside to investigate whether or not their coverage of the issue would be a positive thing for the community until Liz Szabo put in months of legwork for her USA Today report, which appeared a few weeks ago. She and her editors should be congratulated for their brave decision to look more deeply into the Clinic, a courage that was recently honored with high praise from William Heisel on the Annenberg School blog: “Her story in USA Today, Doctor accused of selling false hope to families, is one of the best medical investigations I have read.”

On June 6th, a gallery of the family’s trip to Houston goes up.

On June 11, 2007, Briannah’s mom sent an email to a member of a now abandoned antineoplaston support group, who reposted it. The family had some connections in Houston (I think they lived there before), and after they flew down, spent some time reuniting with friends. Then they visited the Clinic. I turns out that while the staff was “delightful and helpful,” Briannah had not had the appropriate type of head scan prior to coming to Houston:

I was never told by the clinic in all the times that I spoke with them over the phone that it had to be a specific kind of MRI. I was just told that I had to have an MRI with in 2 weeks of our appointment.

At this point, I imagine that the family spends a couple of weeks learning to be independent caregivers for their daughter. Learning how to use the pump and prepare the bags. They stay with a couple who live near the clinic.

According to the local paper on 16 June:

[Briannah’s father] will return to Gilbert on Father’s Day, leaving his wife and their two other daughters in Houston for the first three weeks of the treatment, which could last an undetermined period of time.

“We don’t doubt that the medicine is going to work on her,” [her dad] said.

The treatment begins the same day that article runs. A scan posted in July shows the sheer size of this tumor. It dominates the center of the scan and is a terrifying monster. In August, the family is having a hard time keeping Briannah’s electrolytes under control. Her sodium levels are all over the place, and she’s experiencing the known side effects of antineoplastons. The Clinic claims they have never seen sodium plummet like this before.

At about the same time the Baby Briannah Foundation is incorporated.

On August 31 she had a scan. Two weeks later, the results are in:

This is the first MRI that we have had done since we started the Antineoplaston Therapy in June 2007. The report also stated that the ventricles remain dilated and have “minimally” improved since the previous study!!! […].

I am so grateful for Dr. Burzynski and his awesome discovery of the Antineoplaston Gene Therapy! I sincerely believe that because of these treatments Briannah is with us today and doing as well as she is.

One wonders how much of the improvement is due to the shunts that were fixed in April and how much this is to be expected from a slow growing tumor. There is also the question of how tumors grow–sometimes periods of stability just happen. Notice that it is not shrinking.

The local paper notes on Oct 8:

But since Briannah began experimental treatment in June in Houston, Texas, the youngest of Tammy and Mark [O]’s three daughters has experienced a lessening in the swelling and gained a lot of baby fat. For the first time, her MRI shows no growth in the tumor.

At this point, we’re going to guess that the improvement that we are seeing is related to steroids. Reduction in swelling? Gaining weight (for a baby that was failing to thrive before)? We’re guessing steroids. Burzynski’s patients are often treated with large doses of steroids, according to the FDA, and as reported in USA Today, patients were found to be on 5 to 65 times the anticipated dose of steroids. As the Clinic is basically a one-trick pony, there is no reason to suspect they changed their tune here.

Among the other revelations in this article, we learn that the treatment is costing $6,400 a month and that the little girl’s birthday in mid-October will double as a fundraiser. Also given the completely expected toxicities we saw above, the reporter still manages to open a sentence with the line: “The treatment, considered nontoxic….” I don’t think that the journalist did anything wrong, I just think that the family has a distorted view of what toxicity means.  

On the 11th of December, following another MRI, we are informed that the tumor has shrunk by 9%. It’s hard to take anything the Clinic tells a patient seriously when the FDA observed two-thirds of the reported clinical trial outcomes were apparently inflated. I’m not even sure how clinically significant that would be if it is an accurate result, especially as, according to the Szabo story, experts (which Burzynski is not) will disagree 40% of the time when when interpreting rare brain tumors. These results were reported uncritically in the local paper on 28 Dec. There is something of a vicious feedback cycle here. One must be completely and totally up front about the prognosis at all points with patients. They deserve that, especially when they are as vulnerable as parents as a child with a brain tumor. They have never had good news. ANY change, even “not getting worse,” is a HUGE victory, and expectations need to be managed. Yet the family reports:

“Right now, little Briannah is going to live a normal and a full life,” said [Briannah’s dad]. “We’re just really overjoyed.”

This is, of course, what everyone wants. But is it likely? And when it is reprinted in a paper, it’s difficult to see how that hope could not be massively reinforced, making what comes later all the more devastating.

In Feb 2008, Briannah seems to be having a crisis related to her sodium levels. She may be experiencing salt wasting, which can happen when there is injury to the brain. Via a friend of the family, we learn on Feb 7th:

“Baby Briannah was moved to PICU yesterday because her sodium levels dropped dangerously low. My daughter told me last night that Briannah was on IVs and her sodium level had come up some. Tammy and her husband, Mark, have made what was a difficult decision for them; they have decided to insert a peg (g-tube) for Brianna’s feedings. She has been getting her feedings with an NG tube for about 2 years now. Providing her Sodium level rises to normal today, the Broviac (Catheter) and Peg surgeries are scheduled for 2 PM this afternoon. That will be 3 PM Texas time.”

That night, we get an update on what is happening from Briannah’s mother. The catheter needed to be replaced and a gastric tube has been put in. At the same time, when she goes off of ANP, her sodium plummets. It may be the first time that ANP has actually pushed someone’s sodium in the right direction.

This child is clearly not on the path to the full normal life that everyone hopes for. And despite this trip to the PICU, the local paper reports in May:

Since the treatment began, mom Tammy [O] said the youngest of her three daughters has been happier and healthier than ever.

“We’ve been able to enjoy this year,” [her mom] said. “It’s been wonderful. No trauma. No injuries.”

Further, and perhaps not surprisingly, Briannah’s development has been arrested, though by all accounts she is a happy little girl:

Due to the tumor and a surgery when it was first discovered, she still can’t walk but she actively interacts with her parents and sisters, kicks, sits up and speaks baby talk.

We also learn that the father is turning his DJ business into a fundraising vehicle for Burzynski’s treatment, and in early June, another fundraiser is held at a farm where guests pick peaches to raise money.

In August, we get another optimistic assessment from the local paper, as well as this description of the treatment:

Briannah’s tumor has shrunk 20 percent since she began the nontoxic gene therapy treatment at Houston’s Burzynski Clinic.

The definition of gene therapy that makes this description of antineoplastons accurate would also redefine eating a banana as gene therapy. While the family did share some scans, I can’t find any of the results of this treatment. Further, she is still having some serious issues:

While she’s beginning to briefly sit by herself, doctors don’t know how much Briannah can see, and she also has been having seizures.

It looks like the family took a two hour trip to Sedona right before Christmas.

On January 26, a post goes up on another patients’ website:

An optic glioma, the type of tumor that James has, is considered a “benign” tumor. How deceiving that word can be. Too many believe that it means that this tumor will/can do no harm. Yet, I write tonight with tears flowing and a heavy heart because sweet Baby Briannah lost her life tonight to an optic glioma.

(In fact, it looks like she may have died on the 24th.)

As the family described her passing, the family were with their pastor at Phoenix Children’s when Briannah died.

The local paper described the last hours in more detail:

The fight included shunts placed in her skull to reduce swelling in her brain and, beginning in June 2007, an experimental gene therapy in which she was administered a synthetic organic compound through a tube.

It seemed to be working. The tumor was shrinking, and the once-emaciated Briannah had reached a normal height and weight, though she was unable to walk or talk.

Everything changed last week, when Briannah’s sodium level plummeted, causing her kidneys to shut down. She went into cardiac arrest twice.

“She left her body twice, and they brought her back after 22 minutes, and the second time after 18 minutes,” [her mother] said. “That in itself is a miracle, I’m not kidding you.”

But eventually, [Briannah’s parents] had to make the decision to take her off life support. “When I got to watch Briannah’s body deteriorate, it got to the point where it was easy to let her go,” [her mother] said.

It is difficult to reconcile the idea that the treatment seemed to be working and the poor little girl’s progression and death. We can say, however, that lot of patients have celebrated their fortunes while on Burzynski’s treatment when they in fact were progressing. One of the most scary things that we have found here is a pattern of descriptions of tumors breaking up on the inside and patients rejoicing, when the fact of the matter is that it is far more likely to be a sign of a tumor outgrowing its blood supply. And we’ve seen this for decades:

On February 2, the family celebrated the life of Baby Briannah, as she was known to the community that rallied around her. Her mother bravely posted an image from her daughter’s services on her website. This makes all the more galling the following post on the website:

staff

Usually, this is where we would put an appeal to donate to St. Jude’s. You may still do that, but we are now actively campaigning for an investigation into how the FDA decided to allow Burzynski not only to continue his ridiculous trials, but to actually get a phase III trial after a decade of abominable site visits. Go to thehoustoncancerquack.com and you will find the resources you need to put primary documents–the FDA inspection notes–into the hands of your representatives so they can conduct an investigation. All appeals to understand this made to the FDA have failed, so now we need to press the issue onto the committees that oversee the FDA. Please help us uncover what went wrong at the FDA so we can fix it and so this never happens again.